List of Cell Therapies Companies with Phase 2 Active Clinical Trial - 162
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A2 Biotherapeutics Agoura Hills, California, United States | A2 is dedicated to pushing the boundaries of science to transform medicine and turn the tide on cancer. We are focused on the next frontier in cell therapy: solid tumors. A2 engineers T cells that target the loss of genetic material in tumors, enabling the selective killing of tumor cells while leaving normal cells unharmed. The company was founded in 2018 by Alexander Kamb, Michael Gallo and Paul Kang and has raised $136M since inception. Our investors include The Column Group, Vida Ventures, Samsara BioCapital, Nextech Invest, Casdin Capital, Euclidean Capital, UC Investments (Office of the Chief Investment Officer of the Regents) and Hartford HealthCare Endowment. |
AbClon Seoul, Republic of Korea | AbClon was founded in 2010 by a consortium of Korean and Swedish experts in antibody development. Our mission is to cure once incurable diseases such as cancer. |
AbelZeta Rockville, Maryland, United States | AbelZeta is a global clinical-stage biopharmaceutical company with centers of excellence in Rockville, Maryland and Shanghai, China. AbelZeta is focusing on developing innovative and proprietary cell-based therapeutic products and is committed to ushering in bespoke treatments that harness the body's own immune system to fight against hematological malignancies and solid tumors, as well as inflammatory and immunological diseases. AbelZeta advances research and development in its own GMP facilities at its centers of excellence for early-stage clinical studies, with a pipeline comprised of CAR-T and TIL therapies. |
Adaptimmune Therapeutics Philadelphia, Pennsylvania / Oxfordshire, United Kingdom | Adaptimmune is a fully integrated cell therapy company, designed and built from the ground up with four U.K.- and U.S.-based biotechnology hub locations. Our comprehensive capabilities and teams include preclinical research, clinical development, translational sciences, autologous and allogeneic manufacturing, and in-house commercial and corporate operations. For more information about Adaptimmune, please visit www.adaptimmune.com. If interested in joining our fast growing team, click on the careers tab of this page. |
Adaptive Biotechnologies Corporation Seattle, Washington, United States | Adaptive Biotechnologies Corporation, a commercial-stage company, develops an immune medicine platform for the diagnosis and treatment of various diseases. The company offers immunoSEQ research service and kit that is used to answer research questions, as well as to discover new prognostic and diagnostic signals. It also provides clonoSEQ diagnostic tests, which include immunosequencing services for use in the detection and monitoring of minimal residual disease in patients with select blood cancers. In addition, the company offers a pipeline of clinical products and services that are used for the diagnosing, monitoring, and treatment of diseases, such as cancer, autoimmune conditions, and infectious diseases. Adaptive Biotechnologies Corporation has strategic collaborations with Genentech, Inc. for the development, manufacture, and commercialization of neoantigen directed T cell therapies for the treatment of a range of cancers; Microsoft Corporation to develop diagnostic tests for the early detection of various diseases from a single blood test; and Amgen to develop a therapeutic to prevent or treat COVID-19. It serves the life sciences research, clinical diagnostics, and drug discovery customers. Adaptive Biotechnologies Corporation has a translational collaboration with AstraZeneca to investigate the use of immunoSEQ T-MAP. The company was formerly known as Adaptive TCR Corporation and changed its name to Adaptive Biotechnologies Corporation in December 2011. Adaptive Biotechnologies Corporation was founded in 2009 and is headquartered in Seattle, Washington. |
Adicet Bio Boston, Massachusetts, United States | At Adicet (Nasdaq: ACET), we are defined by our mission to deliver best-in-class gamma-delta T cell therapies for patients fighting autoimmune diseases and cancer. Every one of our team members, whether in the lab, manufacturing, or general business role, comes to work every day with the patient in mind. As pioneers in the cell therapy space, we are honored and humbled by the opportunity to put our talents to bear in developing transformative therapies to defeat autoimmune diseases and cancer. We believe that the best way to do this is through discovering and developing allogeneic gamma-delta T cell therapies, a novel type of cell therapy. Adicet has chosen the gamma-delta T cell because it: • Preferentially traffics to tissues and provides B cell depletion, potentially leading to an immune reset for patients with autoimmune diseases. • Has multiple ways to target and kill cancer cells including solid tumors. We also chose to use cells from donors (allogeneic) to enable our potential therapies to be readily available when needed (taken “off-the-shelf” or available to the patient on demand). We’re not just blazing new trails in cell therapy, we’re having fun along the way. With regular team gatherings, many shared meals, larger company events, and a consistent commitment to our values, we have a strong culture! We are proud to attract and retain some of the greatest talent in the biopharma and cell therapy space, and we’re consistently giving back to our employees. The Adicet benefits package includes generous financial compensation, comprehensive health plans, generous paid time-off, educational reimbursement, and access to many amenities in our state-of-the-art facility. To learn more or inquire about joining our team in Boston, MA or Redwood City, CA, please visit http://www.adicetbio.com |
Aegle Therapeutics Woburn, Massachusetts, United States | Aegle Therapeutics Corp. is a clinical-stage biotechnology company developing novel extracellular vesicle (“EV”) therapies to address rare and serious diseases and disorders with significant unmet medical needs. Aegle’s proprietary platform technology safely isolates native extracellular vesicles (“EVs”) from stem cells. These EVs carry complex assemblies of biologic molecules such as proteins and nucleic acids that can induce a wide variety of effects in recipient cells, including the promotion of regenerative healing, while reducing inflammation and modulating the immune system. Aegle’s EV therapy has the potential to treat a broad range of indications in multiple therapeutic areas, including dermatology, immunology-based diseases, protein deficient disorders and others. |
Affini-T Therapeutics Watertown, Massachusetts, United States | Affini-T is a leading precision immunotherapy company targeting core oncogenic driver mutations to develop potentially curative therapies for patients with solid tumors. Our differentiated cell therapy platforms harness state-of-the-art engineering, synthetic biology, and gene editing capabilities to target even the most devastating cancer-driving mutations, beginning with KRAS. We leverage these tools to optimize T cell functions and rewrite the rules of the solid tumor microenvironment, enabling the potential for sustained clinical outcomes in patients. Building on the world-class innovation inherent in our leadership team, founders and technologies, we are powered to develop transformational medicines that last. |
Akouos Inc. Boston, Massachusetts, United States of America | Akouos is a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for people worldwide who live with disabling hearing loss. |
Alaunos Therapeutics Houston, Texas, United States | Alaunos Therapeutics (previously Ziopharm Oncology, Inc.) a clinical-stage biopharmaceutical company, focuses on discovering, acquiring, developing, and commercializing a portfolio of immuno-oncology therapies to treat patients with cancer. The company develops Sleeping Beauty platform, which is based on the non-viral genetic engineering of immune cells using a transposon/transposase system to engineer T-cells outside of the body for infusion; and Controlled IL-12 to stimulate expression of interleukin 12 or IL-12, a master regular of the immune system, in a controlled manner to focus the patient’s immune system to attack cancer cells. Its product candidates include T cell receptor + T therapies to target solid tumors; chimeric antigen receptor + T cell therapies targeting CD19 for hematologic malignancies; and Ad-RTS-hIL-12 plus veledimex, a gene delivery system to regulate production of IL-12 to treat patients with recurrent glioblastoma multiforme in adults. The company has a license agreement with PGEN Therapeutics, Inc.; research and development agreement with The University of Texas MD Anderson Cancer Center; a patent license agreement with the National Cancer Institute; and a cooperative research and development agreement with the National Cancer Institute. The company was formerly known as ZIOPHARM Oncology, Inc. and changed its name to Alaunos Therapeutics, Inc. in January 2022. Alaunos Therapeutics, Inc. is based in Houston, Texas. |
Allogene Therapeutics South San Francisco, California, United States of America | Allogene Therapeutics, Inc., a clinical stage immuno-oncology company, develops and commercializes genetically engineered allogeneic T cell therapies for the treatment of cancer. The company is developing UCART19, an allogeneic chimeric antigen receptor (CAR) T cell product candidate, which is in Phase I clinical trials for the treatment of pediatric and adult patients with R/R CD19 positive B-cell ALL; ALLO-501, an anti-CD19 allogeneic CAR T cell product candidate that is in Phase I clinical trial for the treatment of R/R non-Hodgkin lymphoma; and ALLO-501A for the treatment R/R large B-cell lymphoma or transformed follicular lymphoma. It is also developing ALLO-715, an allogeneic CAR T cell product candidate that is in a Phase 1 clinical trial for treating R/R multiple myeloma; ALLO-819, an allogeneic CAR T cell product candidates for the treatment of acute myeloid leukemia; ALLO-647, an anti-CD52 monoclonal antibody; CD70 to treat renal cell cancer; and DLL3 for the treatment of small cell lung cancer and other aggressive neuroendocrine tumors. The company has license and collaboration agreements with Pfizer Inc.; Servier; Cellectis S.A.; and Notch Therapeutics Inc., as well as clinical trial collaboration agreement with SpringWorks Therapeutics, Inc. Allogene Therapeutics, Inc. also has a strategic collaboration agreement with The University of Texas MD Anderson Cancer Center for the preclinical and clinical investigation of AlloCAR T candidates across a portfolio of hematologic and solid tumors. The company was founded in 2017 and is headquartered in South San Francisco, California. |
Alzamend Neuro Atlanta, Georgia, United States of America | Alzamend Neuro, Inc., a preclinical stage biopharmaceutical company, focuses on developing products for the treatment of neurodegenerative diseases and psychiatric disorders. The company’s lead product candidate is AL001 for the treatment of Alzheimer’s and other neurodegenerative diseases and psychiatric disorders. It is also developing AL002, a cell-based therapeutic vaccine, which seeks to restore the ability of the patient’s immunological system to Alzheimer’s. The company was incorporated in 2016 and is headquartered in Tampa, Florida. |
American Gene Technologies Rockville, Maryland | American Gene Technologies® International Inc. (AGT™) is a private biotechnology company pursuing cures and treatments for infectious diseases, cancers, and monogenic disorders. AGT™'s emerging gene and cell therapeutics programs are designed to: ✅ Create immunotherapy for HIV disease with gene-modified T cells ✅ Activate innate T cell immunity capable of killing all tumor cells with its vector used in immuno-oncology ✅ Treat PKU by bringing a functional gene into cells that express a dysfunctional gene due to mutation ✅ Advance clinically to provide human proof of concept For over a decade, AGT™ has developed, tested, and banked thousands of lentiviral vectors that can be adapted to the specific needs of new target diseases. AGT™ has developed gene therapy for diseases using this rapid gene delivery platform. GENE THERAPIES AGT™’s platform allows it to pursue clinical “cures” for complex diseases. The company has developed patent-protected gene therapeutics that are breakthroughs in medicine. *** See our extensive award winning patents on our website *** HIV/AIDS Immunotherapy for HIV disease with gene-modified T cells IMMUNO-ONCOLOGY Activates innate T cell immunity capable of killing all tumor cells with its vector used in immuno-oncology PHENYLKETONURIA (PKU) Treats PKU by bringing a functional gene into cells that express a dysfunctional gene due to mutation AGT™'s Core Objectives ✔️ Leverage our lentivirus platform to create highly-effective targeted genetic medicines ✔️ Create solutions for patients across a spectrum of infectious diseases, cancers and inherited disorders ✔️ Collaborate with leading medical researchers from around the globe To learn more and get in touch with American Gene Technologies® Inc., contact us today at https://www.americangene.com/contact-us/ |
Arbor Biotechnologies Cambridge, Massachusetts, US | Arbor Bio is a next-generation gene editing company focused on discovering and developing potentially curative genomic medicines, with the most extensive toolbox of proprietary genomic editors in the industry. We have the unique ability to work backward from disease pathology and choose the optimal editors or combination of editors for the disease in question. |
Arsenal Biosciences South San Francisco , CA | ArsenalBiosciences, Inc., is a privately held, clinical stage programmable cell therapy company focused on the realization of solid tumor cell therapy to defeat cancer. Our discovery engine comprises precise CRISPR-enabled genome editing, integrated circuits incorporating logic gates for improved tumor targeting, and therapeutic enhancements enabling multiple pharmaceutical functions. With our programmable and computationally driven approach, we aim for enhanced and broader efficacy, increased patient safety, reduced provider costs and expanded market access. Our team is engineering living medicines to attack cancer’s inherent multi-faceted nature and bring dramatic improvements to the lives of patients. |
Artax Biopharma Cambridge, Massachusetts, United States | Artax Biopharma is a development-stage biopharmaceutical company dedicated to the development of new therapies for autoimmune and inflammatory diseases. Artax Biopharma is developing the next generation of drugs targeting the interaction between TCR and Nck, which is responsible for T-cell activation. Specific control over T-cells through TCR, provides the compounds with the potential to become first-in-class immunomodulators for the treatment of a wide range of autoimmune and inflammatory diseases. |
Artcline Rostock, Germany | ARTCLINE GmbH develops innovative therapy methods for intensive care medicine based on immune cells. Our current main project is the development of the so-called ARTICE technology for the treatment of bacterial sepsis. ARTCLINE's vision is the development of innovative immune cell-based therapy approaches and to make the technology available to physicians and hospitals worldwide. The safety and effectiveness of the therapy is our top priority. Our extensive cell biological, clinical and industrial experience in the fields of immunology, sepsis diagnostics and therapy help us to realize our vision. Together with scientists and partners, we focus our research activities on the root causes and correlations of sepsis. For many years, the founders and the team of ARTCLINE GmbH have been successfully involved in the transfer of medical inventions from university research to industrial application. We have an extensive network and long standing experience in the development and successful marketing of medical devices and drugs. |
Artiva Biotherapeutics San Diego, California, United States | At Artiva, our mission is to deliver to cancer patients highly effective cellular immunotherapies that are also safe and immediately accessible. Towards this end, Artiva is advancing a pipeline of off-the-shelf, allogeneic NK cell therapies, including CAR-NK cell therapies, that leverage the company’s unique manufacturing and engineering capabilities. Artiva is headquartered in San Diego. |
ASC Therapeutics Milpitas, California, United States | ASC Therapeutics focuses on the development of curative therapies for rare diseases. Our gene, CRISPR and cell therapies were developed at Applied StemCell, a life sciences company for over 12 years, including proprietary gene editing technologies. Our development pipeline includes several preclinical stage projects focusing on monogenic blood disorders that exhibit a high genetic penetrance and conditions requiring immunomodulatory interventions. Contact us for partnership opportunities |
Atara Biotherapeutics Thousand Oaks, California, United States of America | Atara Biotherapeutics Inc., is a leading allogeneic T-cell immunotherapy company pioneering the development of transformative off-the-shelf therapies for patients with serious diseases including solid tumors, hematologic cancers, and autoimmune diseases. With our lead program receiving marketing authorization in Europe, Atara is the most advanced allogeneic T-cell immunotherapy company and intends to rapidly deliver off-the-shelf treatments to patients with high unmet medical need. Our platform leverages the unique biology of EBV T cells and has the capability to treat a wide range of EBV-associated diseases, or other serious diseases through incorporation of engineered CARs (chimeric antigen receptors) or TCRs (T-cell receptors). Atara is applying this one platform, which does not require TCR or HLA gene editing, to create a robust pipeline including: tab-cel for Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD) and other EBV-driven diseases; ATA188, a T-cell immunotherapy targeting EBV antigens as a potential treatment for multiple sclerosis; and multiple next-generation chimeric antigen receptor T-cell (CAR-T) immunotherapies for both solid tumors and hematologic malignancies. |
Atsena Therapeutics Durham, NC | Atsena Therapeutics is a clinical-stage gene therapy company developing novel treatments for inherited forms of blindness. The company has two clinical-stage programs, ATSN-201 for X-linked retinoschisis (XLRS) and ATSN-101 for GUCY2D-associated Leber congenital amaurosis (LCA1). ATSN-201, which leverages the company’s novel spreading capsid AAV.SPR, is being evaluated in XLRS patients in a Phase I/II clinical trial known as the LIGHTHOUSE study. The company’s additional proprietary asset is ATSN-301, a dual AAV vector-based gene therapy to prevent blindness from MYO7A-associated Usher syndrome (USH1B). Interim safety and efficacy data from the company’s ongoing Phase I/II clinical trial in patients with LCA1 have demonstrated ATSN-101 is well tolerated and clinically meaningful improvements in vision were observed 12 months post-treatment. Founded by ocular gene therapy pioneers Dr. Shannon Boye and Sanford Boye of the University of Florida, Atsena is based in North Carolina’s Research Triangle, an environment rich in gene therapy expertise. |
Aurealis Therapeutics Microkatu 1, Kuopio, 70210, FI | Aurealis Therapeutics is a Swiss-Finnish Cell and Gene Therapy Platform Company. Our multi-target cell and gene therapy platform, based on genetically modified safe lactic acid bacteria, addresses unmet medical needs: Chronic Wounds - Diabetic Foot Ulcers, Venous Ulcers, Pressure Ulcers - deadly Cancers - Ovarian Cancer, Peritoneal Carcinomatosis - Inflammation. After successfully completing our Phase 1 clinical study in Diabetic Foot Ulcer (DFU) patients with our lead clinical product AUP-16, our DFU Phase 2 study is ongoing in Italy, Germany and Poland. Venous Ulcers and other ulcers will follow. In Oncology, we are building on impressive pre-clinical data with Oncolytic Bacteria lead candidate AUP-55, which shows increased survival in Ovarian Cancer and peritoneal carcinomatosis. Our pipeline also includes Inflammation, at discovery stage. |
Autolus Therapeutics London, United Kingdom | Focused on the development of precisely targeted, controlled and highly active T cell therapies that are designed to offer cancer patients substantial benefits over existing standard of care. |
AviadoBio London, United Kingdom | At AviadoBio, we are relentlessly chasing cures by translating groundbreaking science and precision delivery into life-changing medicines for people living with frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). With our deep understanding of the brain and our suite of proprietary gene therapy platforms and delivery technologies, we are working relentlessly to overcome the challenges of delivering the right drug to the right place. Our innovative, neuroanatomy-led approach is designed to maximize the therapeutic potential of gene therapy to halt or potentially reverse neurodegenerative diseases. The Company’s technology is based on pioneering research from King’s College London and the UK Dementia Research Institute. |
Aviceda Therapeutics Cambridge, Massachusetts, United States | The Future of Glyco-therapeutics Aviceda Therapeutics is a disruptive clinical-stage biotech company focused on the next generation of immuno-modulators by harnessing the power of glycobiology to address the innate immune system and chronic non-resolving inflammation specifically and profoundly using a propriety HALOS platform Technology. Aviceda is developing glyco-immuno therapeutics targeting devastating ocular and systemic degenerative, fibrotic, and immuno-inflammatory diseases. A combination of cutting-edge technologies and deep biological insights form Aviceda’s engine of transformative product creation. Aviceda has combined the power of our glycobiology and glycochemistry platform with our proprietary nanoparticle technology (HALOS) to engineer transformative disease modifying medicines. Using our cell-based high-throughput screening (HTS) platform, we can rapidly screen, select, and optimize ligands based on maximum affinity and specificity for each different type of innate immune cell. Optimized ligands are tethered to our modular biodegradable nanoparticles using high precision conjugation chemistry to form biologically stable linkages. Our GCT nanoparticles are designed for optimal therapeutic durability and maximum therapeutic efficacy while utilizing the most effective route of administration. Aviceda’s platform is highly adaptable, enabling the development of a broad spectrum of therapeutics for immuno-inflammatory indications with large unmet medical needs. |
Avirmax Biopharma Hayward, California, US | |
Beam Therapeutics Inc Cambridge, Massachusetts, United States of America | Beam Therapeutics Inc., a biotechnology company, engages in developing precision genetic medicines for patients suffering from serious diseases in the United States. The company is developing therapies for the development of sickle cell disease and beta-thalassemia; CAR-T cell therapies for pediatric T-cell acute lymphoblastic leukemia and pediatric acute myeloid leukemia; therapies for alpha-1 antitrypsin deficiency and glycogen storage disorder 1A; and therapies for ocular and central nervous system disorders. It has a research and clinical collaboration agreement with Magenta Therapeutics, Inc. Beam Therapeutics Inc. was founded in 2017 and is based in Cambridge, Massachusetts. |
Be Biopharma Cambridge, Massachusetts, United States | Be Bio is pioneering Engineered B Cell Medicines (BCMs) to dramatically improve the lives of patients who are living with Hemophilia B and other genetic diseases, cancer, and other serious conditions. With eyes locked on the patient, our team of purpose-driven scientists, technologists, manufacturing experts and business builders collaborate to create a bold new class of cell therapies. |
BioCytics Huntersville, North Carolina | BioCytics via the Human Applications Lab is focused on bringing personalized oncology treatments to the market. BioCytics has an ongoing IRB-approved clinical trial (BioCytics 0001; NCT00571389) that allows for the collection and study of blood and tissue samples from consenting cancer patients. We are incubated within Carolina BioOncology - a preferred cancer treatment and Phase I drug testing facility. BioCytics was founded by Dr. John Powderly, MD, medical board certified oncologist and a certified physician investigator (CPI), who is also president of Carolina BioOncology Institute (CBOI). |
BioGenCell Netanya, Israel | BioGenCell was founded with the vision of developing safe, effective, and long-lasting effective treatment for incurable, blood vessel diseases that will be both accessible and patient friendly. Our disruptive TRACT technology platform provides a comprehensive approach for rapid manufacturing and commercialization of cell-therapies. |
BlueSphere Bio Pittsburgh, Pennsylvania, United States | BlueSphere Bio was founded to unlock the potential of personalized T cell therapy for the treatment of cancer by harnessing the unique specificity of each patient’s own T cells for the particular molecular fingerprint of each patient’s own cancer through our novel TCXpress technology. Our proprietary TCXpress platform is an elegant, rapid approach that exploits the patient’s own immune defenses to attack their cancer specifically, and is designed to leave normal tissues untouched, thereby reducing harmful side effects. The platform and implementation allows us to deliver rapid, personalized T cell treatments for a variety of cancer indications. |
Brenus Pharma Lyon, France | Brenus Pharma is a pioneering clinical stage biotech aiming to prevent cancer recurrence, the main challenge for solid tumors - causing 10M deaths each year worldwide. We developped a next-generation cancer treatment, thanks to our “off-the-shelf” therapeutic platform. Our lead candidate is a proteomic-driven immunotherapy targetting colorectal cancer (CRC), the 2nd deadliest cancer worldwide. - Our allogeneic platform generates therapeutics that efficiently educate the immune system, with more potent, diverse and novel targets than any existing therapies. (Characterized by advanced multiomics technologies) - We overcome limitations of several technologies such as mRNA vaccines thanks to our enhanced immunogenicity and standardized GMP manufacturing: we keep control of costs and supply. Awarded “Best Biotech Startup” in 2024 in France, we recently closed a $25M SERIES A to accelerate our clinical development. We are currently envisaging clinical collaborations. Brenus Pharma sets a new paradigm in oncology, by giving back to patients the ability to fight against their disease. |
Cabaletta Bio Philadelphia, Pennsylvania, United States of America | Cabaletta Bio, Inc., a clinical-stage biotechnology company, focuses on the discovery and development of engineered T cell therapies for patients with B cell-mediated autoimmune diseases. Its proprietary technology utilizes chimeric autoantibody receptor (CAAR) T cells that are designed to selectively bind and eliminate B cells, which produce disease-causing autoantibodies or pathogenic B cells. The company’s lead product candidate is DSG3-CAART, which is in Phase I clinical trial for the treatment of mucosal pemphigus vulgaris, an autoimmune blistering skin disease, and Hemophilia A with Factor VIII alloantibodies. Its product candidate pipeline also include MuSK-CAART, a preclinical stage product to treat a subset of patients with myasthenia gravis; FVIII-CAART, a discovery stage product to treat a subset of patients with Hemophilia A; and DSG3/1-CAART, a discovery stage product for the treatment of mucocutaneous pemphigus vulgaris. Cabaletta Bio, Inc. has a collaboration with the University of Pennsylvania; and research agreement with The Regents of the University of California. The company was formerly known as Tycho Therapeutics, Inc. and changed its name to Cabaletta Bio, Inc. in August 2018. Cabaletta Bio, Inc. was incorporated in 2017 and is headquartered in Philadelphia, Pennsylvania. |
Capsida Biotherapeutics Newbury Park, California, United States of America | Capsida Biotherapeutics Inc. is an industry-leading gene therapy platform company creating a new class of targeted, non-invasive gene therapies for patients with debilitating and life-threatening genetic disorders. Capsida's technology allows for the targeted penetration of cells and organs, while limiting collateral impact on non-targeted cells and organs, especially the liver. This technology allows for the delivery of the gene therapy in a non-invasive way through intravenous administration. Capsida's technology is protected by a growing intellectual property portfolio which includes more than 30 patent applications and one issued U.S. patent 11,149,256. The company is exploring using the technology across a broad range of life-threatening genetic disorders. Its initial pipeline consists of multiple neurologic disease programs. The company has strategic collaborations with AbbVie and CRISPR, which provide independent validation of Capsida's technology and capabilities. Capsida is a multi-functional and fully integrated biotechnology company with proprietary adeno-associated virus (AAV) engineering, multi-modality cargo development and optimization, translational biology, process development and state-of-the-art manufacturing, and broad clinical development experience. Capsida's biologically driven, high-throughput AAV engineering and cargo optimization platform originated from groundbreaking research in the laboratory of Viviana Gradinaru, Ph.D., a neuroscience professor at the California Institute of Technology. |
Carina Biotech Mawson Lakes, South Australia | Carina Biotech An Australian clinical stage immunotherapy company established to research and develop chimeric antigen receptor T cell (CAR-T) therapies to treat solid cancers. Carina are working towards producing broad-spectrum CAR-T therapies that can be used to treat multiple solid cancers yet are patient-specific and result in little, if any, off-cancer damage. Using its proprietary platforms, Carina is also developing technologies to improve access to, and infiltration of, solid cancers, and to enhance CAR-T cell manufacturing. We are working to… 1. Expand the clinical indications for T cell therapies 2. Improve the commercial viability of T cell therapies by developing supporting technologies that make T cell therapies more effective or economically viable Headquartered in Adelaide, South Australia, Carina has strong T cell R&D capability across a network of research providers and collaborators including leading scientists at the UniSA, the Women's & Children's Hospital in Adelaide, the University of Adelaide, the Royal Prince Alfred Hospital in NSW and the Seattle Children's Hospital in the United States. |
CARsgen Therapeutics Shanghai, China | CARsgen is a biopharmaceutical company with operations in China and the U.S. focused on innovative CAR T-cell therapies for the treatment of hematologic malignancies and solid tumors. The company has built an integrated cell therapy platform with in-house capabilities that span target discovery, antibody development, clinical trials, and commercial-scale manufacturing. CARsgen has internally developed novel technologies and a product pipeline with global rights to address major challenges of CAR T-cell therapies, such as improving the safety profile, enhancing the efficacy in treating solid tumors, and reducing treatment costs. Our vision is to become a global biopharmaceutical leader that brings innovative and differentiated cell therapies to cancer patients worldwide and makes cancer curable. |
Cellectis Paris, France | Cellectis is a global clinical-stage biopharmaceutical company. Pioneers and innovators in our field, our mission is to develop innovative treatments for patients with unmet medical needs. With 25 years of expertise, we have the best-in-class gene editing platform focusing on immuno-oncology, and gene therapy. Through our efficient and precise TALEN® technology, we create allogeneic CAR-T cells capable of recognizing and combating cancer cells. Today, our three clinical programs target patients with B-cell acute lymphoblastic leukemia (B-ALL), non-Hodgkin lymphoma (NHL) and acute myeloid leukemia (AML). We are fully integrated and a leader in end-to-end gene editing, allogeneic CAR T-cell companies. With our in-house manufacturing, we control our gene and cell therapy process from start to finish with starting materials produced in Paris (France) and CAR-T therapy products created in Raleigh, NC (USA). We also have several ongoing strong collaborations, based on our TALEN® technology, with leading cell & gene therapy companies, including our recent partnership with AstraZeneca, to develop new product candidates in oncology, immunology, and treatment of rare diseases. At Cellectis, we are committed to a cure. Cellectis headquarters are in Paris, France, with additional locations in New York, New York and Raleigh, North Carolina. Cellectis is listed on the Nasdaq Global Market (ticker: CLLS) and on Euronext Growth (ticker: ALCLS). To find out more about us, visit our website: www.cellectis.com. To learn more about our community guidelines, visit: http://www.cellectis.com/en/social-media-guidelines/ Follow our other social media accounts: @cellectis on LinkedIn on X (formerly Twitter) . TALEN® is a registered trademark owned by the Cellectis Group. |
CellOrigin Biotech Hangzhou, Zhejiang | CellOrigin Biotech has a long term focus on iPSC-derived innate immune cells and its applications in new cancer immune cells. Dr. Jin Zhang, the scientific co-founder of CellOrigin used to be trained as a research fellow at the Boston Children’s Hospital and Harvard Medical School. His team worked closely with clinicians at the First Affiliated Hospital of Zhejiang University and for the first time reported the induced pluripotent stem cell or iPSC-derived CAR-macrophages (CAR-iMac), and its applications in cancer immunotherapies. As for now, CellOrigin Biotech holds its proprietary technologies for iPSC-derived CAR-Macrophage. With this platform, they are collaborating with research groups in genome engineering and synthetic biology at Harvard and MIT to fully unleash the potential of iPSC-derived immune cells, which are highly editable, expandable and clonal. Eventually, they would like to achieve a goal of bring more effective, universal and safe immune cell products to cancer patients, especially for those with solid tumors. The investigator initiated trials has been initiated at the First Hospital of Zhejiang University. The core proprietary technology platform and the core patents including the engineered macrophages from pluripotent stem cells has been authorized and is in the process of entering the US, Australia, South Africa, etc. |
Charles River Wilmington, Massachusetts, United States | At Charles River, we are passionate about our role in improving the quality of people’s lives. Our mission, our excellent science and our strong sense of purpose guides us in all that we do, and we approach each day with the knowledge that our work helps to improve the health and well-being of many across the globe. |
Chimeric Therapeutics Carlton, Victoria, Australia | Chimeric Therapeutics is a clinical stage cell therapy company focused on bringing the promise of cell therapy to life for more patients with cancer. We believe that cellular therapies have the promise to cure cancer not just delay disease progression. To bring that promise to life for more patients, Chimeric’s world class team of cell therapy pioneers and experts is focused on the discovery, development and commercialization of the most innovative and promising cell therapies.chm |
Citeline New York, US | Citeline powers a full suite of complementary business intelligence offerings to meet the evolving needs of health care professionals to accelerate the connection of treatments to patients and patients to treatments. These patient-focused solutions and services deliver and analyze data used to drive clinical, commercial and regulatory-related decisions and create real-world opportunities for growth. Our global teams of analysts, journalists and consultants keep their fingers on the pulse of the pharmaceutical, biomedical and medtech industries, covering it all with expert insights: key diseases, clinical trials, drug R&D and approvals, market forecasts and more. |
Complement Therapeutics Munich, Germany | Complement Therapeutics GmbH (CTx) is an early-stage biotechnology company focused on the research and development of novel therapeutics for complement-mediated diseases. The Company is a spinout from the University of Manchester and is based on the pioneering research of its founders into novel targets within the complement cascade. Our lead investigational product (CTx001) is being evaluated as a potential gene therapy for dry age-related macular degeneration/geographic atrophy, a leading cause of blindness. Additional programmes will evaluate potential therapeutic opportunities in other complement-mediated conditions. The Company is also developing a unique quantification methodology, the Complement Precision Medicine (CPM) platform, to enable quantification of over 30 complement cascade proteins enabling more precise diagnosis and monitoring of disease. |
Creative Medical Technology Holdings Inc. Phoenix, Arizona, United States | Creative Medical Technology Holdings, Inc., formerly Jolley Marketing, Inc., is a clinical-stage company. The Company conducts its business operations primarily through its subsidiary, Creative Medical Technologies, Inc. (CMT). The Company is engaged in stem cell research and applications for use to treat male and female sexual dysfunction, infertility, miscarriages and related issues. CMT focuses on completing the testing of its erectile dysfunction (ED) treatment and, if warranted, marketing treatment kits under the name Caverstem to physicians for use with their patients suffering from ED. CMT also intends to test and, if warranted, market licensed stem cell products under its infertility technology license. Procedures for use of the Company's ED stem cell treatment consist of a one-hour out-patient visit in a physician's office. The physician would harvest a patient's bone marrow from the hip using local anesthetic and separate the stem cells using a cell separator. |
Curocell Daejeon, South Korea | Curocell is a cell therapy company that discovers and develops CAR-T therapies for cancer. The company's current efforts focus on unleashing the body’s immune system through broad-spectrum CAR-T technologies to treat blood cancers, as well as solid cancers. The company's programmed T cell product candidates are differentiated by incorporation of T cell modifying technology designed to effectively overcoming the immune suppression, and thereby offers greater efficacy compared to the current CAR-T cell technology. |
Cynata Therapeutics Carlton, Victoria, Australia | Cynata Therapeutics Limited (ASX: CYP) is an Australian clinical-stage stem cell and regenerative medicine company focused on the development of therapies based on Cymerus™, a proprietary therapeutic stem cell platform technology. Cymerus™ overcomes the challenges of other production methods by using induced pluripotent stem cells (iPSCs) and a precursor cell known as mesenchymoangioblast (MCA) to achieve economic manufacture of cell therapy products, including mesenchymal stem cells (MSCs), at commercial scale without the limitation of multiple donors. Cynata’s lead product candidate CYP-001 met all clinical endpoints and demonstrated positive safety and efficacy data for the treatment of steroid-resistant acute graft-versus-host disease (GvHD) in a Phase 1 trial. Clinical trials of Cymerus MSC products in osteoarthritis (Phase 3) and in severe complications arising from COVID-19 (Phase 2) are currently ongoing. Planning is also underway for further clinical trials of Cymerus MSC products in GvHD (through licensee Fujifilm), critical limb ischemia, idiopathic pulmonary fibrosis, renal transplantation, and diabetic foot ulcers. In addition, Cynata has demonstrated utility of its Cymerus MSC technology in preclinical models of numerous diseases, including the clinical targets mentioned above, as well as asthma, heart attack, sepsis, acute respiratory distress syndrome (ARDS) and cytokine release syndrome. |
Diakonos Oncology Houston | Diakonos is a clinical-stage biotechnology company developing a groundbreaking Dendritic Cell Vaccine (DCV) for difficult-to-treat cancers. |
Dompé Milano, Lombardia, Italy | Dompé farmaceutici is an international biopharmaceutical company involved in all activities of the pharmaceutical value chain, from research to development, production and marketing. As a long-established family company, we blend the stability that comes from our 130-year heritage with the agility and dynamism of a start-up. We can turn transformative breakthroughs into treatments because we have the right qualities and capabilities to deliver new drugs. Our 160,000 square meter R&D and production site in L’Aquila, Italy, is the heart of Dompé. Here, we conduct vital research, and produce and distribute our primary care and biotech products. Primary Care includes a broad spectrum of over-the-counter treatment solutions, prescription medications, supplements and medical devices. Our Biotech focuses on the development of treatments for rare diseases. The main therapeutic areas include pain relief, inflammation, ophthalmology, diabetes care, nutrition and oncology. In the pursuit of improving human health, our R&D strives to break through in unconventional places. This is what led us to Nerve Growth Factor (NGF), a Nobel Prize-winning discovery made in 1986. We leveraged 50 years of research to transform this discovery into a breakthrough treatment. Our human recombinant NGF, cenegermin, is the first treatment for a rare degenerative eye disease, neurotrophic keratitis. That deep appreciation of science keeps us committed to investigating neurotrophins. Our relentless pursuit in identifying new treatments for diseases allowed us to harness the potential of bioinformatics to accelerate the drug discovery process. The result is Exscalate, a structure-based virtual screening platform developed in-house and currently one of the most powerful supercomputing and artificial intelligence platforms for drug testing. We are focused on the road ahead. Our hunger to deliver what patients need from science keeps us focused on what matters. We embrace the challenge in science. |
Dragonfly Therapeutics Waltham, Massachusetts, United States | Dragonfly Therapeutics is a clinical-stage biopharmaceutical company committed to discovering, developing and commercializing therapies that use its novel multispecific antibody technology to harness the body's immune system to bring breakthrough treatments to patients. In addition to its wholly owned clinical assets, Dragonfly has a deep pipeline of wholly owned preclinical candidates discovered using its proprietary platform, as well as productive collaborations with Merck, AbbVie, Gilead and Bristol Myers Squibb in a broad range of disease areas. For more information, email info@dragonflytx.com. |
Editas Medicine Cambridge, Massachusetts, United States of America | At Editas Medicine, we are pioneering the possible. Our mission and commitment is to harness the power and potential of CRISPR gene editing to develop a robust pipeline of medicines for people living with serious diseases around the world. Our goal is to discover, develop, manufacture, and commercialize transformative, durable genomic medicines for many diseases. |
Encoded Therapeutics South San Francisco, California, United States of America | Encoded Therapeutics is creating one-time, disease-modifying gene therapies for pediatric central nervous system (CNS) disorders with its cell-selective targeting and regulation platform. The Encoded approach offers potentially unprecedented gene specificity and cell selectivity to unlock novel opportunities by targeting a range of disease mechanisms. Encoded’s technology is compatible with any delivery system to control where and when therapeutic transgenes are expressed, thereby shaping the functionality of target cells and holding broader therapeutic potential beyond CNS disorders. |
Enlivex Therapeutics Ness Ziona, Israel | Our mission is to establish new equilibriums – both in patients’ immune responses and in the field of immunotherapy. As a clinical-stage company focused on specialized cell immunotherapy, we intend to develop and commercialize an allogeneic drug pipeline for rebalancing all manner of immune hyper-responses. Our pioneering Allocetra™ cell-based therapy breaks with old paradigms, offering to effectively treat numerous acute conditions through a radically different clinical approach. By using the body’s native mechanisms to restore – rather than suppress – immune balance, we aim to reshape the way immune, autoimmune, and inflammatory conditions are both thought of and treated. |
Ensoma Boston, Massachusetts, US | |
Epicrispr Biotechnologies Inc. South San Francisco, California, US | Epic Bio is a biotechnology company that focuses on biomedical research and genetics. |
Estrella Biopharma Emeryville, California, United States | Developing transformative cancer medicines with CD19-Redirected ARTEMIS T Cells to improve patients’ lives |
Eureka Therapeutics Emeryville, California, United States | Eureka Therapeutics is a privately held biotechnology company located in the San Francisco Bay Area, focused on breakthrough immunotherapies for the treatment of cancer. Utilizing our antibody drug discovery and engineering technologies, we are advancing safe and effective therapies for targeting previously inaccessible cancer antigens. We have built an early stage pipeline of innovative drugs, with the most advanced candidates at pre-clinical stage. Eureka Therapeutics is a leader in the discovery of novel antibody immunotherapies. We utilize a paradigm-changing technology platform to develop fully-human antibodies against intracellular cancer antigens. In collaboration with Memorial Sloan-Kettering Cancer Center, we were the first-to-demonstrate in vivo efficacy of a human IgG1 antibody against an intracellular cancer antigen. Our most advanced program is at preclinical stage and being developed in partnership with Novartis. |
Evotec Hamburg, DE | Evotec is a biotechnology company that is committed to advancing drug discovery and development. Through flexible business models, we collaborate with pharmaceutical companies, biotechs, foundations, and government agencies on a substantial scale. We believe that by working together, we can make a difference in the lives of patients. Our Pipeline Co-Creation model represents our adaptable, multi-modality, fully integrated end-to-end approach to drive collaborations and services across all phases of drug discovery and development – from discovery of novel targets to achieving Proof of Concept in the clinic and into commercial manufacturing. We aspire to contribute data-driven disease understanding and early disease relevance in humans to bring the probability of success up. We select the right modality, which is then propelled forward on our platforms by our passionate people striving for the fastest and most effective ways towards patient impact. This is enabled by convergence of human ingenuity with data and AI. We also offer specific solutions, products, and CRO/CDMO-like services, always in support of the Biopharma R&D innovator. Our more than 4,000 scientists work closely with numerous partners concurrently, delivering fully integrated research and development portfolios or individual projects with the highest quality standards and efficiency, coupled with great science, passion, engagement, and communication. |
ExCellThera Montreal, Quebec, Canada | ExCellThera is an advanced clinical-stage company specializing in cell expansion and engineering, providing molecules and bioengineering solutions to expand stem cells. |
Forge Biologics Columbus, Ohio, United States | Forge is a gene therapy development engine, focused on enabling access to life changing gene therapies and helping bring them from idea into reality. We partner with innovators in the gene therapy community: scientists, physicians, biotech/pharma companies, and patient groups. We bring expertise in gene therapy manufacturing and therapeutics development to the table, helping champion teams that are navigating the long road from the lab bench to the bedside. With a patients-first approach, we are forging new models for working together to better accelerate these transformative medicines to reach those who need them most. |
Genenta Science Milan, Italy | Genenta (Nasdaq: GNTA) is a clinical stage immuno-oncology company developing a cell-based platform harnessing the power of hematopoietic stem cells to provide durable and safe treatments for solid tumors. Our platform is not tumor type nor target antigen restricted and provides sustained targeted expression of therapeutic payload(s) inside the tumor micro environment. Genenta’s lead product candidate, Temferon™, precisely targets the delivery of interferon-alpha to the tumor micro-environment, minimizing systemic toxicity while breaking tumor-induced immune tolerance. Our treatments are designed as one-time monotherapies, but with the additional potential, when used in combination, to significantly enhance the efficacy of other approved therapeutics. |
Genprex Austin, Texas, United States of America | Genprex, Inc. is a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes. Genprex’s technologies are designed to administer disease-fighting genes to provide new therapies for large patient populations with cancer and diabetes who currently have limited treatment options. Genprex works with world-class institutions and collaborators to develop drug candidates to further its pipeline of gene therapies in order to provide novel treatment approaches. Genprex’s oncology program utilizes its proprietary, non-viral ONCOPREX® Nanoparticle Delivery System, which the Company believes is the first systemic gene therapy delivery platform used for cancer in humans. ONCOPREX encapsulates the gene-expressing plasmids using lipid nanoparticles. The resultant product is administered intravenously, where it is then taken up by tumor cells that express tumor suppressor proteins that are deficient in the body. The Company’s lead product candidate, REQORSA® (quaratusugene ozeplasmid), is being evaluated in three clinical trials as a treatment for non-small cell lung cancer (NSCLC) and small cell lung cancer (SCLC). Both NSCLC clinical programs received a Fast Track Designation from the Food and Drug Administration. Genprex’s diabetes gene therapy approach is comprised of a novel infusion process that uses an endoscope and an adeno-associated virus (AAV) vector to deliver Pdx1 and MafA genes directly to the pancreas. In models of Type 1 diabetes, GPX-002 transforms alpha cells in the pancreas into functional beta-like cells, which can produce insulin but are distinct enough from beta cells to evade the body’s immune system. In a similar approach, GPX-003 for Type 2 diabetes, where autoimmunity is not at play, is believed to rejuvenate and replenish exhausted beta cells. |
GenSight Biologics Paris, France | GenSight Biologics is a clinical-stage biotechnology company discovering and developing novel therapies for neurodegenerative retinal diseases and diseases of the central nervous system. GenSight Biologics’ pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics for retinitis pigmentosa, to help preserve or restore vision in patients suffering from severe degenerative retinal diseases. GenSight Biologics’ lead product candidate, GS010, is in Phase III trials in Leber’s Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible low vision and legal blindness in teens and young adults. Using its gene therapy-based approach, GenSight Biologics’ product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery. |
Glycostem Oss, The Netherlands | Glycostem Therapeutics is a Dutch biotech company established in 2007. Glycostem Therapeutics has developed the world's first GMP compliant NK cell platform that is ready for industrial scale-up. |
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Gradalis Carrollton, Texas, United States | Gradalis is a late-stage clinical biotechnology company developing immunotherapies for multiple cancer indications including an upcoming Phase 3 registration trial in patients with ovarian cancer. The company’s proprietary bi-shRNA therapy platform, which has been proven in its clinical studies, can be utilized to silence a broad range of genes and is applicable to multiple cancer types. About Vigil Vigil® is the first cellular therapy to show positive survival benefits in a randomized controlled clinical study of patients with solid tumors. Vigil is a self-navigating cellular immunotherapy that addresses the major deficits of leading cancer treatments. Vigil uses all of a patient’s tumor-related antigens to identify cancer cells and then enhances the training of the immune system to recognize and attack those cells. Gradalis’s proprietary bi-shRNA platform accomplishes this via three key functions: (1) it blocks furin, the enzyme that generates the immunosuppressive cytokines TGF beta types 1 and 2; (2) it activates effector cells including antigen-presenting dendritic cells and CD8+ T cells via the production of GM-CSF; and (3) it induces an immune response to the full complement of neo-antigens from the patient’s tumor via intradermal administration. In a Phase 2b trial, Vigil showed a positive trend in the primary endpoint of recurrence-free survival in the overall population and a significant improvement in recurrence-free survival and overall survival in a pre-planned subgroup analysis of Stage III/IV newly diagnosed ovarian cancer patients with the BRCA wild type (BRCA-wt) molecular profile. The company is preparing to initiate a clinical study intended for product registration in patients in this subgroup. Additionally, Phase 1 results in an all-comer trial have shown positive signals of activity in multiple tumor types. |
GRI Bio Inc. San Diego, California, United States | GRI Bio, Inc. (NASDAQ: GRI) is a clinical-stage biopharmaceutical company focused on fundamentally changing the way inflammatory, fibrotic and autoimmune diseases are treated. GRI’s therapies are designed to target the activity of Natural Killer T (NKT) cells, which are key regulators earlier in the inflammatory cascade, to interrupt disease progression and restore the immune system to homeostasis. NKT cells are innate-like T cells that share properties of both NK and T cells and are a functional link between the innate and adaptive immune responses. Type 1 invariant NKT (iNKT) cells play a critical role in propagating the injury, inflammatory response, and fibrosis observed in inflammatory and fibrotic indications. GRI’s lead program, GRI-0621, is an inhibitor of iNKT I cell activity and is being developed as a novel oral therapeutic for the treatment of idiopathic pulmonary fibrosis (IPF), a serious disease with significant unmet need. GRI is also developing a pipeline of novel type 2 natural killer T (NKT) cell agonists for the treatment of systemic lupus erythematosus and primary sclerosing cholangitis. Additionally, with a library of over 500 proprietary compounds, GRI has the ability to fuel a growing pipeline. |
Grit Biotechnology Shanghai, China | Grit Bio is a Chinese cell therapy company founded in 2019. The company is developing tumor-infiltrating lymphocyte (TIL) therapy program to address the unmet medical needs and to improve the quality of life for patients with solid tumor. GT101 - Grit’s non-gene-modified TIL product manufactured using proprietary process - is the first TIL product received Investigational New Drug (IND) approval in China and is currently in Phase 1 clinical trial. Grit Bio has established three core technology platforms for its TIL development: StaViral® - a retroviral system tailored for TIL engineering, ImmuT Finder® - a high-throughput screening platforms identifying novel T-cell targets, and TIL expansion platform StemTexp® that preferentially enriches TCM and TSCM populations of TIL. The company is currently developing potentially best-in-class gene-engineered TIL therapy based on these platforms. |
Gyroscope Therapeutics London, United Kingdom | Discovering and developing innovative medicines to preserve people’s sight and fight the devastating impact of blindness around the world. |
Healios Tokyo, Japan | 弊社は、バイオ革命によって急速に発展し始めている再生医療分野において、体性幹細胞再生医薬品/iPSC再生医薬品の研究開発を行っているバイオベンチャーです。2015年6月には東証マザーズに上場。再生・細胞医薬品の開発・製造を世界で先駆けて取り組み、病で苦しむ患者さんに治療法を提供し希望を届けることを目標としています。 Our company is a bio venture which is doing R & D of somatic stem cell regenerating medicine / iPSC regenerating medicine in the regenerative medicine field which is rapidly developing by the bio-revolution. We were listed on TSE Mothers in June 2015. Our long-term object is to deliver hope to patients suffering from illness by providing treatment methods by pioneering the development and manufacture of regeneration and cellular medicines worldwide. |
Hexaell Biotech (Shanghai Weizhizhuo Biotechnology) Shanghai, China | Shanghai Weizhizhuo Biotechnology Co., Ltd. was established in 2015. It has production lines and R&D centers in the core area of Shanghai Jiading Industrial Park and Shanghai Zhangjiang High-tech Park. The company has internationally leading cell differentiation and production technology and is a new biomedical technology company focusing on severe liver diseases and cell therapy. The company's founders come from world-class academic research institutes and top pharmaceutical companies, and are committed to developing the world's most advanced innovative biomedical technologies and products to provide patients with better clinical solutions. China is a country with a high incidence of liver disease, with 500,000 to 1 million new cases of liver failure each year, and the 28-day mortality rate of patients with acute liver failure is as high as 50%. The most effective treatment option at present is liver transplantation, but the high cost of treatment and the shortage of liver sources mean that only about 5,000 patients can undergo liver transplantation each year. In addition, the use of physical adsorption and plasma exchange cannot effectively remove toxins from the blood and promote the recovery of liver function. The team led by Hui Lijian, a researcher at the Institute of Biochemistry and Cell Biology, Shanghai Institutes for Biological Sciences, Chinese Academy of Sciences, has collaborated with many institutions to break through the in vitro culture technology of "hepatocyte-like cells" and construct hiHep cells with the world-leading direct transdifferentiation technology. This technology also won the honor of "Top Ten Advances in Chinese Science" in 2011. With this as the core, Shanghai Weizhizhuo Biotechnology Co., Ltd., which has obtained the exclusive global license for this technology, has built a new HepaCure bioartificial liver support system, which provides a new and effective treatment for patients with liver failure caused by severe liver disease. The HepaCure bioartificial liver is an in vitro liver function support system that can replace liver function in a short period of time, promote the recovery of autologous liver function in patients with liver failure, and buy time for patients planning liver transplantation to wait for a suitable liver source. It is like a small liver dialysis machine. During the dialysis process, it not only detoxifies the blood, but also supplies nutrients to the liver. |
Hrain Biotechnology Pudong, China | Hrain is supported by authoritative scientific advisory committee, cooperating with international top universities and domestic research institutes, including University of Southern California, Duke University, University of California, Los Angeles, Cell and Gene Therapy Center of Academy of Military Medical Sciences, Cancer Diagnosis and Treatment Institute of Xinqiao Hospital, etc. |
iECURE Philadelphia, Pennsylvania, United States | iECURE is a clinical-stage biotechnology company focused on delivering cures and transforming the lives of patients with devastating liver disorders, utilizing new advancements in genetic medicine. We are advancing our pipeline in close partnership with the world-class translational engine at the University of Pennsylvania’s Gene Therapy Program. Using in vivo editing, our methods focus on inserting functioning genes into patients’ genomes, which offers long-term, stable expression of those genes. With our team’s proven track record, as well as the University of Pennsylvania’s deep expertise and translational genetic medicine engine, reversing the course of these devastating diseases is now within reach. |
Imvax Philadelphia, Pennsylvania, United States | Imvax is a clinical-stage biotechnology company developing personalized, whole tumor-derived immunotherapies. Imvax’s combination therapy uniquely captures the complete antigenic signature of a patient’s own tumor and converts it into a highly stimulatory ‘training program’ for the patient’s immune system. The comprehensive nature of this training is intended to result in both innate and adaptive immune stimulation to optimize long-term anti-tumor effects. The company’s lead program is in glioblastoma multiforme (GBM), an extremely aggressive form of brain cancer, with additional programs in other types of solid tumors. |
IN8bio New York, United States | IN8bio, Inc. is a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of gamma-delta T cell therapies for the treatment of cancer. Gamma-delta T cells are naturally occurring immune cells that embody properties of both the innate and adaptive immune systems, and can intrinsically differentiate between healthy and diseased tissue. IN8bio develops ex vivo-expanded and activated gamma-delta T cells based upon its deep expertise in gamma-delta T cell biology, proprietary genetic engineering, and cell-type specific manufacturing capabilities, which we refer to collectively as our DeltEx platform. IN8bio’s platform employs allogeneic, autologous, and genetically modified approaches to develop novel cell therapies, which are designed to effectively identify and eradicate tumor cells. IN8bio is currently the most clinically advanced gamma-delta T cell company and the first company to bring genetically modified gamma-delta T cells into the clinic. For more information about the Company and its programs, visit www.IN8bio.com. |
InGeneron Houston, Texas, United States | InGeneron is a clinical-stage biotechnology company developing novel, safe, and evidence-based cell therapies. We are setting new therapeutic standards by enabling minimally invasive treatments that unlock the healing potential of each patient’s own regenerative cells processed at the point of care for same-day treatment. We currently focus on helping patients impacted by orthopedic conditions and are conducting several clinical trials to validate our technology as a disease-modifying treatment. Based on more than 20 years of research, InGeneron is dedicated to developing therapies supported by clinical evidence and approved by the FDA. |
Intima Bioscience Cambridge, United Kingdom | Intima Bioscience - A clinical stage gene and cell therapy company focused on curative intent in solid tumor cancer |
Istari Oncology Morrisville, North Carolina | Istari Oncology, Inc. is a clinical-stage biotechnology company focused on novel immuno-oncology and immunotherapy platforms for the treatment of glioblastoma and a wide variety of tumors. The company was founded by Darell Bigner, MD, and Matthias Gromeier, MD, of Duke University Medical Center in 2016. Both are leaders in their respective research fields of virology, immunology, monoclonal development and clinical medicine, particularly in the treatment of brain tumors. |
Jaguar Gene Therapy Lake Forest, illinois, United States of America | Jaguar Gene Therapy, LLC is dedicated to accelerating breakthroughs in gene therapy for patients suffering from severe genetic diseases including those that impact sizeable patient populations. The company is made up of a proven team of experts with unparalleled CMC (Chemistry, Manufacturing and Controls), regulatory, clinical and commercial acumen who have first-hand experience in bringing novel gene therapy treatments to patients and their families. Committed to patient safety and product purity, Jaguar is rapidly advancing an initial pipeline of three programs targeting: 1) Type 1 galactosemia; 2) a genetic cause of autism spectrum disorder and Phelan-McDermid syndrome and other severe neurodevelopmental disorders with a SHANK3 mutation or deletion; and 3) Type 1 diabetes. The company continues to evaluate opportunities to expand its pipeline using the strength of the team and close relationships with numerous academic institutions. |
Jasper Therapeutics Jasper Therapeutics, 2600 Bridge Pkwy, Redwood City, CA 94065, United States | Jasper Therapeutics is a biotechnology company focused on targeting mast cells and stem cells to treat chronic diseases. Their mission is to make a significant difference in the lives of patients suffering from these conditions. |
jCyte Newport Beach, California, United States | jCyte is a clinical-stage company focused on the application of progenitor cell-based technology in retinal diseases. The Company's allogeneic product candidate, jCell, is delivered by simple intravitreal injection without the need for surgery or immune suppression. jCyte has completed a phase 1/2a, phase 1/2a extension and phase 2b clinical trials testing jCell in patients with Retinitis Pigmentosa. |
Kriya Therapeutics Redwood City, California, United States of America | Kriya Therapeutics, Inc. is a biopharmaceutical company developing gene therapies for diseases affecting millions of people around the world. With operations in Palo Alto, California and Research Triangle Park, North Carolina, Kriya has raised over $450 million to advance a broad pipeline of gene therapies for ophthalmology, neurology, and metabolic diseases. |
KSQ Therapeutics Lexington, Massachusetts, United States | KSQ Therapeutics has built an industry-leading genome-scale precision functional genomics platform, called CRISPRomics, to pinpoint therapeutic nodes that directly correlate genomic function to disease with unprecedented certainty and speed. The company is deploying CRISPRomics for novel drug development across broad therapeutic areas and is currently advancing a proprietary pipeline of tumor- and immune-focused drug candidates. |
Kyverna Therapeutics Emeryville, California, United States | Kyverna Therapeutics is a clinical-stage cell therapy company with the mission of engineering a new class of therapies for autoimmune and inflammatory diseases. The Kyverna therapeutic platform combines advanced T cell engineering and synthetic biology technologies to suppress and eliminate the autoreactive immune cells at the origin of autoimmune and inflammatory diseases. Kyverna’s pipeline includes KYV-101, a fully human, autologous chimeric antigen receptor (CAR) T-cell therapy with properties well suited for use in B cell-driven autoimmune diseases and KYV-201, a fully human, allogeneic CAR T-cell therapy. By offering more than one mechanism for taming autoimmunity, and with patients currently in treatment for multiple indications in the U.S. and Europe, Kyverna is positioned to act on its mission of transforming how autoimmune diseases are treated. |
Leucid Bio London, United Kingdom | Leucid is progressing novel CAR T therapies that produce a better and more durable response than previous CAR T generations, improving treatment outcomes and saving the lives of patients with refractory cancers. |
Lexeo Therapeutics Lexeo Therapeutics, 430 East 29th Street, 14th Floor, New York, NY 10016, United States | Based in New York City, Lexeo Therapeutics is a clinical-stage genetic medicines company dedicated to transforming healthcare by applying pioneering science to fundamentally change how disease is treated. Building on groundbreaking research from Weill Cornell Medicine and the University of California San Diego, Lexeo partners with preeminent institutions on the cutting edge of gene therapy research. Using a stepwise development approach, Lexeo is leveraging early proof-of-concept functional and biomarker data to advance a pipeline of cardiovascular and APOE4 associated Alzheimer's disease programs, and is led by pioneers and experts with decades of collective experience in genetic medicines, rare disease drug development, manufacturing and commercialization. For more information, please visit www.lexeotx.com. |
Lion TCR Singapore | Lion TCR is a clinical-stage biotechnology company dedicated to the continued development and commercialization of our TCR-T therapy. |
Lisata Therapeutics Basking Ridge, New Jersey, United States | (Formally Caladrius Biosciences; NASDAQ: CLBS) Lisata Inc., (LSTA) a clinical-stage biopharmaceutical company, focuses on developing and commercializing cellular therapies to reverse disease and/or promote the regeneration of damaged tissue. Its product candidates include HONEDRA, a recipient of SAKIGAKE designation that is in Phase II clinical trial for the treatment of critical limb ischemia; OLOGO, a regenerative medicine advanced therapy for treating no-option refractory disabling angina; CLBS16 that is in Phase IIb clinical trial for the treatment of coronary microvascular dysfunction; and CLBS201, a CD34+ cell therapy for the treatment of pre-dialysis patients with chronic kidney disease. |
Lokon Pharma Uppsala, Sweden | LOKON PHARMA is a Swedish biotech company in the forefront of developing immunostimulatory gene therapy for cancer-based on oncolytic viruses. Innovative gene engineering, solid expertise in cancer biology and immunology, as well as a genuine curiosity in life science expand Lokon’s pipeline towards groundbreaking new therapeutics for patients with high unmet medical need. |
Longeveron Inc Miami, Florida, United States | We are a clinical-stage biotechnology company developing cellular therapies for aging-related and life-threatening conditions. Our lead investigational product is Lomecel-B™, which is derived from culture-expanded medicinal signaling cells (MSCs) that are sourced from bone marrow of young healthy adult donors. We believe that by using the same cells that promote tissue repair, organ maintenance, and immune system function, we can develop safe and effective therapies for some of the most difficult diseases and conditions associated with aging. We are currently conducting Phase 1 and 2 clinical trials in the following indications: Aging Frailty, Alzheimer’s disease, the Metabolic Syndrome, Acute Respiratory Distress Syndrome (ARDS), and hypoplastic left heart syndrome (HLHS), which is the current exception to our focus on aging. Our mission is to advance Lomecel-B™ and other cell-based product candidates into pivotal Phase 3 trials, with the goal of achieving regulatory approvals, subsequent commercialization and broad use by the healthcare community. Our philosophy is that healthy aging can be improved through regenerative medicine approaches. Life expectancy has substantially increased over the past century as a result of medical and public health advancements. However, this increase in longevity has not been paralleled by the number of years a person is expected to live in relatively good health, free of chronic disease and disabilities of aging – a period known as healthspan. As we age, we experience a profound decline, in both number and function, in our own MSCs, a decrease in immune system function, decline in blood vessel functioning, chronic inflammation and other issues. Our clinical data suggest that Lomecel-B™ addresses these conditions through multiple mechanisms of action, or MOAs, that simultaneously target key aging-related processes. |
Lyell Immunopharma South San Francisco, California, United States | Lyell Immunopharma, Inc., a T cell reprogramming company, engages in developing T cell therapies for patients with solid tumors. The company develops therapies using technology platforms, such as Gen-R, an ex vivo genetic reprogramming technology to overcome T cell exhaustion; and Epi-R, an ex vivo epigenetic reprogramming technology to generate population of T cells with durable stemness. Its pipeline includes LYL797, a T cell product candidate for the treatment of non-small cell lung cancer and triple negative breast cancers; LYL845 for multiple solid tumors; and NY-ESO-1 for synovial sarcoma and other solid tumor indications. Lyell Immunopharma, Inc. was incorporated in 2018 and is headquartered in South San Francisco, California. |
LyGenesis Pittsburgh, Pennsylvania, United States | LyGenesis is a biotechnology company with an organ regeneration technology platform enabling a patient’s lymph nodes to be used as bioreactors to regrow functioning ectopic organs. LyGenesis’ lead preclinical program is focused on liver regeneration for patients with end stage liver disease. Other therapeutic targets for organ regeneration include the thymus, pancreas, and kidney. Privately held, LyGenesis is headquartered in Pittsburgh, Pennsylvania. |
Marengo Therapeutics Cambridge, Massachusetts, United States | Marengo Therapeutics, a clinical stage biotech company, is pioneering an entirely new way to activate the body’s own immune system to mount a rapid, effective, and durable response against cancer. Our unique therapeutic platform selectively boosts T cells, turbocharging potent anti-tumor activity and promoting long-term immunity to keep cancer at bay. |
MEDIPOST Seongnam-si, South Korea | MEDIPOST is founded in 2000, and today MEDIPOST is leading the global stem cell therapeutics field with the world’s first regulatory-approved allogeneic human Umbilical Cord Blood-derived Mesenchymal Stem Cell(hUCB-MSC) product named CARTISTEM® for patients with knee Osteoarthritis(OA), launched in Korean market in 2012. MEDIPOST operates the largest private cord blood bank “CELLTREE®” in Korea with over 255,000 units of private cord blood units under storage. Each year, over 20,000 private cord blood units are collected and stored at CELLTREE®. MEDIPOST’s research and development is focused on novel off-the-shelf, allogeneic cell therapeutics using human Umbilical Cord Blood-derived Mesenchymal Stromal Cells (hUCB-MSCs) with clinical-stage assets in the disease areas of osteoarthritis (OA), broncho-pulmonary dysplasia (BPD) and Alzheimer’s disease (AD). MEDIPOST’s flagship product, CARTISTEM® (allogeneic Umbilical Cord Blood-derived MSCs + hyaluronic acid hydrogel composite) for knee osteoarthritis was approved in 2012 by Korea’s regulatory agency Ministry of Food and Drug Safety (MFDS) with Biologics License Application (BLA) label “Treatment of knee articular cartilage defects in patients with osteoarthritis (ICRS grade IV) as a result of degenerative disease or repeated trauma (without age limit)” PNEUMOSTEM® (allogeneic Umbilical Cord Blood-derived MSCs) for the prevention of Bronchopulmonary Dysplasia (BPD) in premature infants, completed the first-in-human Phase I safety trial in Korea and the randomized, placebo-controlled Phase II clinical trial is ongoing in Korea. Phase I/II clinical trial in the U.S. has been completed confirming safety with positive efficacy signals. MEDIPOST’s 2nd generation human Umbilical Cord Blood-derived Mesenchymal Stromal Cell (hUCB-MSC) pipeline code named SMUP-IA-01 is an off-the-shelf intra-articular injectable cell product for the patients with early to mid-stage knee osteoarthritis (OA). |
MeiraGTx New York, United States | MeiraGTx is a clinical-stage gene therapy company focused on developing potentially curative treatments for patients living with serious diseases. |
Mendus Stockholm, Sweden | Mendus is dedicated to changing the course of cancer treatment by addressing tumor recurrence and improving survival outcomes for cancer patients, while preserving quality of life. We are leveraging our unparalleled expertise in allogeneic dendritic cell biology to develop an advanced clinical pipeline of novel, off-the-shelf, cell-based immunotherapies which combine clinical efficacy with a benign safety profile. Based in Sweden and The Netherlands, Mendus is publicly traded on the Nasdaq Stockholm under the ticker IMMU.ST. |
MimiVax Buffalo, New York, United States | Our vision, to disrupt cancer with the development of highly innovative immunotherapies aimed at increasing patient survival rates MimiVax is a privately held, clinical-stage biotechnology company focused on the development and commercialization of immunotherapeutic vaccines and targeted therapies for the treatment of cancer. SurVaxM, our lead immunotherapeutic vaccine, has completed a Phase 2a clinical trial in adults with newly diagnosed glioblastoma. |
Minovia Therapeutics Haifa, Israel | Minovia Therapeutics is a clinical-stage international biotechnology company, established in hopes to bring life changing therapies to patients with Mitochondrial diseases, as fast as responsible. Following compassionate use cases performed in Sheba Medical hospital in Israel, using our proprietary Mitochondrial Augmentation Therapy, Minovia is now conducting the first of its kind clinical trial for Pearson syndrome, an ultra-rare pediatric disease. |
Mustang Bio Inc Worcester, Massachusetts, United States | Mustang Bio, Inc., a clinical-stage biopharmaceutical company, focuses on translating medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors, and rare genetic diseases. Its pipeline focuses on gene therapy programs for rare genetic disorders, chimeric antigen receptor (CAR) engineered T cell (CAR T) therapies for hematologic malignancies, and CAR T therapies for solid tumors. The company develop MB-107, a gene therapy program, potential curative treatment for X-linked severe combined immunodeficiency, a rare genetic immune system condition in which affected patients do not live beyond infancy without treatment. Its ex vivo lentiviral gene therapy is in two Phase 1/2 clinical trials. |
Nanoscope Therapeutics Dallas, Texas, United States | Nanoscope is focused on proprietary ambient light activated optogenetic therapy to restore vision in people suffering from all forms of Retinitis Pigmentosa. |
Neogap Therapeutics Stockholm, Sweden | NEOGAP Therapeutics AB is a privately held clinical stage biopharmaceutical company with the main focus on immuno-oncology. The company was founded in 2016 by Dr. Hans Grönlund. We are located at Therapeutic Immune Design, Centre for Molecular Medicine at Karolinska Institute, Stockholm, Sweden. Our vision is to increase overall survival among patients with cancer by boosting tumour-specific responses of the immune system. Our mission is to become an important player in the battle against cancer by development and commercialisation of our innovative cancer immunotherapy. |
Neurona Therapeutics South San Francisco, California, United States | Neurona is a biotherapeutics company developing neural cell therapies with single-dose curative potential for the treatment of chronic neurological disorders. Based on a novel neural cell lineage pioneered by the company’s scientific founders, Neurona has built a robust regenerative platform and is developing neuronal, glial, and gene-edited cell therapy candidates that provide long-term repair of dysfunctional neural networks for multiple neurological disorders. |
Nexcella Los Angeles, California, United States | Nexcella, Inc. is a Los Angeles, California based clinical-stage biopharmaceutical company engaged in the discovery and development of novel cell therapies for oncology and other indications. Our lead candidate, next generation BCMA-targeted CAR-T NXC-201 for relapsed/refractory AL amyloidosis and relapsed/refractory multiple myeloma, has produced 100% and 95% response rates in each indication, respectively, as of December 10, 2023 across 72 patients. NXC-201 has been awarded Orphan Drug Designation (ODD) by the FDA in both AL Amyloidosis and multiple myeloma. We believe NXC-201 has the potential to be the world’s first “Single-Day CRS” CAR-T, enabling faster return home for patients. Expansion into autoimmune indications is planned. Our N-GENIUS platform allows us to discover, develop, and manufacture cutting-edge cell therapies for patients in need. To learn more about Nexcella, Inc. visit us at www.nexcella.com. |
NexImmune Inc Gaithersburg, Maryland, United States of America | NexImmune, Inc., a clinical-stage biotechnology company, engages in developing therapies with curative potential for patients with cancer and other life-threatening immune-mediated diseases. It develops approaches to T cell immunotherapies based on its proprietary Artificial Immune Modulation (AIM) technology. The company has two product candidates in human trials, including NEXI-001 in acute myeloid leukemia, or AML; and NEXI-002 in multiple myeloma, or MM. NexImmune, Inc. was founded in 2011 and is headquartered in Gaithersburg, Maryland. |
NextCure Inc Beltsville, Maryland, United States of America | NextCure, Inc., a clinical-stage biopharmaceutical company, engages in discovering and developing novel immunomedicines to treat cancer and other immune-related diseases by restoring normal immune function. Its lead product candidate is NC318, which is in Phase II clinical trials for the treatment of advanced or metastatic solid tumors. The company is also developing NC410, which is in Phase I for novel immunomedicine designed to block immune suppression mediated by an immune modulator called Leukocyte-Associated Immunoglobulin-like Receptor 1; and NC762, an immunomedicine targeting a molecule called human B7 homolog 4 protein, or B7-H4. Its discovery and research programs include an antibody in preclinical evaluation of other potential novel immunomodulatory molecules. NextCure, Inc. has a license agreement with Yale University. The company was incorporated in 2015 and is headquartered in Beltsville, Maryland. |
NKGen Biotech Santa Ana, California, United States | NKGen Biotech is harnessing the power of the body's immune system through the development of unique Natural Killer (NK) cell therapies. NK cell adoptive immunotherapy enhances a patient's ability to destroy abnormal cells. The mission of NKGen Biotech is to improve patients' lives in a safe and natural way through innovative cell therapy products and to ultimately become the global leader in immune cell therapeutics. |
Northwest Biotherapeutics Bethesda, Maryland, United States of America | Northwest Biotherapeutics, Inc. is a development stage biotechnology company focused on discovering, developing and commercializing immunotherapy products that generate and enhance immune system responses to treat cancer. Currently approved cancer treatments are frequently ineffective, can cause undesirable side effects and provide marginal clinical benefits. The Company’s approach in developing cancer therapies utilizes its expertise in the biology of dendritic cells, which are a type of white blood cell that activate the immune system. The Company’s cancer therapies have been demonstrated in clinical trials to significantly extend both time to recurrence and survival, whilst providing a superior quality of life with no debilitating side effects when compared with current therapies. The Company’s platform technology, DCVax®, uses a patient’s own dendritic cells, the starter engine of the immune system. The dendritic cells are extracted from the body, loaded with tumor biomarkers or ‘‘antigens’’, thereby creating a personalized therapeutic vaccine. Injection of these cells back into the patient initiates a potent immune response against cancer cells, resulting in delayed time to progression and prolonged survival. The Company’s lead product candidate is DCVax®-Brain which targets Glioblastoma Multiforme (‘‘GBM’’), the most lethal form of brain cancer. DCVax®-Brain has entered a Phase II FDA-allowed clinical trial, which is designed and powered as a pivotal trial (i.e. a trial from which a company may go directly to product approval). Following this trial, the Company anticipates filing a biologic license application (or ‘‘BLA’’) with the FDA for DCVax®-Brain. DCVax®-Prostate, which targets hormone independent (i.e. late stage) prostate cancer, has also been cleared by the FDA to commence a Phase III clinical trial, which is also designed and powered as a pivotal trial. |
Novadip Biosciences Mont-Saint-Guibert, Belgium | Novadip Biosciences is a clinical-stage biopharmaceutical company founded to design, develop, and bring to the market a new class of regenerative tissue products to accelerate healing of large bone defects and injuries in a single treatment. Novadip’s lead allogeneic product, NVD-X3, employs a matrix containing multiple bioactive factors that induce accelerated tissue healing. Designed as an “off-the-shelf” product, NVD-X3 can be shipped and stored at room temperature and offers superior intraoperative handling characteristics. Novadip is also developing NVD-003, an autologous therapy derived from ASCs as a potential single treatment to save limbs and restore mobility in patients with congenital pseudarthrosis of the tibia (CPT). CPT is a rare pediatric bone condition. |
Obsidian Therapeutics Cambridge, Massachusetts, United States | Obsidian Therapeutics is pioneering controllable cell and gene therapies to deliver transformative outcomes for patients with intractable diseases. Obsidian’s programs apply our cytoDriveTM technology in cell and gene therapy products to control expression of proteins for enhanced therapeutic efficacy, including our lead program cytoTIL15 engineered to make TILs more effective for more patients. Our aspirations are high, and we’re built to deliver – well-funded with blue chip investors, engaged field-leading advisors, strong partnerships with industry leaders, and a highly experienced, dynamic, innovative and collaborative team, collectively focused on delivering transformative therapies in areas of greatest clinical need. Located in the heart of Cambridge with a new laboratory facility in Bedford, we’re proud of our diverse talented team and committed to cultivating an environment of inclusion where we strive to instill a strong sense of belonging, and support each Obsidianite to continuously learn and contribute their best work. We offer competitive salary and benefits, and potential for employee ownership through stock options. |
Oncternal Therapeutics San Diego, California, United States of America | Oncternal Therapeutics is a clinical-stage oncology company developing first-in-class, novel therapies that target a broad spectrum of cancers with large unmet medical need. By leveraging pathways absent or minimally expressed in normal cells or tissue, we aim to inhibit cancer cell growth while reducing potential harm to healthy cells. We are pursuing trials in chronic lymphocytic leukemia (CLL), mantle cell lymphoma (MCL), breast cancer, and Ewing sarcoma. Looking forward, we intend to rapidly broaden our pipeline and invest in the development of our innovative therapies across multiple cancers. |
OriCell Therapeutics Shanghai, China | Founded in 2015, Oricell Therapeutics aims to develop drugs with good efficacy and affordable prices to satisfy the globally unmet clinical needs through innovations and strives to become the world-leading developer of novel drugs for tumor immunotherapy. |
Ossium Health San Francisco, California, United States | Building a Healthier Future Our mission is to improve the health, vitality, and longevity of human beings through bioengineering. Who We Are At Ossium, we believe that science is humanity’s best weapon in the fight against disease, and we embrace the challenge of mobilizing the world’s healthcare ecosystem to win that fight. In pursuit of this goal, we empower our employees, maintain the highest standards of excellence, and are a force for good. PRINCIPLES The values we live by Impact We are mission-driven and results-oriented. We believe that a healthier world is a better one and that our work should drive positive change. Truth Embracing the truth empowers us to learn from both our successes and our mistakes. We follow the data wherever it leads. Ownership We hold ourselves and each other to the highest bar. We care deeply about the work we do and know that even the smallest details can make a difference. |
Passage Bio Philadelphia, Pennsylvania, United States | Passage Bio is a genetic medicines company based in Philadelphia, PA that is developing a deep pipeline of adeno-associated virus (AAV) delivered therapeutics for the treatment of rare monogenic central nervous system disorders (CNS). We seek to deliver transformative therapies to patients by leveraging our team’s decades of experience, including our founders, as well as the transformative potential genetic medicine technology to develop treatments that improve outcomes for patients with serious, life-threatening, rare CNS disorders. |
PDC*line Pharma Liège, Liège Belgium | Founded in 2014 as a spin-off of the French Blood Bank (EFS), PDC*line Pharma is a French-Belgian biotech company that develops an innovative class of active immunotherapies to treat cancers. This cancer vaccine contains proprietary, potent, and scalable plasmacytoid dendritic cell line (PDC*line), whose remarkable characteristics make them excellently suited for immunotherapy. PDC*line is much more potent to prime and boost antitumor antigen, including neoantigens, specific cytotoxic T-cells than conventional vaccines and improves the response to checkpoint inhibitors. PDC*line offers unique advantages: - PDC*line is a professional and universal antigen-presenting cell much more potent than conventional (myeloid) Dendritic cells; - unlike conventional autologous Dendritic Cells, PDC*line is very easy to expand in large quantities in bioreactors (no need of maturation factors) and having been exposed in vitro to targeted tumor antigens and irradiated, it can be stored for years. - The off-the-shelf product is thawed and injected to treat any patients with a cancer type expressing the selected antigens and expressing HLA-A2. Of note, different HLA may be used or added to extend the target population. - The approach is very versatile and applicable to any cancer type. Based on a first-in-human phase Ib study in melanoma, PDC*line Pharma focuses on the development of a clinical candidate for lung cancer (PDC*lung) and neoantigens (PDC*Neo). The company regroups a team of 42 persons led by Eric Halioua (CEO) and raised close to 61M€ in equity and loans since its foundation. March 2019, PDC*line Pharma granted exclusive license in South Korea and exclusive option in other Asian countries to LG Chem Life Sciences Company, for the development and commercialization of PDC*lung cancer vaccine for lung cancer. The total deal value is 108M€ (123M$) plus significant tiered royalties on net sales in Asia. |
Perceive Biotherapeutics South San Francisco, California, United States | A start-up developing novel ophthalmic therapeutics to treat major unmet needs for patients. |
Phoenix Molecular Designs San Diego, California | Phoenix Molecular Designs is a clinical-stage biotechnology company that is developing small molecule kinase inhibitors as a targeted therapy for cancer. We are focused on treating one of the most aggressive types of breast cancer, Triple Negative Breast Cancer (TNBC), which currently has limited durable options. Our patented cancer compounds position us in the lead to fast track our product to market as it fills an unmet medical need. Our cancer therapy has the potential to save lives and improve quality of life. |
Phoenix Nest Biotech Brooklyn, New York, US | Phoenix Nest Biotech is a biotechnology company that focuses on treating Sanfilippo Syndrome by collaborating with leading academics and utilizing licensed key technologies. |
PolTREG Gdańsk, Poland | PolTREG was established as a spin-off from the Medical University of Gdańsk in order to develop and commercialize patented TREG method. PolTREG is the most advanced company in TREGS cellular therapies worldwide, breakthrough therapies for type 1 diabetes (T1D) and multiple sclerosis (MS). PolTREG has very promising results regarding the safety and efficacy of TREGS therapy (completed phase I/II clinical trials in T1D and phase I in MS), ready for the next phase of trials. The company is after EMA Scientific Advice procedure. |
Prescient Therapeutics South Melbourne, Victoria, Australia | Prescient Therapeutics (ASX: PTX) is a clinical stage oncology company developing personalised medicine approaches to cancer, including targeted and cellular therapies. Cell Therapies OmniCAR: is a universal immune receptor platform enabling controllable cell activity and multi- antigen targeting with a single cell product. OmniCAR’s modular CAR system decouples antigen recognition from the cell signalling domain. It is the first universal immune receptor allowing post- translational covalent loading of binders to immune cells. OmniCAR is based on technology licensed from Penn and Oxford University; and other assets. Prescient is developing OmniCAR-T cells programs for next-generation CAR-T therapies for AML; Her2+ solid tumours, including breast, ovarian and gastric cancers; and GBM. CellPryme: Prescient's novel, ready-for-the-clinic, CellPryme-M technology enhances adoptive cell therapy performance by shifting T and NK cells towards a central memory phenotype, improving persistence, and increasing the ability to find and penetrate tumours. CellPryme-M is a 24-hour, non-disruptive process during cell manufacturing. Targeted Therapies PTX-100 is a first in class compound with the ability to block an important cancer growth enzyme known as geranylgeranyl transferase-1 (GGT-1). It disrupts oncogenic Ras pathways by inhibiting the activation of Rho, Rac and Ral circuits in cancer cells, leading to apoptosis (death) of cancer cells. PTX-100 is now in a Phase 1b expansion cohort study in T cell lymphomas, where it has shown encouraging efficacy signals and safety. PTX-200 is a novel PH domain inhibitor that inhibits an important tumour survival pathway known as Akt, which plays a key role in the development of many cancers, including breast and ovarian cancer, as well as leukemia. Unlike other drug candidates that target Akt inhibition, PTX-200 has a novel mechanism of action that specifically inhibits Akt without non-specific kinase inhibition effects. |
Prevail Therapeutics New York, New York, United States of America | Prevail Therapeutics was acquired by Eli Lilly and Company. At Prevail Therapeutics, we are developing a pipeline of gene therapies to slow or stop the underlying disease process for patients with Parkinson’s disease and other neurodegenerative disorders. We are leveraging recent breakthroughs in human genetics that point to potential disease-modifying targets for neurodegenerative diseases. Our programs utilize a precision medicine approach targeting patient populations with urgent unmet needs, where there are currently no available therapies that modify the progressive course of their disorders. |
Prime Medicine Cambridge, Massachusetts, United States of America | Prime Medicine was founded to bring the promise of gene editing to patients. We use Prime Editing, a next-generation technology that can “search and replace” to restore normal genetic function almost anywhere in the genome. Prime Editing searches to find the precise place in the genome to edit and replaces the segment of faulty DNA with a correct copy of DNA. A single Prime Editor can correct the individual mutations found across patients, meaning Prime Editing can potentially address more than 90 percent of known disease-causing genetic mutations. We envision a world where this technology can cure, halt, and ultimately prevent genetic diseases, providing lifelong benefit to patients. Prime Medicine offers the opportunity to shape the future of gene editing and transform how medicines are used to treat disease. Realizing the promise of Prime Editing requires a talented team with diversity of viewpoints and expertise. We’re building a group of dedicated, scientifically curious individuals who are passionate about helping patients live longer, healthier lives. |
Q Therapeutics Salt Lake City, Utah | Q Therapeutics is a biotechnology company based out of 417 S Wakara Way, Salt Lake City, UT, United States. |
Quell Therapeutics London, United Kingdom | An exciting new cell therapy immunology biotech committed to transforming patient's lives in solid organ transplantation and auto-immune conditions. Our company is growing and we are exciting about the future! Our vision is to become a world leader in new cell therapy and the people are at the heart of what we do. We treat our people with respect, working collaboratively across the business, taking ownership and accountability for the outcomes and results we delivery. Most of all we work with integrity to innovate and bring the best version of ourselves to the job every day. |
Rege Nephro Co., Ltd. 46-29 Yoshidashimoadachi-cho, Sakyo-ku, Med-Pharm Collaboration bldg. Kyoto University, Kyoto, Kyoto 606-8501, JP | Rege Nephro is a clinical-stage biotech company developing cell therapy for chronic kidney disease (CKD) and small molecule for autosomal dominant polycystic kidney disease (ADPKD), and founded in 2019. There are 13 million patients with CKD in Japan, of which 340,000 are severe CKD patients forced to undergo artificial dialysis. The healthcare cost of dialysis is $12 billion, 4% of the total healthcare cost of Japan. However, there is still no curative treatment for CKD. Rege Nephro tries to treat patients suffering from CKD with cell therapy using nephron progenitor cells differentiated from iPS cells. |
Regenerative Patch Technologies Portola Valley, California, United States | Regenerative Patch Technologies LLC (RPT) is a CA-based biotechnology company focused on the development of stem cell-based therapies designed to reverse blindness caused by advanced dry age related macular degeneration (AMD). |
ReNeuron Bridgend, Wales, United Kingdom | Our focus at ReNeuron is on our CustomEx exosome technology platform, producing exosomes with unique tissue targeting tropisms to deliver a payload of choice to a preferred cell type. Our mission is to partner and develop novel exosome therapeutics for diseases with significant unmet needs. |
Repare Therapeutics Montreal, Quebec, Canada | Repare Therapeutics is a leading clinical-stage precision oncology company enabled by our proprietary synthetic lethality approach to discovering and developing novel therapeutics. We use our genome-wide, CRISPR-enabled SNIPRx® platform to systematically discover and develop highly targeted cancer therapies focused on genomic instability, including DNA damage repair. Our company was founded in 2016 by field-leading academics to systematically employ synthetic lethality insights and platforms and develop new precision oncology medicines. For more information, please visit www.reparerx.com. Scam Prevention Notice It has come to our attention that individuals posing as representatives of Repare Therapeutics have been engaging in fraudulent recruiting activities, offering fake job opportunities to unsuspecting candidates. These scams are designed to deceive job seekers into providing personal information or financial details under false pretenses. Please note that we will only contact you about a position that is posted on our careers page and official email communications from Repare employees will only come from a @reparerx.com email. Repare has a formal process which includes candidate application through the Repare career page (https://www.reparerx.com/careers/). If the job is not posted on the official Repare Therapeutics careers page, then it is not a legitimate position. If you have any questions or concerns regarding job opportunities at Repare Therapeutics, please don't hesitate to reach out to our HR department directly via Contact Us section (https://www.reparerx.com/contact/). Thank you for your vigilance and understanding. |
Rinri Therapeutics Sheffield, South Yorkshire, United Kingdom | Rinri Therapeutics is a private biotechnology company developing first-in-class cell therapies to restore hearing. Hearing loss affects 350m people in the world; 90% caused by sensorineural hearing loss (SNHL). There are currently no disease-modifying therapeutics for SNHL patients. Rinri's pioneering technology seeks to reverse SNHL through the regeneration of the damaged sensory cells in the inner ear, the cochlea. Rinri is backed by Boehringer Ingelheim Venture Fund (BIVF), UCB Ventures, BioCity and the University of Sheffield. |
Rocket Pharmaceuticals New York, New York, United States of America | Rocket Pharmaceuticals (NASDAQ: RCKT), Ltd is a biotechnology company focused on seeking cures through molecular and gene therapy. We are developing first-in-class treatments for patients with rare and often devastating orphan diseases using a lentiviral-based and adeno-associated virus (AAV) therapy approach. We have developed rewarding partnerships with a number of leading international research institutions as well as with centers of manufacturing and cell processing expertise, while building an internal team to drive the research, development, clinical and regulatory programs. Ultimately we aim to launch and globally commercialize innovative gene therapies in disease areas with high unmet need. |
Sangamo Therapeutics Brisbane, California, United States | Sangamo Therapeutics is a genomic medicine company conducting research & development across four distinct but complementary technology platforms – gene therapy, cell therapy, in vivo genome editing, and in vivo genome regulation. The optionality and diversity inherent to Sangamo’s technology platform enables us to design therapeutic approaches to resolve the underlying genetic causes of disease, using whichever technology is best suited to deliver that treatment. Because Sangamo’s technology platforms are related, we are applying learnings across studies to optimize each individual platform and uncover research and development paths forward. Sangamo is best known for developing our proprietary gene editing technology, zinc finger nucleases (ZFNs). Our technology team continues to optimize ZFNs for the three key dimensions of genome editing: precision, efficiency, and specificity. Ex vivo gene-edited cell therapy is the most straightforward application of ZFN technology. It builds on early experience with HIV clinical studies and is applied to hematological disorders and oncology. Our cell therapy portfolio also includes ex vivo engineered regulatory T-cells (CAR-Tregs) for application in autoimmunity and inflammation. The insights gained about molecular biology, cassette engineering, and delivery through in vivo application of ZFNs led to our gene therapy program using AAV, which is advancing in the clinic. Zinc finger protein engineering and AAV delivery are also foundational for our zinc finger protein-transcription factor (ZFP-TF) genome regulation technology, which we are applying in CNS diseases. With our robust history of genomic medicine firsts, unmatched technology platform, and growing manufacturing and clinical development capabilities, Sangamo believes we are the company to realize the vision of genomic medicine. |
Sanguine Bioscience Woburn, Massachusetts, US | |
SCG Cell Therapy Singapore | Established and headquartered in Singapore, SCG Cell Therapy Pte Ltd (SCG) is a leading biotechnology company that combines regional advantages in Singapore, China and Germany, to cover the entire value chain from innovative drug research and discovery, manufacturing, clinical development and commercialization. SCG specializes in the development of novel immunotherapies in infections and its associated cancers. The company targets the most common cancer-causing infections: helicobacter pylori, human papillomavirus, and hepatitis B, and develops a broad and unique pipeline of T cell therapies, antibodies, and therapeutic vaccines against infections and to prevent and cure its associated cancers. SCG collaborates with leading scientists and researchers to bring first-in-class medical products/technologies to enhance innovation in medical product development. |
Seraxis Germantown, Maryland, United States | Seraxis is a privately owned biotechnology company. Our GMP lab is located in Germantown, Maryland, in the heart of the vibrant BioHealth Capital Region. Launched in early 2013, Seraxis used proprietary technologies to develop a cell replacement therapy, SR-01, that is more effective and safer than embryonic stem cell-derived therapies. Our highly pure insulin-producing cells are micro-encapsulated through a proprietary technology that enables the function of human pancreas cells in type-1 diabetes models without the need for immuno-suppression. Seraxis intends to bring SR-01 to the clinic in order to provide a cure for the devastating disease of diabetes. |
Skyline Therapeutics Cambridge, Massachusetts, US | Skyline Therapeutics is a biotechnology company that focuses on the discovery, development and delivery of innovative gene therapy treatments. |
Smart Immune Paris, Île-de-France, France | Smart Immune is a clinical-stage biotechnology company developing ProTcell, a thymus-empowered T-cell therapy platform to fully and rapidly re-arm the immune system, enabling next-generation allogeneic T-cell therapies for all. The company is a spin-off from the Imagine Institute of Genetic Diseases, and was founded in 2017 by Karine Rossignol, PharmD-HEC, Marina Cavazzana, MD-PhD and Isabelle André, PhD to transform outcomes for patients with life-threatening diseases such as high-risk blood cancers and primary immunodeficiencies. Smart Immune has clinical partnerships with leading institutions in the US and Europe. The ProTcell platform, already in Phase I/II clinical trials, enables the accelerated recovery of a complete immune repertoire in patients fighting cancer and infection. The ProTcell platform introduces potent, allogeneic T-cell progenitors which are then differentiated by the thymus into fully functional T-cells, to ultimately develop an ‘off the shelf’ T-cell medicine. The company is headquartered in Paris, France, at Paris Biotech Santé. |
Solid Biosciences Boston, Massachusetts, United States | Solid Biosciences (Solid) is a life science company singularly focused on solving Duchenne muscular dystrophy (Duchenne). We aim to be the center of excellence across the entire disease spectrum, serving as a catalyst to bring together those with the expertise in science, technology, disease management and care. Disease-focused and founded by a family directly impacted, our mandate is simple yet comprehensive - attack the roots of the disease and improve daily life for patients. |
Sorrento Therapeutics San Diego, California, United States | Sorrento Therapeutics is an antibody-centric, clinical stage biopharmaceutical company developing new treatments for cancer, inflammation and autoimmune diseases. Sorrento's lead products are multiple late-stage biosimilar and biobetter antibodies, as well as clinical CAR-T therapies targeting solid tumors. |
SOTIO Biotech Prague, Czech Republic | SOTIO Biotech is shaping the future of cancer immunotherapies by translating compelling science into patient benefit. SOTIO’s robust clinical pipeline includes a differentiated superagonist of the attractive immuno-oncology target IL-15, a proprietary technology designed to improve on the efficacy of CAR T therapies and a new generation of potent and stable antibody-drug conjugates (ADCs). SOTIO is a member of the PPF Group. |
SparingVision Paris, France | SparingVision is a genomic medicines company, translating pioneering science into vision-saving treatments. Founded to advance over 20 years of world-leading ophthalmic research from its scientific founders at the Paris Vision Institute, SparingVision is leading a step shift in how ocular diseases are treated, moving beyond single gene correction therapies. At the heart of this is a pipeline of gene independent treatments for rod-cone dystrophies. Lead products, SPVN06 and SPVN20, address mid and late stages of retinitis pigmentosa (RP)respectively. RP is the most common inherited retinal disease affecting two million people worldwide. These novel medicines could form the basis of a suite of new sight-saving treatments with potential applications across many other retinal diseases, regardless of genetic cause. The Company is supported by a strong, internationally renowned team who aim to harness the potential of genomic medicine to deliver new treatments to all ocular disease patients as quickly as possible. SparingVision has raised €60 million to date and its investors include 4BIO Capital, Advent France Biotechnology, Bpifrance, Foundation Fighting Blindness (US), Fondation Voir & Entendre, UPMC Enterprises, Jeito Capital and Ysios Capital. For more information, please visit www.sparingvision.com. |
SpliceBio Barcelona, Spain | SpliceBio is a biotechnology company exploiting Protein Splicing to develop the next generation of gene therapies. Our proprietary platform is based on technology developed in the Muir Lab at Princeton University after more than 20 years of pioneering intein and protein engineering research by our co-founders. The company is backed by UCB Ventures, Ysios Capital, NEA, Gilde Healthcare, Novartis Venture Fund and Asabys Partners. |
Steminent Biotherapeutics Taipei, Taiwan | Steminent Biotherapeutics Inc. is the leading stem cell clinical development company in Taipei, with subsidiaries in San Diego and Shanghai, dedicated to the development of novel cellular therapeutics for the treatment of diseases with unmet or under-served medical needs. Steminent utilizes advanced, proprietary processes and know how to isolate, purify, amplify and manufacture standardized stem cell products of the highest quality for research and clinical development in multiple indications. Steminent’s Stemchymal® allogeneic cell therapy R&D program has generated a portfolio of clinical stage therapeutic candidates for multiple diseases including: Phase II; Spinocerebellar Ataxia (PolyQ SCA), Phase I; Osteoarthritis of the Knee, and Phase I: Acute Liver Failure. 幹細胞醫藥產業先驅的仲恩生醫,以推動異體間葉幹細胞治療為核心,擁有世界一流的幹細胞研發團隊與技術。獨家核心技術平台Stemchymal®,以領先全球的幹細胞分離、純化、增生技術,應用於自體及異體的脂肪間葉幹細胞治療。 仲恩生醫以最嚴謹的製藥規範與品質管理,不斷累積科學實證,並透過國內外頂尖醫學中心臨床試驗證實仲恩生醫幹細胞產品的安全與療效。除此,仲恩生醫透過與臨床醫師及國內外合作夥伴緊密的合作,持續地針對臨床未被滿足的需求,持續開發Stemchymal®優質細胞產品於急性肝衰竭及其他適應症之應用,期許未來多國的二期臨床試驗的結果,以及在其他不治和難治病症上的努力和不斷的進步,將可盡早實現幹細胞藥物在臨床應用的價值,嘉惠無藥可醫之病患。 2016年藉由台日再生醫療的策略結盟,深化台日雙方的合作關係,更促進再生醫學產業化和實用化的發展。2018年向美日兩國申請的臨床試驗二期申請案,相繼於7月14日和7月29日正式通過。一旦於日本完成異體間葉幹細胞新藥的二期臨床試驗,將依日本再生醫藥專法申請孤兒藥資格及最高7至10年暫時性藥證許可。 |
Strand Therapeutics Boston, Massachusetts, United States of America | Strand was founded by biological engineers working together at the Massachusetts Institute of Technology (MIT). Building on the idea of creating smart therapies capable of making sophisticated decisions, they sought to apply this concept to the emerging field of mRNA therapeutics. This area was previously untapped by traditional synthetic biology, leading us to build our very own mRNA programming language, creating the world’s first platform for mRNA smart therapies. Strand’s mRNA programming technology promises to make mRNA therapies safer and more effective by programming the location, timing, and intensity of therapeutic protein expression inside a patient’s body via mRNA-encoded logic circuits. These circuits have the ability to implement cell-type specific expressions by sensing and classifying the unique mRNA expression signatures of cells and control the dosage of protein expression through responses to exogenously administered small molecules. |
Synbio Technologies Monmouth Junction, New Jersey, US | Synbio Technologies aims to become the most trusted provider of DNA solutions that empower scientific discoveries by providing highly-accurate and cost-effective synthetic biology services and products for researchers around the world. We have developed a full range of DNA reading (sequencing), DNA writing (synthesis), and DNA editing (engineering) capabilities for various applications, including diagnostic DNA probes, precision medicine, protein production, antibody discovery, vaccine development, novel enzymes, molecular breeding, biofuel implication, and more. Our scientific capabilities cover all facets of DNA synthesis and engineering, offering a full range of support for molecular biology work, including but not limited to, oligo synthesis, gene synthesis, subcloning & PCR cloning, plasmid preparation, mutagenesis, DNA variant libraries, CRISPR sgRNA, protein expression and purification in multiple systems. Relying on our strong DNA sequencing, synthesis, engineering technology platform, and expertise in bioinformatics analysis, we have developed proprietary algorithms to facilitate and expedite the antibody discovery process. Our antibody discovery services include hybridoma sequencing, immune repertoire sequencing, antibody design and production, AI-guided heavy chain and light chain pairing, antibody humanization, affinity maturation, and sdAb. |
Takara Bio Shiga, Japan | Takara Bio Inc. is committed to improving the human condition through biotechnology. Starting as the biomedical business of Takara Shuzo, a fermentation and beverage company founded in 1925 (now Takara Holdings Company, Inc.), Takara Bio has focused on developing research tools for the life sciences, cell and gene therapy technologies, and nutraceuticals since its launch in 1967. The three business units of TAKARA BIO INC are: • Genetic Engineering Research - manufactures and sells research reagents and scientific instruments used by biotechnology researchers worldwide, as well as contract research services. • Agribio - produces and sells health food products whose functionality have been proven by biotechnology, and conducts a mushroom business based on technologies for large-scale mushroom production. • Gene Medicine - conducts clinical development projects to work toward commercializing cell and gene therapies centered on a highly efficient gene transduction method and a lymphocyte expansion-culture system, both using RetroNectin® reagent. |
Taysha Gene Therapies Dallas, Texas, United States | Taysha Gene Therapies, Inc., a gene therapy company, focuses on developing and commercializing adeno-associated virus-based gene therapies for the treatment of monogenic diseases of the central nervous system. It primarily develops TSHA-101 for the treatment of GM2 gangliosidosis; TSHA-118 for the treatment of CLN1 disease; and TSHA-102 for the treatment of Rett syndrome. Taysha Gene Therapies, Inc. has a strategic partnership with The University of Texas Southwestern Medical Center to develop and commercialize transformative gene therapy treatments; and collaboration with Yale University to advance mini-gene payloads for an AAV gene therapy for the treatment of neurodevelopmental disorders. The company was incorporated in 2019 and is based in Dallas, Texas. |
TC BioPharm Holytown, North Lanarkshire, Scotland, United Kingdom | TC BioPharm is a publicly traded, clinical-stage cell therapy company developing advanced allogeneic CAR-T cell therapy products for the treatment of cancer, as well as developing gamma delta T cell therapies for the treatment of infectious disease. We have a growing team based in our Glasgow office, alongside a wide range of scientific advisors, and partner organisations. |
TCRCure Guangzhou, China | TCRCure Biopharma Corp is a world-class, “One-Stop Shop” cell therapy operation built across the U.S. and China. We deliver the latest cell therapy advances directly to patients with solid tumors. As of 2021, we have treated more than 50 patients and built more than a dozen CAR-T/TCR-T pipelines. TCRCure’s cutting-edge platform technologies include antigen-specific TCR/CAR discovery, immune-cell engineering, and industry-scale viral vector production. We operate 40,000 sq. ft. of specialized facilities on both sides of the Pacific, including scalable GMP capacity for cell production and viral manufacturing. TCRCure works closely with world-class hospitals in China and the United States for clinical research in different solid tumors. Current indications including cervical cancer, ovarian cancer, nasopharyngeal carcinoma, lung squamous carcinoma, melanoma, sarcoma, endometrial cancer, and brain cancer. |
Tenaya Therapeutics South San Francisco, California, United States | Tenaya Therapeutics, Inc. develops therapeutics for heart failure. It offers cellular regeneration platform, that enables in vivo reprogramming of cardiac fibroblasts into cardio myocytes by delivering proprietary transcription factors; gene therapy platform, that enables the targeted delivery of a payload to cardiac fibroblasts; and precision medicine platform, which provides personalized medicine approach to treat heart disease. The company was incorporated in 2016 and is based in South San Francisco, California. |
TolerogenixX Heidelberg, Germany | TolerogenixX vision is to innovate Immunosuppression with its newly developed MIC Cell Treatments. MIC Cell Treatment is a curative ATMP approach to achieve a sustained regulation of the immune system in Transplant - and Autoimmune Disease Patients. TolerogenixX has been able to generate strong pre-clinical as well as clinical data in the indications of Transplantation and Autoimmune Diseases e.g. SLE since its foundation in 2016. |
Tr1X San Diego, California, United States | Tr1X Inc. (pr. "Trix") is a biotechnology company dedicated to the development of novel cellular immunotherapies for patients with autoimmune and inflammatory diseases. The Company uses its proprietary platform for the generation of first-in-class cell therapy products aimed at rebalancing the immune system and restoring homeostasis, leading to long term tolerance. Tr1X is headquartered in La Jolla, CA. |
TRACT Therapeutics Chicago, Illinois, United States | TRACT Therapeutics, Inc. has developed a novel approach for restoring immune balance in patients receiving a solid organ transplant or in patients suffering from an autoimmune disease. This therapeutic approach may potentially reduce the chance of organ rejection following a transplant or transfer a patient suffering from an autoimmune disease into a state of remission. The proprietary technology is based on a platform which can be utilized to treat solid organ transplant patients, as well as a number of autoimmune disorders such as Crohn’s Disease. For organ transplant, it shows great promise in preventing rejection of a donated organ, thereby, reducing the need for a second transplant. Over the next few years, TRACT’s TregCel™ personalized therapy could transform the medical approach in transplant medicine by dramatically reducing or eliminating the lifelong use of toxic anti-rejection drugs. |
Transgene Bas-Rhin, France | Cancer immunotherapy Transgene (Euronext: TNG) is a biotechnology company focused on designing and developing novel immunotherapies for the treatment of cancer. |
Triumvira Immunologics Austin, Texas, United States | At Triumvira Immunologics, Inc., our team is dedicated to a vision of finding a cure and providing new hope for patients with life-threatening disease. Co-founded in 2015 by licensing technology out of McMaster University and Bloom Burton & Co., Triumvira is working to achieve this vision by developing a proprietary T cell Antigen Coupler (TAC) technology, thought to be safer and more efficacious than current cancer treatments including chimeric antigen receptor (CAR) and engineered T cell receptor (TCR) therapies. We are an emergent US company with operations in the United States and Canada. We bring together talent from a wide range of disciplines including immunobiology, drug development, fundraising, commercialization and finance, with decades of experience across big pharma and small biotech, multiple industries and academia. We are passionate about science that pushes the limits, the patients that drive the heart of our work, and the wellness of our employees. We embrace flexibility. If you are looking for a career where you are encouraged to be innovative, challenge the status quo, and have some fun while doing it, Triumvira is the place for you. For more information about Triumvira career opportunities, please see our Careers web page at: https://triumvira.com/contact-us/careers/ |
Uniqure NV Amsterdam, Netherlands | uniQure is delivering on the promise of gene therapy - single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary and partnered gene therapies to treat patients with severe genetic diseases. |
ViGenCell Seocho, Seoul-t'ukpyolsi, South Korea | ViGenCell is a medical company that specializes in immune cell therapy. |
Virogin Biotech Vancouver, Canada | Virogin Biotech is a biotechnology company that specializes in oncolytic virus drug development to create impactful drugs for patients with the highest medical need. |
Vivet Therapeutics Paris, France | Vivet Therapeutics is dedicated to developing innovative gene therapy treatments for orphan diseases. Vivet Therapeutics is focused on optimizing gene therapy through collaborating with the Fundacion para la Investigacion Medica Aplicada (CIMA, Universidad de Navarra) to develop new AAV vectors specifically targeting the liver and generating new technologies to optimize gene delivery and long term expression. |
Vor Biopharma Cambridge, Massachusetts, United States | We are a cell therapy company that combines a novel patient engineering approach with targeted therapies to provide a solution for patients suffering from hematological malignancies. |
Voyager Therapeutics Cambridge, Massachusetts, United States | Voyager Therapeutics, Inc. (Nasdaq: VYGR) is a biotechnology company dedicated to leveraging the power of human genetics to modify the course of – and ultimately cure – neurological diseases. Our pipeline includes programs for Alzheimer’s disease, amyotrophic lateral sclerosis (ALS), Parkinson’s disease, and multiple other diseases of the central nervous system. Many of our programs are derived from our TRACER™ AAV capsid discovery platform, which we have used to generate novel capsids and identify associated receptors to potentially enable high brain penetration with genetic medicines following intravenous dosing. Some of our programs are wholly owned, and some are advancing with partners including Alexion, AstraZeneca Rare Disease; Novartis Pharma AG; Neurocrine Biosciences, Inc.; and Sangamo Therapeutics, Inc. For more information, visit www.voyagertherapeutics.com. Voyager Therapeutics® is a registered trademark, and TRACER™ is a trademark, of Voyager Therapeutics, Inc. |
Wugen St. Louis, Missouri, United States | Wugen is a clinical-stage biotechnology company developing the next generation of off-the-shelf memory natural killer (NK) and CAR-T cell therapies for cancer. Wugen is leveraging its proprietary Moneta platform and deep genomic engineering expertise to pioneer a new class of memory NK cell therapies to treat hematological and solid tumor malignancies. Wugen is based out of St. Louis, Missouri and San Diego, California. |
Xencor Inc Pasadena, California, United States | Xencor, Inc., a clinical stage biopharmaceutical company, focuses on the discovery and development of engineered monoclonal antibody and other protein therapeutics to treat severe and life threatening diseases. It develops its antibody product candidates to treat cancer and autoimmune diseases. The company’s product candidates include XmAb5871, an immune inhibitor, which has been completed Phase II clinical trial for the treatment of autoimmune diseases; AIMab7195, which is in development and commercialization stage. Its product candidates also comprise XmAb14045, a bispecific antibody which is in Phase I clinical trial for the treatment of acute myeloid leukemia; plamotamab (XmAb13676) that is in Phase I clinical trial to treat B-cell malignancies; and XmAb18087, which is in Phase I clinical trial for neuroendocrine tumors and gastrointestinal stromal tumors. In addition, the company offers Tafasitamab, an antibody drug candidate for the treatment of patients with relapsed/refractory diffuse; AMG424, a bispecific antibody that targets CD38 and CD3, which is in Phase I clinical trial for the treatment of various myeloma; and AMG509, a bispecific antibody that is in preclinical development stage to treat prostate cancer. Further, it is developing bispecific antibodies to treat various cancers, such as XmAb20717, XmAb22841, and XmAb23104 which are in preclinical Phase. Additionally, it offers XmAb24306, initial cytokine candidate which is in Phase I clinical trial. The company has collaboration and license agreement with Novartis Institutes for BioMedical Research, Inc.; Amgen Inc.; Catalent Pharma Solutions LLC.; Janssen Biotech, Inc.; MorphoSys AG; Incyte Corporation; and The University of Texas MD Anderson Cancer Center. Xencor, Inc. was founded in 1997 and is headquartered in Monrovia, California. |
Xintela AB Lund, Skåne, Sweden | Xintela develops medical products in stem cell therapy and targeted cancer therapy based on the Company's cell surface marker integrin α10β1 which is found on mesenchymal stem cells and on certain aggressive cancer cells. The stem cell marker is used to select and quality-assure the patent-protected stem cell product XSTEM®, which is in clinical development for treatment of knee osteoarthritis and difficult-to-heal leg ulcers. The company produces XSTEM for the clinical studies in its GMP-approved manufacturing facility. In cancer therapy, which is run by the wholly owned subsidiary Targinta AB, therapeutic antibodies, targeting integrin α10β1 (First-in-Class) are being developed for the treatment of triple-negative breast cancer and the brain tumor glioblastoma. |
XyloCor Therapeutics Wayne, Pennsylvania, United States | XyloCor Therapeutics is a development-stage, biotechnology company dedicated to developing first-in-class gene therapies for patients with unmet medical need in cardiovascular disease. |
YolTech Therapeutics Shanghai, CN | YolTech Therapeutics is a biotech focuses on combining mRNA and gene editing technology to develop next-generation mRNA drugs and in vivo gene editing drugs.Our company is a pre-clinical stage gene editing biotech, has built leading genome editing platform and mRNA-LNP library. It possesses strong capability of novel Cas and base editors discovery and exceptional production capacity for LNP drug manufactory, with strong IP protection globally. It has created a pipeline with 10 genetic medicines focusing on cardiovascular diseases, infectious diseases and rare diseases. |