List of Gene Therapy Companies with Phase 2 Active Clinical Trial - 153

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 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.

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.

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

Adrenas Therapeutics is a company that was created with a single mission: to work with scientists, physicians, and patients in developing a gene therapy for people affected by CAH. Adrenas is a subsidiary of BridgeBio, a public company dedicated to finding, developing, and delivering breakthrough medicines for genetic diseases to patients as quickly and safely as possible.

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 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 (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, 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.

Altheia Science is a pioneering start-up founded by two world renowned Italian scientists, Prof. Alessandra Biffi, MD and Prof. Paolo Fiorina, MD, PhD, together with AurorA-TT. Altheia Science’s pipeline exploits pioneering therapeutic tools for autoimmune diseases and cancer. The modulation of PD-L1 expression at the molecular and protein level is key to devise advanced treatments. This can be achieved by first-in-class molecules controlling the PD-L1 pathway and/or by lentiviral vector-based engineering of patients’ hematopoietic stem cells. Altheia Science’s approach intends to drastically modify the natural history of diseases with high clinical impact in autoimmunity and cancer by modulating PD-L1 expression, achieving tangible and durable benefit for each patient.

Alto Neuroscience is a clinical-stage biopharmaceutical company that integrates the biology of the patient into drug development to improve the lives of people with mental health conditions. Through its AI-enabled biomarker platform, Alto Neuroscience combines rich sources of information on patients’ brain activity and behavior to rapidly develop

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 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.

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.

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.

Ascidian Therapeutics is redefining the treatment of disease by rewriting RNA. By editing exons at the RNA level, Ascidian therapies enable precise post-transcriptional editing of genes, resulting in full-length, functional proteins at the right levels, in the right cells, at the right time. With active discovery and preclinical programs in ophthalmology, neurological and neuromuscular disorders, and other rare diseases, Ascidian is opening new therapeutic possibilities for patients in need of breakthroughs.

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

Aspa Therapeutics, a subsidiary of BridgeBio Pharma, is a biotechnology company focused on developing a treatment for Canavan disease, a devastating inherited condition without an FDA-approved therapy. Aspa is led by a team of veteran biotechnology executives. Together with patients and physicians, the company aims to bring a safe and effective treatment for Canavan disease to patients as quickly as possible.

Atamyo Therapeutics is focused on the development of a new generation of safe and effective gene therapies for neuromuscular diseases. A spin-off of gene therapy pioneer Genethon, Atamyo leverages unique expertise in AAV-based gene therapy and muscular dystrophies from the Progressive Muscular Dystrophies Laboratory at Genethon. Atamyo’s most advanced programs address different forms of limb-girdle muscular dystrophies (LGMD).

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 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 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.

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.

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.

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 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.

Founded in 2018, Belief BioMed is aiming to become a globally leading gene therapy company by being committed to providing innovative therapies with improved efficacy for monogenic disorder diseases, age-related degenerative diseases, and certain malignant diseases through its AAV vector technology from early discovery to commercialization. The R&D and production strengths of Belief BioMed have been recognized by top investment institutions and enterprises. Belief BioMed has offices, research centers and manufacturing facilities in Shanghai, Hong Kong, Beijing and Suzhou China and North Carolina US.

Benitec is a biotechnology company developing a proprietary therapeutic technology platform that combines RNA interference (RNAi) with gene therapy with a goal of providing sustained, long-lasting silencing of disease-causing genes from a single administration. Benitec is using its technology, called DNA-directed RNA interference (ddRNAi) to develop a pipeline of product candidates for the treatment of several chronic and life-threatening human diseases , including oculopharyngeal muscular dystrophy (OPMD) and hepatitis B. By combining the specificity and gene silencing effect of RNAi with gene therapy, ddRNAi has the potential to produce long-lasting silencing of disease-causing genes from a single administration, which could minimize the requirement for patients to take regular doses of medicine. Benitec’s objective is to become the leader in discovering, developing, clinically validating and commercializing ddRNAi-based therapeutics for a range of human diseases with high unmet clinical need or large patient populations and, as a result, provide a better life for patients with these diseases.

Bio-Path is a biotechnology company developing targeted therapies for acute myeloid leukemia (AML), lymphoma, chronic lymphocytic leukemia, gynecological cancer and other challenging cancers. The company’s lead product candidate, prexigebersen (Liposomal Grb2 Antisense), formerly BP1001, is currently being assessed in a Phase 2 clinical study in previously untreated AML patients, who are not eligible for or who have decided to forego intensive chemotherapy because of their fragile health, and in refractory/relapsed AML patients. Additionally, BP1001-A is being assessed in a Phase 1 study in refractory/relapsed patients with solid tumors. The Company’s second product BP1002 (Liposomal Bcl-2 Antisense) is being evaluated in Phase 1 trials in patients with refractory/relapsed lymphoma and AML.

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 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.

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.

CatalYm is a Germany-based biotechnology company that researches and develops gene therapies for the treatment of cancer.

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.

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 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 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 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., 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.

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.

Dogwood is pioneering Halneuron®, a highly selective sodium channel blocker being developed to treat moderate-to-severe chemotherapy induced neuropathic pain (CINP), cancer pain, and other pain indications. It is also developing the antiviral combination therapies IMC-1 and IMC-2 to treat diseases associated with a viral triggered abnormal immune response such as Long-COVID (“LC”) and fibromyalgia (“FM”)

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.

EdiGene is a clinical-stage company focused on translating gene-editing technologies into transformative genetic medicines for patients with significant unmet medical needs. Anchored by its proprietary technologies of gene editing, bioinformatics, and high throughput genome-editing screening, EdiGene advances the pipeline on therapeutic platforms of LEAPER-based in vivo RNA base editing, ex vivo gene-editing hematopoietic stem cells, and ex vivo gene-editing T cells for allogeneic CAR-T.

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.

At EG427 our unique, non-replicative Herpes Simplex Virus type 1 (HSV-1) based vector platform delivers, with pinpoint precision, highly selective, durable expression of disease modifying transgenes for use in the treatment of peripheral nervous system disorders and beyond. Our lead asset EG110A, for the treatment of neurogenic bladder dysfunction, is expected to be in the clinic by early 2024. Our revolutionary approach to the treatment of this disease aims at providing patients with a comprehensive, long-term solution to their bladder management, through a highly selective molecular biology approach overcoming the drawbacks of current standard of care.

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.

enGene Inc. has developed a highly flexible nucleotide (DNA and RNAi) delivery technology that targetsmucosal tissues and can be used to treat numerous prevalent, chronic diseases via the induction or suppression of protein expression levels. Our platform technology has the ability to significantly impact diseases of mucosal tissues such as the gastrointestinal tract, lung and bladder as well as provide systemic release of proteins from the gut to treat diabetes, anemia, hemophilia and others.

Epic Bio is a biotechnology company that focuses on biomedical research and genetics.

We are ESTEVE, a global pharmaceutical company with one defined mission: improving lives. Founded in 1929 and headquartered in Barcelona, we have a strong international footprint, with affiliates across Spain, Portugal, Italy, Germany, France, the UK, and the USA. As a leader in speciality pharmaceuticals, we focus on creating highly specialized treatments that address significant unmet medical needs. Our expertise extends to Contract Manufacturing, where we produce Active Pharmaceutical Ingredients (APIs) through world-class facilities in Spain, Mexico, and China. Driven by our core values—people matter, transparency, and accountability—we’re committed to innovation, quality, and making a meaningful difference in healthcare. Because we care not only about what we do, but also about how we do it. #ESTEVE #AdvancingHealthTogether #Makeitsustainable #Pharmaceuticals #Innovation #SpecialtyPharma #APIs

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 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 is an advanced clinical-stage company specializing in cell expansion and engineering, providing molecules and bioengineering solutions to expand stem cells.

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.

Fractyl Health is a leader in creating innovative therapies to address the root cause of type 2 diabetes. We were founded based on groundbreaking research and biological insights showing the key role of the intestine in metabolic control. Our therapies are uniquely designed to target and control this controller for the treatment of metabolic disease. Our lead program, Revita DMR, is a first-in-class procedural therapy that is now in late-stage clinical trials for the treatment of type 2 diabetes. Our team is made up of passionate innovators at the intersection of biology and technology, working to deliver better real-world outcomes for people with metabolic disease.

Frantz Viral Therapeutics is focused on the development of topical treatments for pre-cancerous conditions related to human papillomavirus (HPV) infections in the cervix, vulva, and anus.

Frontera is a leading clinical-stage biotechnology company that seeks to develop the best gene therapy medicines to improve the lives of patients across multiple disease areas. Frontera's APEX Technology & Manufacturing Platform is an innovative adeno-associated virus (AAV) gene expression system. It includes novel and clinically validated AAV vectors, efficient and fully-integrated CMC processes, and access to early clinical data, making it possible to develop novel gene therapy products with quicker speed-to-market and lower cost.

GEMMABio, a new therapeutics company, will serve as the research and innovation arm to speed the research of and global access to life-changing advanced therapies for those living with rare diseases. Alongside Franklin Biolabs, GEMMABio will advance gene therapy research and deliver therapeutics to patients around the world significantly faster.

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.

At Geneos, our passion is to develop personalized therapies to unleash the most powerful force against cancer – your body’s own immune system. Our GT-EPIC Platform is designed to identify relevant neoantigen targets and then design, manufacture, and deliver tumor specific neoantigen-targeted personalized immunotherapies.

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 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.

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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.

Discovering and developing innovative medicines to preserve people’s sight and fight the devastating impact of blindness around the world.

HitGen Inc. (SSE: 688222.SH), founded in 2012, is headquartered in Chengdu, China, with subsidiaries in Cambridge, UK and Houston, USA. HitGen is committed to building a world-class innovative biopharmaceutical enterprise and contributing to the better life and health of mankind. Engaged in the discovery and optimization of small molecules and nucleic acid drugs, HitGen dedicates itself to cultivating an internationally leading drug discovery and optimization system centered on four key technology platforms, including DNA-encoded library technology (including DEL design, synthesis and screening, and application expansion), fragment-based drug discovery and structure-based drug design technologies (FBDD/SBDD), synthetic therapeutic oligonucleotide technology (STO), and targeted protein degradation technology (TPD). It provides new molecular entities (NMEs) at different stages of research and development for the pharmaceutical industry, through its diversified business models including research and development services, out-licensing of projects at different R&D stages, and new drug launches in the long term, with an aim to address unmet clinical needs with innovative therapeutic solutions. As a leader in the field of DEL technology, HitGen has been committed to the development, application and improvement of DEL technology since its establishment. HitGen has approximately 20 in-house drug discovery projects at different stages of research and development, among which 4 have obtained IND approvals and entered into clinical trials. HitGen is in collaboration with pharmaceutical, biotech and chemical companies, foundations and research institutes in the Americas, Asia, Europe, Africa, and Oceania. For more information, please call +86-28-85197385, +1-508-840-9646 or visit www.hitgen.com. For business development: bd@hitgen.com

HuidaGene Biotechnology Co., Ltd (辉大基因) is a global biotechnology company focusing on the discovering, engineering, and developing CRISPR-based genetic medicine to rewrite the future of genomic medicine. Based in Shanghai and New Jersey, HuidaGene is committed to addressing patient's needs globally with various preclinical therapeutic programs covering ophthalmology, otology, myology, and neurology. Company's CRISPR-based therapeutics offer the potential to cure patients with life-threatening conditions by repairing the cause of their disease. We are committed to transform the future of genome-editing medicine.

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.

Immorna is a rapidly expanding biotechnology company, focusing on the development of mRNA-based therapeutics and vaccines. Immorna is utilizing multiple mRNA platforms, including conventional, self-replicating and circular mRNA. Since it’s founding in 2019, Immorna has built a robust CMC platform for mRNA synthesis, purification, and analytical testing that is well suited for clinical and commercial development. In addition, with its state-of-the-art screening tools, Immorna has developed an arsenal of mRNA delivery vehicles, including polymers and lipid nanoparticles featuring multiple proprietary ionizable cationic lipids suitable for intramuscular, intravenous and tissue-targeting delivery. Immorna has a growing intellectual property portfolio and a diverse mRNA development pipeline spanning cancer immunotherapy, infectious diseases, rare genetic diseases, and medical cosmetology, and quickly advancing its oncology and infectious disease drug candidates into their clinical stages.

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.

INOVIO is a biotechnology company committed to powering a new way forward in DNA medicines to save and protect lives worldwide. The company is focused on rapidly bringing to market precisely designed and delivered DNA medicines to potentially treat and protect people from serious and life-threatening diseases associated with HPV, cancer, and infectious diseases.

Intima Bioscience - A clinical stage gene and cell therapy company focused on curative intent in solid tumor cancer

Invectys, Inc. is a privately owned clinical-stage company, headquartered in Houston, Texas, which is developing a new generation of First-in-Class products for cancer patients. Invectys has two wholly owned subsidiaries, Invectys, SAS (Paris) which is focused on scientific research and innovation and Invectys USA, Inc. (Houston) which is directing the clinical development of the Company’s lead HLA-G product. Since 2010, Invectys has raised over $60 million in private funds to develop its two innovative platforms of immunotherapy products which target “universal” tumor antigens.

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.

iVIEW Therapeutics Inc. is a clinical stage biotechnology company focusing on innovative ophthalmic therapeutics. We are driven by the pursuit of innovative therapies for clear vision and healthier eyes.

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 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 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.

Korro Bio was founded to turn extraordinary scientific insights into life-altering new treatments for patients. We harness the body’s natural RNA editing machinery to make precise, single-base RNA edits to modulate protein function, addressing the medical needs of patients. Our mission is to rewrite the future for patients living with debilitating disease by repairing the underlying genetic cause.

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 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 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 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.

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.

Next generation CRISPR technology for programmed cell death. Focused on developing therapies for infectious disease and the microbiome. Locus Biosciences’ novel approach to precision antimicrobials works by taking advantage of a part of an immune system present in many bacteria called the CRISPR-Cas system. The CRISPR-Cas system protects bacteria from invaders such as viruses by creating small strands of RNA called CRISPR RNAs, which match DNA sequences specific to a given invader. When the CRISPR RNAs find a match, they activate Cas proteins that cut the DNA.

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.

Longbio Pharma is founded in 2018, and located in Zhangjiang Hi-tech Park, Shanghai. There are 40+ scientists, 10+years experiences in protein drug development (antibodies and fusion proteins) .The co-founder team successfully developed two drugs for marketing (accumulated global sales USD 30 Billion). Longbio Pharma is focusing on R&D the innovative drugs of autoimmune and rare diseases (allergy and complement).

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, 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.

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.

Matrix Biomed, Inc. is a biopharmaceutical company focused on delivering gene-based therapeutics. Gene-based therapeutics are directed to address both the dysregulation of multiple genes as well as their interactions in gene regulatory networks. Only by addressing the dysregulation of multiple genes and their interactions in gene regulatory networks, Matrix Biomed, Inc. is able to deliver a therapy designed to truly revert a disease state to healthy state. Currently, Matrix Biomed, Inc. gene-based therapeutics are directed to therapies and drug products to make cancer treatment more manageable and successful through the elimination of the dose-limiting, life-threatening, and treatment-inhibiting toxicities associated with chemotherapy and radiotherapy while targeting the master genes responsible for cancer growth and development, hypoxia inducible factor -1 (HIF-1) and hypoxia inducible factor-2 (HIF2).

Maze Therapeutics is translating novel genetic insights into lifesaving medicines, through an approach that integrates human genetics and functional genomics. Advances in these areas have enabled us to decode the mysteries of genetic modifiers in a range of severe diseases, which we aim to turn into medicines that mimic protective mutations. We have assembled a world-class team that we need to realize this vision, including creative scientific pioneers in genetics, experienced company builders, and biotech industry leaders.

MED Institute accelerate product development. MED Institute is dedicated to bringing new medical device products to market that are safe and effective for patients. From concept to commercialization, our team has the knowledge and experience to guide your products through the complex steps required for market approval.

MeiraGTx is a clinical-stage gene therapy company focused on developing potentially curative treatments for patients living with serious diseases.

OUR MISSION: Deliver Innovation. Empower Discovery. Improve Life. For over 25 years, Mirus Bio has pioneered the development of transfection reagents and proprietary technologies for nucleic acid delivery applications. Our work is both technical and cutting-edge, and we promote a culture of creativity, responsibility, and respect. Our team members are engaged and motivated by the desire to support our customers and partners to achieve strong results. Mirus, together with Gamma Biosciences, is establishing the future of science and medicine by developing products that enable product developers within the cell and gene therapy space. As we approach a new era of addressing and conquering life-altering diseases, we will continue to expand our expertise and leadership in transfection to be integral in the future of advanced therapies.

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.

Myrtelle Inc. is a patient-centered, innovation-driven gene therapy company transforming the treatment of neurological diseases. Our mission is to develop and deliver novel treatments for devastating disorders of the central nervous system, beginning with our lead program, gene therapy treatment for Canavan disease. Myrtelle’s social media channels are places for community members to connect and learn about Canavan disease and Myrtelle initiatives. Discussion of medical and health topics should never be construed as medical advice. Contact a health care professional in your area for personal medical advice. Myrtelle Inc. does not represent that the information on our social media channels is accurate, complete, reliable, useful, timely, or current and Myrtelle does not undertake an obligation to update any such information. Myrtelle does not endorse any opinions or statements expressed by others on our social media channels. Myrtelle does not expressly endorse opinions or statements expressed by individuals, businesses, or channels we may ‘follow’, ‘like’, or ‘share’. At times, Myrtelle may link to external websites. Myrtelle is not responsible for the content or policies of these websites. Content submitted to our social media channels is public and will not place Myrtelle under any obligation to you.

Nanoscope is focused on proprietary ambient light activated optogenetic therapy to restore vision in people suffering from all forms of Retinitis Pigmentosa.

Neurogene is focused on developing life-changing genetic medicines for patients & families affected by rare, devastating neurological diseases.

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.

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., 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.

We are a leading technology platform that unlocks unlimited possibilities for the future of medicine through our intelligent modular design and tailored hyper targeting.

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.

Oncolytics Biotech Inc. is developing pelareorep, a safe and well-tolerated intravenously delivered immuno-oncolytic virus (IOV) that targets cancer through a unique mechanism of action with two components, selective tumor lysis and activation of the innate and adaptive immune systems, creating an inflamed phenotype to treat a variety of solid tumors and hematological malignancies.

Oncovir, Inc is a pharmaceutical corporation dedicated to the development of nucleic-acid-based clinical therapies for cancer, infectious, immune, and degenerative disorders. We promote Hiltonol® (poly-ICLC) as an experimental viral mimic and broad activator of innate and adaptive immunity. While initially developed as an interferon inducer, Hiltonol® has much broader biological effects in humans, including specific antiviral, immune activating, vaccine adjuvant, and antitumor actions.

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.

“One treatment a year. Life changing.” Oxular is developing disruptive treatments for retinal diseases. Oxular’s sustained-release formulations are engineered to last up to one year following single administration and perform in specific small spaces in the eye. This approach provides unique and precise drug distribution to tissues specifically involved in retinal diseases. Oxular’s drug administration technology is engineered to access these critical tissues through minimally invasive delivery. This unique combination aims to substantially improve patient quality of life by increasing therapeutic effectiveness and patient safety, while reducing side-effects and minimizing the frequency of treatments. Oxular’s product development pipeline includes treatments for prevalent retinal diseases, such as diabetic macular edema, as well as rare and orphan indications, including treatments for ocular cancers.

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.

PepGen Inc., a clinical-stage biotechnology company, focuses on the development of oligonucleotide therapeutics for use in the treatment of severe neuromuscular and neurologic diseases. Its lead product candidate is PGN-EDO51, an EDO peptide that id in Phase I clinical trial to treat individuals with Duchenne muscular dystrophy (DMD). The company is also developing PGN-EDODM1, an EDO peptide-conjugated PMO, which is in Phase 1 clinical trial for the treatment of myotonic dystrophy type 1, as well as EDO therapeutic candidates, such as PGN-EDO53, PGN-EDO45, and PGN-EDO44 for the treatment of DMD. PepGen Inc. was founded in 2018 and is headquartered in Boston, Massachusetts.

A start-up developing novel ophthalmic therapeutics to treat major unmet needs for patients.

Phoenix Nest Biotech is a biotechnology company that focuses on treating Sanfilippo Syndrome by collaborating with leading academics and utilizing licensed key technologies.

At PMV, we believe that by specifically targeting each mutant p53 we can fundamentally disrupt the course of cancer. Our research and development efforts are dedicated to selectively targeting mutant p53, providing unique therapies for any patient whose tumor harbors these gene mutations.

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 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 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.

PYC Therapeutics (ASX: PYC) is a clinical stage biotechnology company pioneering a new generation of RNA therapeutics for genetic diseases. It works by combining two complementary platforms: RNA-based drug design and PYC’s proprietary drug delivery technology. PYC's library of naturally derived cell penetrating peptides provide its delivery platform to overcome the major challenges of RNA therapeutics - getting into the target cell. PYC believes its PPMO (Peptide conjugated Phosphorodiamidate Morpholino Oligomer) technology enables a safer and more effective RNA therapeutic to address the underlying drivers of a range of genetic diseases for which no treatment solutions exist today. The Company is leveraging its leading-edge science to develop a pipeline of novel therapies including two programs focused on inherited eye diseases and preclinical discovery efforts focused on neurodegenerative diseases. PYC’s discovery and laboratory operations are located in Australia, and the Company’s preclinical and clinical operations are based in San Fransisco California.

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.

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 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.

Sensorion is a pioneering clinical-stage biotechnology company which specializes in the development of novel therapies to restore, treat and prevent within the field of hearing loss disorders Its clinical-stage portfolio includes one Phase 2 product: SENS-401 (Arazasetron) progressing in a planned Phase 2 proof of concept clinical study of SENS-401 in Cisplatin-Induced Ototoxicity (CIO) and, with partner Cochlear Limited, a study of SENS-401 in patients scheduled for cochlear implantation. Sensorion has entered into a broad strategic collaboration with Institut Pasteur focused on the genetics of hearing. It has two gene therapy programs aimed at correcting hereditary monogenic forms of deafness including OTOF-GT, targeting deafness caused by a mutation of the gene encoding for otoferlin, and hearing loss related to mutation in GJB2 gene to potentially address important hearing loss segments in adults and children (GJB2-GT).

Skyline Therapeutics is a biotechnology company that focuses on the discovery, development and delivery of innovative gene therapy treatments.

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.

SNIPR Biome is the leading CRISPR Microbiome company. Our mission is to develop CRISPR-based medicines that give hope for effective and safe treatment of difficult-to-treat diseases in the future. Precision killing of bacteria has the impact to revolutionise the management of untreatable and difficult-to-treat infections as well as complex diseases directly impacted by the human microbiota.

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 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.

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.

Acquired by Sumitomo Dainippon Pharma of Japan. Spirovant Sciences is a gene therapy company focused on changing the course of cystic fibrosis and other respiratory diseases. The company's current focus is on developing novel gene therapies for cystic fibrosis and other pulmonary diseases. Spirovant is located in Philadelphia, PA's thriving Life Sciences Corridor and has a diverse management team, researchers, and partners.

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.

Spur Therapeutics (formerly Freeline Therapeutics) is a clinical-stage biotechnology company focused on developing life-changing therapies for debilitating chronic conditions. By optimizing every component of our product candidates, we are improving genetic expression and targeted delivery to realize outsized clinical results. Building on the successes of our two potential first-in-class therapies for Gaucher disease and adrenomyeloneuropathy (AMN), we’re moving from rare diseases toward more widespread conditions, including forms of Parkinson’s and dementia, and even certain cardiovascular diseases. Expanding our impact, and pushing forward to new frontiers of genetic medicine. Toward life-changing therapies, and brighter futures. Toward More™

We are a clinical stage oncology focused company developing novel gene therapies focused on dermatological and visceral cancers.

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 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.

Established in 2007, Suzhou Ribo Life Science Co., Ltd. (Ribo) is a clinical stage company devoted to the development of innovative RNAi technologies and oligonucleotide therapeutics as an effort to address unmet clinical needs in the world with the tools, capacity and RNA therapeutics that we build up in China. Fueled by our mission to make China's oligonucleotide therapeutics serve the world, we seek. Based on its cutting edge RNAi technologies, Ribo has established oligonucleotide therapeutic platform that vertically integrates the complete set of technologies to support the full life cycle of oligonucleotide therapeutics from early R&D to commercialization.

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 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, 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 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.

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.

TERN THERAPEUTICS is a privately-held biotechnology company founded in 2023 with a new vision for speeding the development of transformative, one-time gene therapy medicines for rare diseases. Guided by a team of leading physicians, scientists, and business leaders and in collaboration with patient communities, we are driven to deliver transformative treatments with urgency to those living around the world with rare disease.

TransCode's lofty goal is delivering a cancer-free future for all cancer patients. Our core belief is that cancer can be overcome through the intelligent design and efficient delivery of targeted therapeutics. Our lead therapeutic candidate, TTX-MC138, targets microRNA-10b, or miRNA-10b, considered the master regulator of metastatic cell viability in a range of cancers, including breast, pancreatic, ovarian, colon cancer, glioblastomas, and several others. Our other preclinical programs include two solid tumor programs, TTX-siPDL1, an siRNA-based modulator of programmed death-ligand 1, or PD-L1, and TTX-siLIN28B, an siRNA-based inhibitor of RNA-binding protein LIN28B. TransCode also has three cancer agnostic programs, TTX-RIGA, an RNA- based agonist of the retinoic acid-inducible gene I, or RIG-I, targeting activation of innate immunity in the tumor microenvironment; TTX-CRISPR, a CRISPR/Cas9-based therapy platform for the repair or elimination of cancer-causing genes inside tumor cells; and TTX-mRNA, an mRNA-based platform for the development of cancer vaccines that activate cytotoxic immune responses against tumor cells. Until now, targeting biomarkers that are the primary drivers of cancer onset, progression and recurrence have been locked behind the challenge of delivery – cytosolic delivery to engage these known targets. TransCode believes it has overcome this barrier, opening the door to a broad array of cancer therapies -- a disease with the highest unmet need that affects everyone. It is through the design and optimization of this technology that TransCode has devised an elegant solution -- TTX -- a proprietary delivery platform for the transport of targeted therapeutics to cancer cells. With their world class team and know how, TransCode has a rapidly expanding platform of drug candidates designed to target a variety of tumor indications with the goal of long-term treatment survival.

Cancer immunotherapy Transgene (Euronext: TNG) is a biotechnology company focused on designing and developing novel immunotherapies for the treatment of cancer.

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.

Virogin Biotech is a biotechnology company that specializes in oncolytic virus drug development to create impactful drugs for patients with the highest medical need.

Vironexis is focused on transforming the future of cancer treatment by pioneering AAV-delivered T-cell immunotherapy. Our TransJoin™ AAV Gene Therapy Platform enables the creation of off-the-shelf, single-dose gene therapies designed to overcome the key challenges and shortcomings of current immunotherapies, including CAR-T and bispecific antibodies. Our current pipeline includes more than ten product candidates for blood-based cancers, solid tumor metastasis prevention, and a cancer vaccine. Our lead program, VNX-101, for the treatment of CD19+ acute lymphoblastic leukemia, is anticipated to begin clinical trials in the fourth quarter of 2024.

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.

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, 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 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.

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 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.