List of Cell Therapies Companies with Phase 3 Active Clinical Trial - 81
Search through the full list with powerful filtering options
Want this in Excel? Get Quote NowCompany | About |
---|---|
2seventy Bio Cambridge, Massachusetts, United States | Our name, 2seventy bio, reflects why we do what we do - TIME. Cancer rips time away, and our goal is to work at the maximum speed of translating human thought into action – 270 miles per hour – to give the people we serve more time. We are building the leading immuno-oncology cell therapy company, focused on discovering and developing new therapies that truly disrupt the cancer treatment landscape With a deep understanding of the human body’s immune response to tumor cells and how to translate cell therapies into practice, we’re applying this knowledge to deliver next generation cellular therapies that focus on a broad range of hematologic malignancies, including the first FDA-approved CAR T cell therapy for multiple myeloma, as well as solid tumors. Our research and development is focused on delivering therapies that are designed with the goal to “think” smarter and faster than the disease. Importantly, we remain focused on accomplishing these goals by staying genuine and authentic to our “why” and keeping our people and culture top of mind every day. |
4D Molecular Therapeutics Emeryville, California, United States of America | 4DMT is a clinical stage biopharma company inventing and developing innovative products to unlock the full potential of genetic medicine to treat large market and rare diseases. Our vector discovery platform Therapeutic Vector Evolution harnesses the power of directed evolution, a Nobel Prize-winning technology, with approximately one billion synthetic AAV capsid-derived sequences to invent customized and evolved vectors with improved therapeutic profiles compared to wild-type vectors. Our product design, development and manufacturing engine empowers us to efficiently create our valuable and diverse product pipeline. This combination of bold innovation and relentless execution gives 4DMT the capability to revolutionize genetic medicines and to strive for potential curative therapies. We have built a deep portfolio of genetic medicine product candidates, with five product candidates in clinical trials for seven patient populations. We are taking genetic medicine to new heights and into new disease areas to overcome challenges not addressed by existing medicines. This incredible challenge drives our relentless and talented team. Our people are the essence of our inspiring, innovative and collaborative environment that encourages individual contributions and provides opportunities for career development. We seek the best in the industry who identify with our mission and our bold and innovative results-driven approach. Our 4 Guiding Principles drive the way we work together: - Dare to Cure - Break Boundaries - Beyond Yourself - Prepare & Execute Relentlessly Join us in creating the future of genetic medicine. |
Abcuro Newton, Massachusetts, United States | Abcuro is a clinical stage biotechnology company developing treatments for autoimmune and cancer indications modulated by cytotoxic T and NK cells that express the inhibitory immune checkpoint receptor KLRG1 (killer cell lectin-like receptor G1). The company is advancing ABC008, a first-in-class anti-KLRG1 antibody designed to deplete cytotoxic T cells that attack healthy muscle tissue in patients with inclusion body myositis (IBM), into clinical studies. IBM is a progressive and debilitating inflammatory skeletal muscle condition with no available pharmaceutical therapies. Abcuro has received FDA orphan drug designation for ABC008 in IBM. The company is also advancing ABC015, an anti-KLRG1 blocking antibody capable of reactivating inhibited cytotoxic T and NK cells in the tumor microenvironment. |
Abeona Therapeutics New York, New York, United States of America | Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing cell and gene therapies for serious diseases. Prademagene zamikeracel (pz-cel) is Abeona’s investigational autologous, COL7A1 gene-corrected epidermal sheets currently in development for recessive dystrophic epidermolysis bullosa. The Company’s fully integrated cell and gene therapy cGMP manufacturing facility served as the manufacturing site for pz-cel used in its Phase 3 VIITAL™ trial, and is capable of supporting commercial production of pz-cel upon FDA approval. The Company’s development portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical need. Abeona’s novel, next-generation AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases. |
Adverum Biotechnologies Inc. Redwood City, California, United States of America | Adverum Biotechnologies, Inc., a clinical-stage gene therapy company, engages in developing gene therapy product candidates to treat ocular and rare diseases. Its pipeline of product candidates includes ADVM-022, an adeno- associated virus (AAV). 7m8-aflibercept for the treatment of wet age-related macular degeneration; ADVM-043, an investigational gene therapy candidate for the treatment of alpha-1 antitrypsin deficiency; and ADVM-053, a preclinical gene therapy product candidate for the treatment of hereditary angioedema. The company has collaboration agreements with Editas Medicine, Inc. to leverage its proprietary AAV vectors for genome editing technologies to treat up to five inherited retinal diseases; and Regeneron Pharmaceuticals, Inc. for the development of up to eight distinct ocular therapeutic targets. The company was formerly known as Avalanche Biotechnologies, Inc. and changed its name to Adverum Biotechnologies, Inc. in May 2016. Adverum Biotechnologies, Inc. was founded in 2006 and is headquartered in Redwood City, California. |
AIVITA Biomedical Irvine, California, United States | AIVITA Biomedical is an Irvine, CA based company focused on the advancement of commercial and clinical-stage programs utilizing curative and regenerative medicines. We leverage our unique expertise in stem cell growth and directed, high-purity differentiation to develop safe, efficient and economical manufacturing processes for therapeutic development. These proprietary capabilities enable us to turn therapeutic discovery into reality, developing revolutionary cell-based treatments and healthcare products for the patients who need them. AIVITA is developing treatments for cancer, vision loss, and is bringing next-generation skincare formulations to consumers through its commercial product offerings. |
ALK Abello ALK Corporate Headquarters, Boege Alle 1, Hoersholm, DK-2970, DK | At ALK, we go to work every day to make a difference for the many people with allergies around the world. It is our ambition to make life with allergy surprisingly simple for as many people as possible, and we build upon our strong heritage as a pioneer in allergy immunotherapy, when we continue to innovate, develop and market new products and solutions to help improve the health of people with allergies. Every day, our employees contribute to our aspiration of becoming the world leader in allergy by coming to work to do their best in the pursuit of providing new and better allergy treatments and solutions. We work in a high-energy, international setting where it is imperative that our people thrive and develop. Creativity, influence and opportunities are key elements in developing our people. ALK is a global company with production sites in Denmark, France, Spain and the United States. ALK was founded in Copenhagen in 1923 and today we employ around 2,900 people around the world. Our global headquarters is located in beautiful surroundings in Hørsholm, north of Copenhagen. Read more about ALK at www.alk.net |
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/ |
Amicus Therapeutics Philadelphia, Pennsylvania, United States of America | Amicus Therapeutics, Inc., a biotechnology company, engages in the discovery, development, and commercialization of therapies to treat a range of rare and orphan diseases. The company offers Galafold, an orally administered small molecule pharmacological chaperone for the treatment of Fabry disease. It is also conducting Phase 3 (ATB200-03) clinical study of AT-GAA for Pompe disease. The company has collaboration and license agreements with Nationwide Children's Hospital and University of Pennsylvania. Amicus Therapeutics, Inc. was founded in 2002 and is headquartered in Philadelphia, Pennsylvania. |
Ansun BioPharma San Diego, California, United States | Ansun Biopharma is a late-stage clinical development company dedicated to saving the lives of vulnerable patient populations through the creation of first-in-class biologics. Our lead product, DAS181, is a multi-function sialidase fusion protein for host-cellprotection in respiratory virus infection, including parainfluenza, influenza and COVID19,. We are also advancing several additional programs in oncology and autoimmune diseases using our sialidase platform technology. |
Arcellx Inc Gaithersburg, Maryland, United States | Arcellx, Inc., a clinical-stage biotechnology company, engages in the development of various immunotherapies for patients with cancer and other incurable diseases. The company’s lead ddCAR product candidate is CART-ddBCMA, which is in phase 1 clinical trial for the treatment of patients with relapsed or refractory (r/r) multiple myeloma (MM). It is also developing ACLX-001, an immunotherapeutic combination composed of ARC-T cells and bi-valent SparX proteins targeting BCMA to treat r/r MM; ACLX-002 and ACLX-003 for treating r/r acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS); and other AML/MDS product candidates, as well as solid tumor programs. The company was formerly known as Encarta Therapeutics, Inc. and changed its name to Arcellx, Inc. in January 2016. Arcellx, Inc. was incorporated in 2014 and is headquartered in Gaithersburg, Maryland. |
Beacon Therapeutics Alachua, Florida, United States of America | Beacon Therapeutics is an ophthalmic gene therapy company founded in 2023 to restore and improve the vision of patients with a range of prevalent and rare retinal diseases that result in blindness. The Company has an established scientific foundation that combines a late-stage development candidate to treat X-linked retinitis pigmentosa (XLRP), as well as two preclinical programs, one targeting dry age-related macular degeneration (AMD) and another in-licensed from the University of Oxford targeting cone-rod dystrophy (CRD), an inherited retinal disease. Lead development candidate AGTC-501, is a gene therapy program in Phase II clinical trials for the treatment of XLRP, an inherited monogenic recessive disorder that causes progressive vision loss in boys and young men. XLRP is predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. AGTC-501 expresses the full length RPGR protein, thereby addressing the full complement of photoreceptor damage caused by XLRP, including both rod and cone loss. Beacon Therapeutics also has access to a target generation technology platform that will identify, screen, and search secreted proteins in the ophthalmology space. The Company is supported by funds from Syncona and additional investors including Oxford Science Enterprises. |
BioCardia Sunnyvale, California | We are a clinical-stage regenerative medicine company developing novel therapeutics for cardiovascular diseases with large unmet medical needs. BioCardia is developing proprietary comprehensive biotherapeutic solutions for cardiovascular disease in its CardiAMP™ and CardiALLO™ therapies. Clinical results supporting both therapeutic programs are compelling and build upon the most rigorous data in the field to date with intramyocardial delivery of marrow-derived cells. These programs are enabled by the Company's Helix™ transendocardial delivery systems and Morph® vascular access products, which are partnered to enable other promising biotherapeutic programs. |
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). |
BioNTech SE Mainz, Germany | BioNTech is a global next generation immunotherapy company aspiring to translate science into survival. Around 6.100 pioneers currently work with us, employing a wide array of computational discovery and therapeutic drug platforms for the rapid development of novel biopharmaceuticals. Our broad portfolio of oncology product candidates includes individualized and off-the-shelf mRNA-based therapies, innovative chimeric antigen receptor (CAR) T cells, several protein-based therapeutics, including bispecific immune checkpoint modulators, targeted cancer antibodies and antibody-drug conjugate (ADC) therapeutics, as well as small molecules. Based on our deep expertise in mRNA vaccine development and in-house manufacturing capabilities, we are developing multiple mRNA vaccine candidates for a range of infectious diseases alongside its diverse oncology pipeline. To learn more about us, please visit www.BioNTech.com. Our privacy statement can be found here: https://www.biontech.com/int/en/home/data-privacy-statement.html Our imprint can be found: https://www.biontech.com/int/en/home/imprint.html |
bluebird bio Somerville, Massachusetts, United States of America | bluebird bio, Inc., a biotechnology company, engages in researching, developing, and commercializing of transformative gene therapies for severe genetic diseases and cancer. Its gene therapy programs include LentiGlobin for the treatment of ß-thalassemia and sickle cell disease; and Lenti-D for the treatment of cerebral adrenoleukodystrophy. The company’s product candidates in oncology include bb2121 and bb21217, which are chimeric antigen receptor T (CAR T) cell product candidates for the treatment of multiple myeloma. It has a strategic collaboration with Bristol-Myers Squibb to discover, develop, and commercialize disease-altering gene therapies in oncology; and Regeneron Pharmaceuticals, Inc. to discover, develop, and commercialize various immune cell therapies for cancer. The company also has collaborations with Medigene AG to discover T cell receptor (TCR) product candidates in the field of cancer; Gritstone Oncology, Inc. to discover TCR product candidates in the field of cancer; TC BioPharm Limited to research and develop gamma delta CAR T cells directed at hematologic and solid tumor targets; Forty Seven, Inc. to pursue clinical proof-of-concept for an antibody-based conditioning regimen; and Novo Nordisk A/S to develop in vivo genome editing treatments for genetic diseases, as well as clinical trial collaboration with Magenta Therapeutics. The company was formerly known as Genetix Pharmaceuticals, Inc., and changed its name to bluebird bio, Inc. in September 2010. bluebird bio, Inc. was founded in 1992 and is headquartered in Cambridge, Massachusetts. |
Brainstorm Cell Therapeutics New York, New York, United States of America | BrainStorm Cell Therapeutics Inc. (NASDAQ) develops autologous stem cell therapies for neurodegenerative disorders such as ALS, MS, and Parkinson’s disease. These diseases have limited treatment options and represent unmet medical needs. NurOwn® is BrainStorm’s proprietary process for differentiating autologous mesenchymal stem cells (MSC) into neurotrophic factor (NTF)-secreting cells, transplanted at or near the affected tissue site. Our technology converts MSCs into a living drug delivery system for NTFs. We have proof-of-concept in various animal models of neurodegenerative diseases, including Parkinson's, Huntington's, ALS, MS, and peripheral nerve injury. We have completed two single-arm clinical trials in ALS patients in Israel, demonstrating good safety and tolerability profiles and strong efficacy signals. We also finalized a Phase 3 randomized, double-blind, placebo-controlled clinical trial at multiple US sites, supported by a $16 million USD grant from the California Institute for Regenerative Medicine (CIRM) and another grant from the ALS Association. Currently, we are planning a Phase 3b clinical trial of NurOwn® in ALS under a Special Protocol Assessment (SPA) with the U.S. Food and Drug Administration (FDA). For more information, visit www.brainstorm-cell.com |
Briacell Therapeutics Philadelphia, Pennsylvania, United States | BriaCell Therapeutics Corp. (Nasdaq: BCTX, BCTXW) (TSX: BCT) is a clinical-stage biotechnology company specializing in targeted immunotherapies for cancer. BriaCell achieved positive proof-of-concept in a Phase I/IIa clinical trial for Bria-IMT™ in patients with advanced breast cancer. BriaCell is conducting a Phase I/IIa clinical trial of Bria-IMT™ in combination with a PD-1 inhibitor, retifanlimab (provided by Incyte under a collaboration agreement). Additionally, BriaCell is developing Bria-OTS™, the first “off-the-shelf” personalized immunotherapy for advanced breast cancer. |
Capricor Therapeutics Beverly Hills, California, United States of America | Capricor Therapeutics, Inc., a biotechnology company, focuses on the development of transformative cell- and exosome-based therapeutics for the treatment and prevention of a broad spectrum of diseases. The company’s lead candidate, CAP-1002, an allogeneic cardiac-derived cell therapy, which has completed phase II clinical trial for the treatment of patients with late-stage Duchenne muscular dystrophy (DMD); CAP-1002, which is in Phase II clinical trial for the treatment of cytokine storm associated with COVID-19; and completed various trials investigating the use of CAP-1002 for the treatment of cardiac conditions, including heart failure and post myocardial infarction with cardiac dysfunction. It is also developing CAP-2003 that is in pre-clinical development for the treatment of trauma related injuries and conditions; and two vaccine candidates, which are in preclinical stage for the potential prevention of COVID-19. Capricor collaborates with Lonza Houston, Inc. for the development of CAP-1002, its cell therapy candidate for the treatment of DMD and other indications. The company was founded in 2005 and is headquartered in Beverly Hills, California. |
Cartesian Therapeutics Gaithersburg, Maryland, United States | Cartesian Therapeutics is a fully integrated, clinical-stage biopharmaceutical company developing novel cell and gene immunotherapies to treat cancer and autoimmune diseases. |
Castle Creek Biosciences, Inc. Exton, Pennsylvania, United States | Castle Creek Biosciences, Inc., a late-stage cell and gene therapy company, is using two lentiviral platforms to develop and commercialize therapies for genetic diseases. |
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. |
Cellphire Therapeutics Rockville, Maryland, United States | Cellphire Therapeutics, Inc. is a Rockville, Maryland-based biotechnology company developing next-generation therapeutics. The company is applying its proprietary cell stabilization technology to all cell types, including platelets, to develop lifesaving products. Its lead product, Thrombosomes®, a freeze-dried hemostatic agent derived from human platelets, is currently in a Phase I clinical trial. Cellphire’s technology has potential applications across a wide range of medical applications from trauma to wound care, imaging, targeted drug delivery, and regenerative medicine. |
CellTrans Chicago, Illinois, United States | CellTrans is pioneering cell therapies for clinical application. |
CG Oncology Irvine, California, United States | CG Oncology, Inc. (Nasdaq: CGON), is a late-stage clinical biopharmaceutical company focused on developing and commercializing a potential backbone bladder-sparing therapeutic for patients afflicted with bladder cancer. We see a world where urologic cancer patients can benefit from our innovative oncolytic immunotherapies to live with dignity and have an enhanced quality of life. Our lead candidate, cretostimogene grenadenorepvec, is an intravesically delivered oncolytic immunotherapy agent in a Phase 3 trial for the treatment of BCG-unresponsive non-muscle invasive bladder cancer. Cretostimogene grenadenorepvec is also in a Phase 2 study in combination with KEYTRUDA® (pembrolizumab) in the same indication. Other types of bladder cancer are being evaluated with cretostimogene grenadenorepvec in combination with OPDIVO® (nivolumab). |
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. |
Cook MyoSite Pittsburgh, Pennsylvania | Cook MyoSite is on a mission to make regenerative medicine a part of everyday medicine. As an industry leader in muscle cell technology, we’re establishing new paradigms for muscle-related disorders. Established in 2002, Cook MyoSite was created to guide the Cook Group organization into the expanding world of cellular technologies. We’re proud to be a part of the vibrant medical community in Pittsburgh, Pennsylvania. |
CorestemChemon Seongnam-si, Gyeonggi-do | CorestemChemon is a global biotechnology corporation centered on stem cell-based new drug development and non-clinical research divisions. Motivated by the urgent need for medication for patients, the bio-pharmaceuticals division remains dedicated to research and development. In 2014, the unit successfully commercialized Neuronata-R inj, a stem cell therapy for the rare disease Amyotrophic Lateral Sclerosis (ALS), making it the fifth stem cell therapy approved for use globally. Alongside ALS treatment, the division has pipelines for various diseases such as lupus, Neuromyelitis Optica (NMO), and multiple system atrophy (MSA). Leveraging the success of the ALS stem cell therapy, the unit plans to concentrate on developing treatments for rare and incurable neurological and autoimmune diseases to dominate new market. |
CRISPR Therapeutics South Boston, Massachusetts, United States of America | CRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. |
DalCor Pharmaceuticals Montreal, Québec, Canada | DalCor Pharmaceuticals: DalCor Pharmaceuticals is pioneering precision medicine for patients with cardiovascular disease. By combining genetic and clinical insights, the company’s development program, dalcetrapib, is intended to reduce fatal and non-fatal myocardial infarction (MI) following a recent acute coronary syndrome and deliver superior cardiovascular outcome in a specific genetic subset of patients. |
Dendreon Seal Beach , CA | Dendreon is a commercial-stage biopharmaceutical company and pioneer in the development of immunotherapy. Dendreon’s flagship product, PROVENGE (sipuleucel-T), was the first FDA-approved immunotherapy made from a patient’s own immune cells. More than 40,000 men with advanced prostate cancer have been prescribed PROVENGE in the U.S. since 2010. Dendreon also is evaluating the use of PROVENGE in early-stage prostate cancer, with the hope of curing more men of the disease. Dendreon is headquartered in Seal Beach, Calif. |
Elpida Therapeutics Los Angeles, California | Elpida Therapeutics is a biopharmaceutical company that focuses on developing treatments for rare diseases, offering hope to children and families affected. |
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. |
Gameto New York, New York, United States | Gameto is a biotechnology company whose mission is to redefine female reproductive health by developing therapies that improve lives. Women’s health is a vastly underserved therapeutic area. Most conditions have few treatment options, and those available often cause frequent complications and offer limited clinical benefit. We aim to change that. Gameto is committed to using cutting-edge scientific advances to deliver better solutions to patients. Using cellular engineering, we have developed a platform to produce female reproductive cell lines. We use proprietary combinations of transcription factors to reprogram hiPSCs into engineered ovarian and endometrium cell lines that mimic the functions of endogenous cells, producing and responding to all hormones. Our team of cellular engineers is using this platform to advance a portfolio of cellular therapeutics for female reproductive diseases. Our first program, Fertilo, is a therapeutic for IVF and egg freezing, aiming to make the process shorter, safer and more effective. Additional applications of our platform include, an organoid of the female reproductive system, Deovo, and a cell-based therapeutic to ameliorate the health consequences of primary ovarian insufficiency and menopause, Ameno. |
GID Bio Louisville, Colorado | GID Group’s SVF-2 device and therapy, pending FDA approval, gives providers and patients a major development in cellular medicine to help treat osteoarthritis pain and to delay or provide an alternative to total knee replacement surgery. GID’s technology harvests, isolate and reimplants the right type and combination of one’s own healing cells. Our first FDA-approved pivotal clinical study, a closely watched rigorous trial, had no serious adverse events and showed significant improvements in pain and function. The FDA has granted conditional approval for a second final pivotal clinical trial. Cellular therapy addresses an unmet need in 14 million people living with osteoarthritis of the knee, offering a less time consuming, painful and costly treatment for everyone that requires no joint surgery and no drugs for osteoarthritis pain. Help relieve the $200 billion burden of osteoarthritis management on our healthcare system. Heal intelligently. |
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. |
Seattle, Washington, United States | Meet your business challenges head on with cloud computing services from Google, including data management, hybrid & multi-cloud, and AI & ML. |
Hansa Biopharma Lund, Sweden | Hansa Biopharma, is a pioneering commercial-stage biopharmaceutical company on a mission to develop and commercialize innovative and life altering treatments for patients with rare immunological conditions. Hansa scientists are working on developing immunomodulatory treatments for organ transplants, rare IgG-mediated autoimmune conditions, and gene therapy, as well as exploring the potential application of the technology platform in oncology. |
Humacyte Durham, North Carolina, United States | Humacyte, Inc., is developing a disruptive biotechnology platform to deliver universally implantable bioengineered human tissues and organs designed to improve the lives of patients and transform the practice of medicine. The Company develops and manufactures acellular tissues to treat a wide range of diseases, injuries and chronic conditions. Humacyte’s initial opportunity, a portfolio of human acellular vessels (HAVs), is currently in late-stage clinical trials targeting multiple vascular applications, including vascular trauma repair, arteriovenous access for hemodialysis, and peripheral arterial disease. Pre-clinical development is also underway in coronary artery bypass grafts, pediatric heart surgery, treatment of type 1 diabetes, and multiple novel cell and tissue applications. Humacyte’s HAVs were the first product to receive the FDA’s Regenerative Medicine Advanced Therapy (RMAT) expedited review designation and received priority designation for the treatment of vascular trauma by the U.S. Secretary of Defense. |
Immatics Tubingen, Germany | Immatics N.V., a clinical-stage biopharmaceutical company, focuses on the discovery and development of T cell receptor (TCR) based immunotherapies for the treatment of cancer in the United States. The company is developing targeted immunotherapies with a focus on treating solid tumors through two distinct therapeutic modalities, such as adoptive cell therapies (ACT) and antibody-like TCR Bispecifics. Its ACTengine product candidates include IMA201 that targets melanoma-associated antigen 4 or 8 in patients with solid tumors; IMA202 that targets melanoma-associated antigen 1 in patients with various solid tumors, including squamous non-small cell lung carcinoma and hepatocellular carcinoma; IMA203 that targets preferentially expressed antigen in melanoma in adult patients with relapsed and/or refractory solid tumors; and IMA204, an anti-tumor therapy that targets the malignant tumor cell. Its TCR Bispecifics product candidates include IMA401, a cancer testis antigen for the treatment of solid tumor; and IMA402 for the treatment of solid and hematological malignancies. The company also develops IMA101, a multi-target precision immunotherapy; and IMA301, an off-the-shelf ACT. It has a strategic collaboration agreement with GlaxoSmithKline plc to develop novel adoptive cell therapies targeting multiple cancer indications; MD Anderson Cancer Center to develop multiple T cell and TCR-based adoptive cellular therapies; Celgene Switzerland LLC to develop novel adoptive cell therapies targeting multiple cancers; Genmab A/S to develop T cell engaging bispecific immunotherapies targeting multiple cancer indications; Amgen Inc.; and MorphoSys to develop novel antibody-based therapies against various cancer antigens that are recognized by T cells. Immatics N.V. is headquartered in Tubingen, Germany. |
ImmunityBio San Diego, California, United States | ImmunityBio (previously NantKwest (NASDAQ: NK)) is a pioneering, next generation, clinical-stage immunotherapy company focused on harnessing the unique power of our immune system using natural killer (NK) cells to treat cancer, infectious diseases and inflammatory diseases. NK cells are the body’s first line of defense due to the innate ability of NK cells to rapidly identify and destroy cells under stress, such as cancer or virally-infected cells. ImmunityBio is uniquely positioned to implement precision cancer medicine, with the potential to change the current paradigm of cancer care. Natural Killer cells are ancient cells in the human body designed to recognize and detect cells under stress or infected. Our “off-the-shelf” activated Natural Killer (NK) platform have the capacity to destroy cancer and virally infected cells from the body. The safety of our NK cells as well as their activity against a broad range of cancers have been tested in multiple phase 1 clinical trials in the United States, Canada and Europe. In addition to our NK cells capability to be administered in the outpatient setting as an “off-the-shelf” living drug, it serves as a universal cell-based therapy without need for individualized patient matching. Moreover, our NK cell based platform has been bioengineered to incorporate chimeric antigen receptors (CARs) and antibody receptors to further optimize targeting and potency in the therapeutic disease. Our targeted therapeutic areas include: (1) cancer, focusing on bulky hematological cancers and solid tumors as well as cancer stem cells, (2) infectious diseases, including viral, fungal and bacterial infections, and (3) inflammatory diseases, ranging from rare inherited diseases to more prevalent autoimmune disorders. |
Immutep Sydney, New South Wales, Australia | Immutep is a globally active biotechnology company that is a leader in the development of LAG-3 related immunotherapeutic products for the treatment of cancer and autoimmune disease. Immutep is dedicated to leveraging its technology and expertise to bring innovative treatment options to market for patients and to maximize value to shareholders. |
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. |
Innate Pharma SA Marseille, France | Innate Pharma S.A. is a commercial stage oncology-focused biotech company dedicated to improving treatment and clinical outcomes for patients through therapeutic antibodies that harness the immune system to fight cancer. Innate Pharma’s commercial-stage product, Lumoxiti, in-licensed from AstraZeneca, was approved by the FDA in September 2018. Lumoxiti is a first-in class specialty oncology product for hairy cell leukemia (HCL). Innate Pharma’s broad pipeline of antibodies includes several potentially first-in-class clinical and preclinical candidates in cancers with high unmet medical need. Pioneers in the biology of Natural Killer cell, Innate Pharma has expanded its expertise in the tumor microenvironment and tumor-antigens, as well as antibody engineering. This innovative approach has resulted in a diversified proprietary portfolio and major alliances with leaders in the biopharmaceutical industry including Bristol-Myers Squibb, Novo Nordisk A/S, Sanofi, and a multi-products collaboration with AstraZeneca. |
Innovacell GmbH Innsbruck, Austria | Innovacell Biotechnologie AG is an innovative biotechnology company that develops personalized cell therapies for treatment of incontinence. |
Institut Pasteur Paris, FR | The Institut Pasteur, a non-profit foundation with recognized charitable status set up by Louis Pasteur in 1887, is today an internationally renowned center for biomedical research with a network of 33 members worldwide. In the pursuit of its mission to tackle diseases in France and throughout the world, the Institut Pasteur operates in four main areas: research, public health, training, and development of research applications. The Institut Pasteur is a globally recognized leader in infectious diseases, microbiology, and immunology, with research focusing on the biology of living systems. Among its areas of investigation are emerging infectious diseases, antimicrobial resistance, certain cancers and brain connectivity diseases. The Institut Pasteur's outstanding research is facilitated by the development of a technological environment of the highest standard, with core facilities for nanoimaging, computational biology and artificial intelligence. Since its inception, 10 Institut Pasteur scientists have been awarded the Nobel Prize for Medicine, including two in 2008 for the 1983 discovery of the human immunodeficiency virus (HIV) that causes AIDS. www.pasteur.fr/en |
Intellia Therapeutics Cambridge, Massachusetts, United States of America | Intellia Therapeutics is a leading genome editing company whose mission is to develop potentially curative gene editing treatments that can positively transform the lives of people living with severe and life-threatening diseases. We are focused on the development of proprietary therapeutics using a recently developed biological tool known as the CRISPR/Cas9 system. The promise of the CRISPR/Cas9 system is the driving force behind the creation of Intellia. Our founders have a shared belief that the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course. This technology offers the potential for us to develop curative therapeutic options for patients with chronic diseases by addressing the underlying cause of the disease. It can bring new hope and cures to people who now have nowhere to turn for help. Our combination of deep scientific expertise and clinical development experience, along with our leading intellectual property portfolio, puts Intellia in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Intellia has the right people, assets and ambitious vision needed to take full advantage of the CRISPR/Cas9 technology’s attributes – high potency, specificity, simplicity of use, broad applicability, and multifunctional programmability – and accelerate the advancement of curative products into the clinic. View our social media community guidelines at https://www.intelliatx.com/terms-and-conditions/ . |
Iovance Biotherapeutics San Carlos, California, United States, Philadelphia, Pennsylvania, Tampa, Florida | Iovance Biotherapeutics (NASDAQ: IOVA) aims to be the global leader in innovating, developing and delivering tumor infiltrating lymphocyte (TIL) therapies for patients with cancer. We are pioneering a transformational approach to cure cancer by harnessing the human immune system’s ability to recognize and destroy diverse cancer cells in each patient. The Iovance TIL platform has demonstrated promising clinical data across multiple solid tumors. We are committed to continuous innovation in cell therapy, including gene-edited cell therapy, that may extend and improve life for patients with cancer. We are adding to our talented team as we execute our commercial launch, advance our research and development efforts, and build our internal manufacturing capabilities. Iovance is headquartered in the San Francisco Bay Area. Our Iovance Cell Therapy Center (iCTC), a state-of-the-art manufacturing facility, is located in Philadelphia. We also have a research site in Tampa, Florida. Any information expressed by Iovance is subject to the risk factors and information on forward-looking statements contained in its filings with the Securities and Exchange Commission and available here: https://ir.iovance.com/sec-filings |
Kite Pharma Santa Monica, California, United States; El Segundo, Oceanside, Foster City, Frederick, Maryland, Philadelphia, Pennsylvania, London, United Kingdom, England, Netherlands | At Kite, a Gilead Company, our singular focus is cell therapy to treat and potentially cure cancer. Our goal is to bring the promise of cell therapy to as many eligible patients as possible who may benefit. Since 2009, Kite has been at the forefront of cancer immunotherapy. Today, we are proud to be the leader in engineered T cell therapy for hematologic malignancies, but we recognize that this is just the beginning. Patients are at the heart of our focus on the cure, which is why we are committed to pursuing cell therapy as a transformative treatment option for as many people with cancer as possible who may benefit. Cancer is personal to us. Unlike traditional pharmaceutical manufacturing, our therapies are not created on an assembly line. Cell therapy specialists at our manufacturing facilities reengineer a patient’s own immune cells to attack and fight their cancer cells — one patient at a time. That sense of connection to the people we serve motivates us daily at Kite. Our corporate headquarters are located in Santa Monica, California, and we have dedicated cell therapy manufacturing facilities in California, Maryland and the Netherlands. If you’re passionate about changing the future of cancer treatment, let’s talk. |
Krystal Biotech Pittsburgh, Pennsylvania, United States | Krystal Biotech, Inc. is using gene therapy to develop effective and novel treatments for skin diseases. Our goal is to make a meaningful difference in the lives of underserved patient populations with debilitating skin diseases. We work to accomplish that through scientific innovation, operational excellence and believe that “nature operates in the shortest way possible”. (Aristotle) |
MaaT Pharma Lyon, France | MaaT Pharma has established the most complete approach to restoring patient-microbiome symbiosis to improve survival outcomes in life-threatening diseases. Committed to treating blood cancers and graft-versus-host disease, a serious complication of allogeneic stem cell transplantation, MaaT Pharma has already achieved proof of concept in acute myeloid leukemia patients. Supporting the further expansion of our pipeline into larger indications, we have built a powerful discovery and analysis platform to evaluate drug candidates, determine novel disease targets and identify biomarkers for microbiome-related conditions. Our therapeutics are produced through a standardized cGMP manufacturing and quality control process to safely deliver the full diversity of the microbiome. MaaT Pharma benefits from the commitment of world-leading scientists and established relationships with regulators to spear-head microbiome treatment integration into clinical practice. |
Mesoblast Melbourne, Victoria, Australia | Mesoblast (ASX:MSB; Nasdaq:MESO) is developing and commercializing allogeneic cellular medicines to treat serious and life-threatening inflammatory diseases with significant, unmet medical needs. The Company's Phase 3 off-the-shelf mesenchymal lineage cell product candidates are: • RYONCIL™ (remestemcel-L) for steroid-refractory acute graft versus host disease (acute GVHD) • Remestemcel-L for moderate to severe acute respiratory distress syndrome (ARDS) due to COVID-19 infection • REVASCOR® for advanced chronic heart failure, and • MPC-06-ID for chronic low back pain due to degenerative disc disease. The Company’s proprietary manufacturing processes yield industrial-scale, cryopreserved, off-the-shelf, cellular medicines. These cell therapies, with defined pharmaceutical release criteria, are planned to be readily available to patients worldwide. Mesoblast’s approach to product development is to ensure rigorous scientific investigations are performed with well-characterized cell populations in order to understand mechanisms of action for each potential indication. Extensive preclinical translational studies guide clinical trials that are structured to meet stringent safety and efficacy criteria set by international regulatory agencies. All trials are conducted under the continuing review of independent Data Safety Monitoring Boards comprised of independent medical experts and statisticians. Mesoblast has an extensive patent portfolio comprising approximately 1,000 patents and patent applications with protection extending through 2040 in all major markets. This intellectual property portfolio covers composition of matter, manufacturing, and therapeutic applications of mesenchymal lineage cells. The Company believes this patent estate provides strong global protection. |
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. |
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. |
Ocugen Malvern, Pennsylvania, United States of America | Ocugen, Inc. is a biopharmaceutical company focused on discovering, developing, and commercializing gene therapies to cure blindness diseases and developing a vaccine to save lives from COVID-19. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with one drug – “one to many” and our novel biologic product candidate aims to offer better therapy to patients with underserved diseases such as wet age-related macular degeneration, diabetic macular edema, and diabetic retinopathy. We are co-developing Bharat Biotech’s COVAXIN™ vaccine candidate for COVID-19 in the U.S. market. |
Ocuphire Pharma 37000 grand river avenue, farmington hills, mi, united states | Ocuphire Pharma Inc is a clinical-stage ophthalmic biopharmaceutical company focused on developing and commercializing innovative small molecule therapies for eye disorders. The company aims to address unmet medical needs in the ocular space, particularly in retinal and refractive disorders. Ocuphire Pharma offers research and development services to create novel treatments for various eye conditions. The company collaborates with pharmaceutical partners to advance its drug candidates through clinical trials and commercialization. Its product pipeline includes Nyxol, an ophthalmic solution for dim light and night vision disturbances; APX3330, an oral inhibitor for diabetic retinopathy and macular edema; APX2009, aimed at treating wet age-related macular degeneration; and Phentolamine Ophthalmic Solution (RYZUMVI), approved for pharmacologically induced mydriasis and under development for other conditions. |
Orca Bio Menlo Park, California, United States | Orca Bio is a clinical-stage biotechnology company developing a pipeline of high precision allogeneic cell therapy products that are designed to safely replace patients’ diseased blood and immune system with a healthy one. Our manufacturing platform sorts donor blood with single-cell precision and a high level of purity and speed, enabling us to create proprietary, optimal therapeutic mixtures of immune and stem cells that have the potential to transform allogeneic cell therapy. Our talented team is driven by a passion for science and compassion for patients. |
Orchard Therapeutics London, England, United Kingdom | Orchard Therapeutics, a Kyowa Kirin company, is a global gene therapy leader focused on ending the devastation caused by genetic and other severe diseases by discovering, developing, and commercializing new treatments that tap into the curative potential of hematopoietic stem cell (HSC) gene therapy. In this approach, a patient’s own blood stem cells are genetically modified outside of the body and then reinserted, with the goal of correcting the underlying cause of disease with a single treatment. Founded in 2015, Orchard’s roots go back to some of the first research and clinical developments involving HSC gene therapy. Our team has played a central role in the evolution of this technology from a promising scientific idea to a potentially life-transforming reality. Today, Orchard is advancing a pipeline of HSC gene therapies designed to address serious diseases where the burden is immense for patients, families and society and current treatment options are limited or do not exist. For more information, please visit www.orchard-tx.com. |
Organogenesis Canton, Massachusetts, United States | Organogenesis™ is a leading regenerative medicine company focused on the development, manufacture, and commercialization of solutions for the advanced wound care and surgical and sports medicine markets. Organogenesis offers a comprehensive portfolio of innovative regenerative products to address patient needs across the continuum of care. |
OSE Immunotherapeutics Nantes, France | OSE Immunotherapeutics is an integrated biotech company focused on developing first-in-class assets targeting cancer and inflammatory diseases. A current well-balanced first-in-class clinical pipeline including: • Tedopi® (T-cell specific immunotherapy): Company’s most advanced product; positive results for Ph 3 in NSCLC in secondary resistance after CKI failure. Other sponsored combo Ph 2 trials in solid tumors ongoing. • OSE-279 (anti-PD1) advanced preclinical stage • OSE-127 (humanized mAb antagonist of IL-7R); ongoing Ph 2 in Ulcerative Colitis; ongoing preclinical research in leukemia • VEL-101/FR104 (anti-CD28 mAb) developed in partnership with Veloxis in transplantation; ongoing Ph 1/2 in renal transplant (sponsor Nantes University Hospital); Ph 1 ongoing in the US (sponsor Veloxis). • BI 765063 (anti-SIRPα mAb on CD47/SIRPα pathway) developed in partnership with Boehringer Ingelheim in advanced solid tumors; positive Ph 1 dose escalation results in mono and in combo, in particular with anti-PD-1 antibody ezabenlimab; BI sponsored international Ph 1b clinical trial ongoing in combo with ezabenlimab alone or with other drugs in recurrent/metastatic HNSCC and HCC. OSE Immunotherapeutics expects to generate further significant value from its two proprietary drug discovery platforms, which are central to its ambitious goal to deliver next-generation first-in-class immunotherapeutics: • BiCKI® platform focused on immuno-oncology (IO) is a bispecific fusion protein platform built on the key backbone component of anti-PD1 combined with a new immunotherapy target to increase anti-tumor efficacy. Most advanced BiCKI® candidate is targeting anti-PD1xIL-7. • Myeloid platform focused on optimizing the therapeutic potential of myeloid cells in IO & immuno-inflammation (I&I). OSE-230 (ChemR23 agonist mAb), most advanced candidate generated by the platform, with the potential to resolve chronic inflammation. |
Pacira BioSciences Tampa, Florida, United States of America | Pacira BioSciences Inc. is a leading provider of non-opioid pain management and regenerative health solutions. We are dedicated to offering patients—and the clinicians who treat them—cutting-edge therapies to treat pain with the potential to reduce or eliminate the need for opioids. Founded in 2006 and headquartered in Tampa, FL, Pacira has offices in Parsippany, NJ as well as San Diego, CA. With more than 700 employees who share a passion for delivering improved patient care and reducing the need for opioids, Pacira is here to challenge and transform the way we think about – and treat – pain. We are committed to providing solutions that address unmet medical need and improve clinical results, and empowering patients and healthcare providers to choose non-opioid options. Our non-opioid product portfolio is aimed at opioid minimization, long-lasting pain control, and enhanced recovery. Find out why we aim for change at Pacira.com/commitments |
Paracrine San Diego, California, United States | Paracrine is a San Diego-based company in late-stage clinical development of an Immuno-NeuroVascular Regenerative Therapy - a multi-dimensional biologic therapy for complex diseases. Our primary focus is an FDA approved Phase III Trial in chronic diabetic foot ulcers where feasibility data from four prior studies demonstrated twice the healing rate in less than half the time and no recurrence out to 18 monhts. |
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. |
Precigen Germantown, Maryland, United States of America | Precigen (Nasdaq: PGEN) is a dedicated discovery and clinical stage biopharmaceutical company advancing the next generation of gene and cell therapies using precision technology to target the most urgent and intractable diseases in immuno-oncology, autoimmune disorders, and infectious diseases. Our technologies enable us to find innovative solutions for affordable biotherapeutics in a controlled manner. Precigen operates as an innovation engine progressing a preclinical and clinical pipeline of well-differentiated unique therapies toward clinical proof-of-concept and commercialization. |
Priothera Saint Louis, France | Priothera - We are dedicated to improving the lives of patients suffering from hematological malignancies by delivering an innovative immune modulator to enhance the curative potential of allogeneic Hematopoietic Stem Cell Transplantation (HSCT) |
ProKidney Winston-Salem, North Carolina, United States of America | ProKidney Corp., a clinical-stage biotechnology, engages in developing cellular therapy candidates. It is developing Renal Autologous Cell Therapy, an autologous homologous cell admixture that is in a Phase III development program, as well as Phase II clinical trials for the treatment of moderate to severe diabetic kidney disease; and Phase I clinical trial for patients with congenital anomalies of the kidney and urinary tract. The company was founded in 2015 and is headquartered in Winston-Salem, North Carolina. |
Protara Therapeutics New York, New York, United States | Protara Therapeutics, Inc., a clinical-stage company, engages in the identifying and advancing transformative therapies for people with rare and specialty diseases. The company's lead program is TARA-002, an investigational cell therapy for the treatment of lymphatic malformations. It also develops intravenous choline chloride, an investigational phospholipid substrate replacement therapy for the treatment of intestinal failure associated liver disease. The company was formerly known as ArTara Therapeutics, Inc. and changed its name to Protara Therapeutics, Inc. in May 2020. Protara Therapeutics, Inc. is based in New York, New York. |
PTC Therapeutics South Plainfield, New Jersey, United States of America | PTC Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of medicines for the treatment of rare disorders. The company offers Translarna (ataluren) and Emflaza (deflazacort) for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients. It is developing Translarna, which is in Phase 2 clinical study for the treatment of nonsense mutation aniridia and nonsense mutation Dravet syndrome/CDKL5; and RG7916 and RO7034067 for the treatment of patients with spinal muscular atrophy, as well as PTC596 and PTC299, a small molecule dihydrooratate dehydrogenase (DHODH) inhibitor that inhibits de novo pyrimidine nucleotide synthesis, which is in Phase 1 clinical development stage to treat cancer patients. The company is also developing gene therapy product candidate that include PTC-AADC for the treatment of Aromatic L-amino acid decarboxylase deficiency. It has collaborations with F. Hoffman-La Roche Ltd and Hoffman-La Roche Inc., and the Spinal Muscular Atrophy Foundation to develop and commercialize compounds identified under its spinal muscular atrophy sponsored research program; research collaboration with Massachusetts General Hospital for the treatment of rare genetic disorders resulting from pre-mRNA; and Akcea Therapeutics, Inc. to commercialize Tegsedi and Waylivra for the treatment of rare diseases in Latin America and the Caribbean. PTC Therapeutics, Inc. was founded in 1998 and is headquartered in South Plainfield, New Jersey. |
Replimune Group Inc Woburn, Massachusetts, United States of America | Replimune Group, Inc., a biotechnology company, develops oncolytic immune-gene therapies to treat cancer. It uses its proprietary Immulytic platform to design and develop product candidates that are intended to activate the immune system against cancer. The company's lead product candidate is RP1, a selectively replicating version of herpes simplex virus 1, which is in Phase I/II clinical trials for a range of solid tumors; and that is in Phase II clinical trials for patients with cutaneous squamous cell carcinoma. It is also developing RP2, which is in Phase I clinical trials for an anti-CTLA-4 antibody-like protein in order to block the inhibition of the immune response otherwise caused by CTLA-4; and RP3 to express immune-activating proteins that stimulate T cells. Replimune Group, Inc. was founded in 2015 and is headquartered in Woburn, Massachusetts. |
Resilience San Diego, California, US | |
Sanguine Bioscience Woburn, Massachusetts, US | |
Sarepta Therapeutics Cambridge, Massachusetts, United States | Sarepta Therapeutics, headquartered in Cambridge, Massachusetts, is a global biotechnology company on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development. Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing. For information on our Community Guidelines, please visit sarepta.com/community-guidelines. We want to share a reminder with all job seekers and candidates regarding the persistence of recruiting fraud. Please read a message about recruiting fraud and steps you can take to protect yourself here: https://www.sarepta.com/recruiting-fraud |
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. |
TG Therapeutics New York, New York, United States | TG Therapeutics is a biopharmaceutical company focused on the acquisition, development and commercialization of novel treatments for B-cell diseases. In addition to a research pipeline including several investigational medicines, TG has completed a Phase 3 program for ublituximab, an investigational glycoengineered monoclonal antibody that targets a unique epitope on CD20-expressing B-cells, to treat patients with relapsing forms of multiple sclerosis (RMS). |
Thermo Fisher Scientific Waltham, Massachusetts, US | Thermo Fisher Scientific Inc. is the world leader in serving science, with annual revenue of approximately $40 billion. Our Mission is to enable our customers to make the world healthier, cleaner and safer. Whether our customers are accelerating life sciences research, solving complex analytical challenges, increasing productivity in their laboratories, improving patient health through diagnostics or the development and manufacture of life-changing therapies, we are here to support them. Our global team delivers an unrivaled combination of innovative technologies, purchasing convenience and pharmaceutical services through our industry-leading brands, including Thermo Scientific, Applied Biosystems, Invitrogen, Fisher Scientific, Unity Lab Services, Patheon and PPD. |
TVAX Biomedical Lenexa, KS | TVAX Biomedical is a biotechnology company currently performing phase 2b/3 clinical trials to test a unique cancer immunotherapy as a treatment for pediatric brain cancer. |
Ultragenyx Pharmaceutical Inc. Allschwil Basel-Country, Switzerland | Leading with purpose. Every day. “Ultragenyx was founded to advance innovative medicines for rare and ultrarare diseases that have never been treated before. We are delivering transformative therapies across multiple indications, and we have one of the most robust and diverse clinical pipelines in rare disease. Our focus is on doing the right things for patients both during development and commercialization to deliver on the promise of these therapies in a way that's meaningful for rare disease communities.” Emil D. Kakkis, M.D., Ph.D. Chief Executive Officer and President We have an inclusive culture of value and respect. Ultragenyx brings novel products to those living with rare and ultrarare diseases with a focus on debilitating genetic diseases. Founded in 2010, we have rapidly built a diverse portfolio of approved therapies and products aimed at diseases with a high unmet medical need for which many have no approved therapies. It takes courage, care, talent, and dedication to make a meaningful impact for those living with rare diseases, their families and the rare disease community. We put our people first, so they can take care of the rare disease community. We purposefully cultivate and nurture a dynamic, supportive work environment where we encourage every team member to share their new ideas and help us unlock more possibilities. Our team is made up of exceptional and diverse people with a strong passion and commitment to helping those living with rare diseases. By embracing generosity, curiosity, inclusion, and humility we are constantly learning together—fostering an environment that supports profound growth and fulfillment. If you want to have a meaningful impact, do the best work of your career while having fun and growing professionally and personally, come join our team! Visit our community guidelines at: https://ultragenyx.co/community |
Vera Therapeutics South San Francisco, California, United States | Vera Therapeutics, Inc., a clinical stage biotechnology company, focuses on developing and commercializing treatments for patients with serious immunological diseases in the United States. Its lead product candidate is atacicept, a fusion protein self-administered as a subcutaneous injection that is in Phase IIb clinical trial for patients with immunoglobulin A nephropathy. The company was formerly known as Trucode Gene Repair, Inc. and changed its name to Vera Therapeutics, Inc. in April 2020. Vera Therapeutics, Inc. was incorporated in 2016 and is headquartered in South San Francisco, California. |
Vericel Cambridge, Massachusetts, United States | Vericel is a leading provider of advanced therapies for the sports medicine and severe burn care markets. The Company combines innovations in biology with medical technologies, resulting in a highly differentiated portfolio of innovative cell therapies and specialty biologics that repair injuries and restore lives. We currently market two autologous cell therapy products and one specialty biologic product in the United States. Personal integrity, teamwork, collaboration, and innovative technology are the foundations of our work. We seek to practice transparency in our clinical trials and research, and in our relationships with each other, our patients, and the investors who support us. PRIVACY AND COMMUNITY GUIDELINES When you engage with Vericel social media channels and our content providers on social media, you also agree to abide by the following Community Guidelines (found here: https://bit.ly/3pg3UKQ), which apply in addition to the Terms and Conditions or other legal notices specific to each social media channel. Please note that third-party social media channels also have access to information you share via social media. For more details, read LinkedIn's Privacy Policy here: https://bit.ly/3GF2r5Y. For additional information about privacy at Vericel, please see our Privacy Policy (bit.ly/43ErZcx) and Terms and Conditions of Use (bit.ly/4568Cdx). If you engage with us, you consent and give Vericel permission to communicate with you and to use any information, text and media associated with the interaction or post. |
Vir Biotechnology San Francisco, California, United States | Vir Biotechnology, Inc. (in Europe, Humabs BioMed, https://bit.ly/46lQGN2) is an immunology company focused on powering the immune system to address unmet patient needs in infectious disease and beyond. Our powerful R&D engine combines our proprietary monoclonal antibody (mAb) platform with machine learning and artificial intelligence-enhanced capabilities, enabling us to engineer our rich database of human antibodies and deliver transformative medicines for diseases with significant global burden. Our current pipeline consists of multiple clinical candidates targeting hepatitis B and hepatitis delta viruses and human immunodeficiency virus, including a potential functional cure for chronic hepatitis B and treatment for chronic hepatitis delta. Our portfolio of broad, potent investigational monoclonal antibodies also targets influenza A and B viruses, COVID-19, respiratory syncytial virus/human metapneumovirus and human papillomavirus, and our investigational T cell vaccine aims to prevent HIV. Explore our full pipeline: https://bit.ly/3tr7FPm We collaborate with some of the world’s leaders in industry as well as government agencies and non-profit organizations enabling us to rapidly advance cutting-edge scientific discoveries and innovative technologies with the potential to benefit people around the world. We are proud to have been ranked the fastest-growing company in North America on the 2022 Deloitte Technology Fast 500™. Join us as we pursue our mission to transform lives. It has come to our attention that there have been various recruitment scams targeting job seekers on LinkedIn. Vir Biotechnology, Inc. is committed to your privacy and will never request sensitive personal information including banking details through email, text message, social media or video software platforms. We also will never request a financial commitment from a candidate as part of the application process. Learn more about Vir’s recruiting process: https://bit.ly/461EgJN |
VISEN Pharmaceuticals Shanghai | VISEN is an innovative biopharmaceutical company focused on endocrine diseases. We are dedicated to providing innovative therapies and compassionate, patient-centric care, because we believe that achieving better treatment processes and outcomes results in living better lives. Putting patients’ need first, VISEN is committed to providing first-in-class or best-in-class products and treatments for endocrine diseases. Our therapeutic areas cover endocrine diseases in adults and children, and rare endocrine diseases. VISEN comprises seasoned professionals with multinational pharmaceutical experiences and leverages cutting-edge technologies and leading resources across the world. We are focused on the Chinese market, and have established offices in Shanghai, Beijing, Hong Kong and Taipei. We have also launched our Greater China R&D and manufacturing site in Suzhou to enhance R&D, manufacturing and commercialization efforts. Our goal is to enable Chinese endocrine patients to benefit from the world's most advanced and reliable treatment solutions earlier. In 2018, VISEN Pharmaceuticals (VISEN) was formed by Ascendis Pharma A/S (Nasdaq: ASND) and an investor syndicate led by Vivo Capital (along with participation by Sofinnova Ventures), to develop and commercialize Ascendis Pharma’s endocrinology therapies in Greater China, which includes mainland China, Hong Kong, Macau, and Taiwan. In Jan 2021, VISEN had closed a $150 million Series B financing led by Sequoia China with participation from OrbiMed, Sherpa Healthcare Partners, Cormorant, HBM Healthcare Investments, Pivotal bioVenture Partners China, Logos Capital, and CDG Capital, as well as all of the existing investors, including Ascendis Pharma A/S, Vivo Capital and Sofinnova Investments. |