List of Gene Therapy Companies with Phase 1 Active Clinical Trial - 78

Abata Therapeutics - We are bringing an entirely new approach to the treatment of autoimmune disease by engineering Tregs as targeted therapies that stop immune-mediated destruction, restore homeostasis – a state of harmony – and promote repair in the affected tissues. In addition to our lead program in progressive MS, Abata has early programs in Type 1 Diabetes (T1D) and inclusion body myositis (IBM). We bring together industry experts and deeply engaged pioneers in Treg biology, T cell receptor and antigen discovery, disease pathogenesis, and molecular and imaging biomarkers. We are bold in our mission and purpose and aim for nothing less than a transformative impact on people’s lives.

Founded in 2016, AffyImmune is a clinical-stage biotechnology company that optimizes CAR T cells for the treatment of solid tumors using our Tune & Track platform. AIC100, our first product, spares healthy cells through our unique affinity-tuning, enhancing its safety and efficacy. The initial indication for AIC100 is refractory thyroid cancer, which has a very poor prognosis and very few effective therapies. The antigen targeted by AIC100 is also overexpressed in a variety of additional cancers, including stomach, triple negative breast cancer and a number of others.

Ajinomoto is a global leader in the research, development, manufacture and sale of amino acid-based products for the pharmaceutical, nutraceutical, sports nutrition, health and beauty industries as well as food ingredients and consumer food products.

Alcyone Therapeutics is a biotechnology company pioneering next-generation precision gene-based therapies for complex neurological conditions. The company integrates innovation in neuroscience, precision dosing platforms, and manufacturing capabilities to deliver transformative therapies to patients. Alcyone leverages the synergy between FalconTM, the company’s proprietary intrathecal precision dosing and biodistribution platform that incorporates deep knowledge of cerebral spinal fluid (CSF) dynamics, computational modeling, and bioengineering, and four novel gene-based therapeutics platforms developed at the Abigail Wexner Research Institute at Nationwide Children’s Hospital (AWRI). This comprehensive approach allows for the optimization of central nervous system (CNS) dosing and delivery to better target the pathophysiology and anatomy specific to various neurological diseases. Alcyone’s lead programs utilize X chromosome reactivation for X-linked disorders and targets the treatment of Rett syndrome, and gene replacement for the treatment of IGHMBP2-related disorders including spinal muscular atrophy with respiratory distress type 1 (SMARD1) and Charcot Marie Tooth disease type 2S (CMT2S).

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/

Aspen is developing induced-pluripotent stem cells (iPSCs) to address diseases with high unmet medical need, beginning with autologous neuron replacement for Parkinson’s disease (PD) and extending across the brain and affected organs.

AvenCell - Based on our deep science and focused on patients in need, we develop next generation immunotherapies for hard-to-treat cancers.

On August 20, 2024, Base Therapeutics announced that its AccuBase® base editor has officially received authorization for two patents from the United States Patent and Trademark Office (USPTO). This marks that both of Base Therapeutics' AccuBase® technologies have secured patent protection in China and overseas markets. Base Therapeutics' strategic positioning in the gene editing field is at the forefront internationally, making it one of the biopharmaceutical companies with the most authorizations domestically and abroad.

Biosyngen is a cell therapy company focusing on unmet needs with technology targeting solid tumors and lymphoproliferative diseases. The company’s first-in-class products have entered into Phase I/II clinical trials for nasopharyngeal cancer (ODD and Fast-Track), lymphoproliferative disease and lung cancer. The company continues to enrich its pipeline with further IND submissions. Biosyngen drives its global strategy from the dual HQ in Singapore and Guangzhou. Leverages on the public-private biopharma ecosystem for the better cause. Committed to giving cancer patients a better chance to survive, our pipeline is enriched, covering other major indications which are on track for regulatory registration. Biosyngen seeks breakthrough in collaborations, particularly in translational R&D and clinical development. The company has positioned GMP cell therapy manufacturing facilities in two locations - in Singapore and Guangzhou covering global needs. To quicken drug development from bench-to-bedside, a parallel effort in the form of the Translational Medicine Centre is set up. It is public-private-partnership connecting stakeholders in R&D, Clinical Development, Hospitals, Pharma, Enterprises and Venture Capital.

Capstan Therapeutics is a biotechnology company dedicated to advancing in vivo cell reprogramming. The core platform technology comprises proprietary targeted lipid nanoparticles conjugated with a recombinant protein binder such as monoclonal antibodies (tLNPs) designed to deliver payloads, including mRNA or gene editing tools, that can reprogram specific cell types in vivo. The platform technology has the potential to generate transformative therapies with applications across a broad range of disease areas, including oncology, autoimmune disorders, fibrosis, and monogenic blood disorders.

Carisma Therapeutics Inc. is a clinical stage biopharmaceutical company focused on utilizing our proprietary macrophage and monocyte cell engineering platform to develop transformative immunotherapies to treat cancer and other serious diseases. We have created a comprehensive, differentiated proprietary cell therapy platform focused on engineered macrophages and monocytes, cells that play a crucial role in both the innate and adaptive immune response. Carisma is headquartered in Philadelphia, PA.

Century Therapeutics, Inc., a biotechnology company, develops transformative allogeneic cell therapies for the treatment of solid tumor and hematological malignancies. The company’s lead product candidate is CNTY-101, an allogeneic, induced pluripotent stem cells (iPSCs)-derived chimeric antigen receptors (CAR)-iNK cell therapy targeting CD19 for relapsed, refractory B-cell lymphoma. It is also developing CNTY-103, a CAR-iNK candidate targeting CD133 + EGFR for recurrent glioblastoma; CNTY-102, a CAR-iT or CAR-iNK candidate targeting CD19 + CD79b for relapsed, refractory B-cell lymphoma and other B-cell malignancies; and CNTY-104, a CAR-iT or CAR-iNK multi-specific candidate for acute myeloid leukemia. Century Therapeutics, Inc. has a research collaboration with Outpace Bio, Inc. for CAR engineering. The company was founded in 2018 and is headquartered in Philadelphia, Pennsylvania.

Codagenix Inc. is a clinical stage biotechnology company with vaccine products against influenza, respiratory syncytial virus, dengue virus, and others currently under development. Codagenix uses a computer-based algorithm, called SAVE (Synthetic Attenuated Virus Engineering) to design viral genomes that are identical to the wild type strain at the amino acid level, but make less protein in human cells. The highly attenuated viruses make excellent vaccines or oncolytics against solid tumors. Codagenix represents the next wave in vaccine technology, untethered from traditional, time-consuming and unpredictable approaches to attenuating viruses.

Comanche is a biopharmaceutical company developing novel siRNA molecules for the treatment of preeclampsia. Our purpose is to lower the risks of pregnancy and prematurity worldwide by safely sustaining natural pregnancy and allowing for fetal maturation. We envision a world where all pregnant women have access to safe and effective therapies that are evidence-based, cost-effective and sustainable. Our mission is to prevent or treat preeclampsia at its root cause.

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.

CorrectSequence Therapeutics (Correctseq) is a clinical-stage biotech company employing its proprietary transformer base editing (tBE) technology to pioneer next-generation gene editing therapies. Our leading pipeline candidate, CS-101, utilizing innovative base editor targeting HBG, curing β-hemoglobinopathies, has obtained IND approval from the China NMPA. Clinical data demonstrates its superior performance, indicating significant potential for CS-101 to become a best-in-class gene editing therapy for curing patients with β-hemoglobinopathies. Proof-of-concept (POC) data in mice for in vivo pipelines using tBE-editing therapies via lipid nanoparticle (LNP) delivery are available, including targets for metabolic dysfunction and associated diseases. Ex vivo multiplex editing of T cells on multiple targets simultaneously preserved T cell growth and function in vivo compared to non-edited cells, establishing tBE as the ideal gene editing tool for the next-generation cell therapy development. tBE offers significant advantages in controlling off-target effects and enhancing in vivo editing efficiency, making it ideal for both multiplex editing and precise single-target editing. It is compatible with various delivery system, including ex vivo editing in hematopoietic stem cells (HSCs) and T cells, as well as in vivo editing via LNPs or adeno-associated viruses (AAVs). We are developing multiple pipeline programs targeting genetic diseases, metabolic disorders, and cardiovascular diseases. Our focus on biotechnology innovation, research and development underscores our commitment to providing efficient, reliable and safe solution for unmet medical needs.

CureVac (Nasdaq: CVAC) is a pioneering multinational biotech company founded in 2000 to advance the field of messenger RNA (mRNA) technology for application in human medicine. In more than two decades of developing, optimizing, and manufacturing this versatile biological molecule for medical purposes, CureVac has introduced and refined key underlying technologies that were essential to the production of mRNA vaccines against COVID-19, and is currently laying the groundwork for application of mRNA in new therapeutic areas of major unmet need. CureVac is leveraging mRNA technology, combined with advanced omics and computational tools, to design and develop off-the-shelf and personalized cancer vaccine product candidates. It also develops programs in prophylactic vaccines and in treatments that enable the human body to produce its own therapeutic proteins. Headquartered in Tübingen, Germany, CureVac also operates sites in the Netherlands, Belgium, Switzerland, and the U.S. Further information can be found at www.curevac.com.

Incorporated in 2018, CytoMed was spun off from the Agency for Science, Technology and Research (A*STAR), Singapore’s national research and development agency in the public sector. CytoMed is a biopharmaceutical company focused on harnessing its licensed proprietary technologies to create novel cell-based immunotherapies for the treatment of human cancers. The development of novel technologies has been inspired by the clinical success of existing CAR-T in treating hematological malignancies as well as the current clinical limitations and commercial challenges in extrapolating the CAR-T principle into treatment of solid tumors.

CytomX Therapeutics, Inc. is committed to changing the treatment of cancer with our novel Probody® therapeutic platform. We have a broad pipeline comprised of five clinical-stage programs, with even more in development. Our commitment to transforming lives with safer, more effective therapies is driven by our curiosity and passion for innovation, and our belief that by acting with integrity in an honest, respectful, ethical manner, we have the power to change lives. Our workplace embodies collaboration, open communication, celebrating our successes and holding each other to the highest possible standards. CytomX embraces diversity and seeks to enhance and maintain our culture of equity and inclusion which encourages individuals to be themselves, feel involved, respected and connected. We at CytomX believe that we benefit from each other’s ideas and experiences. We accept our differences and learn from one another, improve together, and embrace change to achieve our corporate and individual objectives. This belief applies to how we work together at CytomX, how we work with others outside CytomX, how we represent CytomX in our communities, and how we endeavor to bring our scientific innovations to a diverse patient population. CytomX acknowledges the importance of diversity, equity and inclusion initiatives and that they require an evolving and ongoing commitment. CytomX is an equal opportunity employer and does not discriminate against any individual or potential candidate because of race, color, religion, sex, national origin, sexual preference or any other legally protected category. CytomX is located in South San Francisco, California, the birthplace of biotechnology. Learn more about us and how we are advancing science to treat cancer differently at www.cytomX.com.

Elixirgen Therapeutics is a biotechnology company advancing its controllable self-replicating RNA technology to develop therapies for a broad spectrum of diseases, including rare diseases, aging-related disorders, and cancer. Elixirgen Therapeutics is located in the Science + Technology Park at Johns Hopkins in Baltimore.

Encellin is a biotechnology company specializing in cell encapsulation technology for cell-based therapeutics. The company's initial pipeline focuses on endocrine disorders with a clear mechanism of action, particularly focusing on Hypoglycemia. Encellin was founded based on the technology developed in the lab of Dr. Tejal Desai at the University of California in San Francisco.

Epigenic Therapeutics is a frontier biotechnology company dedicated to developing next generation gene editing therapy utilizing regulation of epigenetic genome for a variety of diseases. Founded in 2021 by leading scientists focused on discovering gene editing technologies and developing gene editing therapies, the company has multiple product candidates in the pipeline, including treatment for metabolic, cardiovascular, viral hepatitis, ocular and rare diseases.

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

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.

Excision BioTherapeutics is a private company focused on developing advanced gene editing therapeutics to address the medical need for the treatment of life-threatening disease caused by viral infections. Our mission is to advance gene editing therapeutics for our indications into safe and efficacious medicines that will greatly improve the lives of infected individuals around the world.

Exegenesis Bio is a clinical stage global gene therapy company with operations in Philadelphia, Boston and China. The company’s innovative gene therapy pipeline is based on proprietary capsids, promoters and unique protein engineering designs. Two programs have advanced to the clinical stage: (1) Type I Spinal Muscular Atrophy (SMA) AAV gene therapy in China (2) neurovascular AMD (wet AMD) rAAV gene therapy in USA The company has built state-of-the-art cGMP manufacturing facilities that include 500L and 2,000L disposable bioreactors for viral vectors and 30L disposable fermenters for plasmids. Exegenesis Bio has raised over $150 M since inception in 2019 and currently employs over 200 scientific and operations staff worldwide. Website: https://www.exegenesisbio.com/ Contacts: Company Information: Contact@ExegenesisBio.com Business Development: BD@ExegenesisBio.com Careers: Careers@ExegenesisBio.com

Expression Therapeutics, Inc. is a fully integrated clinical stage gene and cell therapy company developing novel, potentially curative, therapeutics for hematology and oncology. The Company, based in Atlanta, Georgia, has a state-of-the-art 43,000 square foot manufacturing facility in West Chester, Ohio. Expression Therapeutics, Inc. also built a state-of-the-art scalable 43,000 square foot manufacturing facility in Cincinnati, Ohio.

Fate Therapeutics (Nasdaq: FATE) is a clinical-stage biopharmaceutical company dedicated to the development of next-generation cellular immunotherapies for cancer and immune disorders.

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.

A Biotechnology Research private company. Mission is to take on one of the largest problems in all of medicine – the leading cause of disability, chronic pain, and a significant risk factor for opioid addiction – osteoarthritis (OA).

GeneCraft is a pre-clinical-stage biotech company dedicated to improving treatment and clinical outcomes for patients through gene therapy that harness the power of the immune system. The company's technology allows for the development of gene therapies for the treatment of cancer.

GentiBio, Inc., is an early stage biotherapeutics company co-founded by pioneers in Treg biology and synthetic immunology to develop engineered regulatory T cells (EngTregs) programmed to treat autoimmune, alloimmune, autoinflammatory and allergic diseases. GentiBio's proprietary autologous and allogeneic EngTregs platform integrates key complimentary technologies needed to successfully restore immune tolerance and overcome major limitations in existing regulatory T-cell therapeutics. GentiBio is at the forefront of leveraging a unique therapeutic modality that can be used to address the fundamental cause of many diseases that result from overshooting and/or malfunctioning of the immune system.

GenVivo will make first- and best-in-class immune therapies that are immediately and easily deployable, highly effective, and which improve survival and quality of life beyond current therapies.

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HDT Bio is a vaccine and immunotherapy Biotechnology company developing drugs for immunotherapy of cancers and infectious diseases with next-generation approaches designed to enable the body’s immune system to prevent and cure disease. We have engineered our technologies to Access, Activate, and Amplify the immune system’s natural ability to create antibodies and T-cells that fight cancer and infectious diseases such as SARS-CoV-2. We are developing multiple product candidates from our proprietary discovery platforms, including LION™, which has the potential to provide health benefits to a broad patient population. Our products are designed to be cost-effective solutions to unmet medical needs and complementary to existing, cutting-edge immunotherapies. HDT Bio offers a comprehensive compensation plan which includes health insurance for employees and dependents (inclusive of multiple plants to choose from, including a $0 premium plan with HSA and company contribution), generous PTO (11 observed holidays and week long year-end closure), stock options with Carta Tax Advisory for equity and tax support, flexible spending accounts (FSAs), commuting benefits (ORCA or paid parking), and retirement benefits with company match. HDT Bio Corp. is an equal opportunity employer committed to a diverse and inclusive workforce. This environment is encouraged by HDT's culture, which supports women in science through our Women+ group, creates inclusive events to highlight the diverse cultures represented by our staff, and with the adoption of an open office environment for all.

Hopstem Bioengineering Co., Ltd. was founded on January 2017 in Hangzhou, by neuroscientists and stem cell biologists from Johns Hopkins University. Hopstem has established world-leading iPSCs platform of neural differentiation and cell engineering. Hopstem has developed several critical patented technologies，as well as CMC platform for iPSC-derived cell therapy products. Hopstem has iPSC reprogramming patent, GMP iPSC line with global licensing rights, iPSC-derived cell product manufacturing and quality system, and a variety of cell therapy products and pipelines, targeting CNS diseases, optical diseases and tumor. Hopstem’s first clinical product - neural progenitor cell hNPC01 targets neural injury diseases such as stroke and traumatic brain injury, has received China's IND approval and entered clinical trials. Hopstem is committed to translating the most cutting-edge technologies to provide global patients with safe, efficient and affordable cell therapy products.

iCell Gene Therapeutics is developing Chimeric Antigen Receptor (CAR) engineered cells with improved functional properties and expanded applicability. We are a clinical stage company focused to demonstrate clinical proof of concept for unique, patent protected, targets and strategies. Our pipeline includes multiple additional first in man CAR strategies to target hematological cancers, non-hematological cancers and autoimmune disorders.

The company is focusing on research and development of immunotherapies for cancers, inflammatory and infectious diseases based on its patented PD-1-enhanced DNA vaccine and Anti-Δ42PD1 Antibody Immuno Blocking technology platforms; with 2 vaccines in first-in-human clinical trials.

Immusoft is a cell therapy company focused on developing novel therapies for rare diseases using sustained delivery of therapeutic proteins from a patient’s own cells. The company is developing a technology platform called Immune System Programming (ISP™), which modifies a patient’s B cells and instructs them to produce gene-encoded medicines. The B cells that are reprogrammed using ISP become miniature drug factories that are expected to survive in patients and provide therapeutic benefit for many years. The company is based in Seattle, WA.

Interius is an early-stage biotechnology company expanding the potential of cell and gene therapy by developing next-generation delivery technology based on inventions from the laboratory of Dr. Saar Gill at the University of Pennsylvania.

KaliVir Immunotherapeutics was founded in 2019 to pioneer a unique and novel approach to cancer treatment that engages a virus' natural ability to replicate in and kill cancer cells. This modality is known as oncolytic viral immunotherapy. KaliVir's cutting-edge, next generation oncolytic viral immunotherapy programs combine technologies developed in-house and licensed from University of Pittsburgh's world-class oncolytic virus and immunotherapy research programs.

Kiromic BioPharma is an allogeneic Gamma Delta T-cell therapy company featuring unique, proprietary, end-to-end bioinformatic, AI targeting, and manufacturing technologies to address solid tumors. We are a clinical-stage, fully integrated biotherapeutics company using its proprietary DIAMOND® artificial intelligence (AI) 2.0 target discovery engine to develop and commercialize cell therapies focusing on immuno-oncology. Kiromic is developing a multi-indication allogeneic cell therapy platform that exploits the natural potency of Gamma Delta T-cells to target solid tumors. Kiromic’s DIAMOND® AI is where data science meets target identification to dramatically compress the years and hundreds of millions of dollars required to develop a live drug. The Company maintains offices in Houston, Texas. To learn more, visit www.kiromic.com and connect with us on Twitter and LinkedIn.

Legend Biotech Corporation, a clinical-stage biopharmaceutical company, engages in the discovery and development of novel cell therapies for oncology and other indications. Its lead product candidate, LCAR-B38M/JNJ-4528, is an autologous CAR-T cell therapy that targets the B-cell maturation antigen. The company is conducting multiple clinical trials to evaluate LCAR-B38M/JNJ-4528 as an earlier line of therapy for multiple myeloma (MM), as well as a comparison of the treatment with standard triplet therapy in Revlimid-refractory MM. It also has a broad portfolio of earlier-stage autologous product candidates targeting various cancers, including non-hodgkins lymphoma (NHL), acute myeloid leukemia, and T cell lymphoma. In addition, the company is developing an allogeneic CAR-T product candidate targeting CD20 for the treatment of NHL, which is currently in an investigator-initiated Phase 1 clinical trial in China. Further, it has various product candidates in early preclinical and clinical development for the treatment of solid tumors, as well as infectious diseases. The company was founded in 2014 and is based in Somerset, New Jersey. Legend Biotech Corporation is a subsidiary of Genscript Biotech Corporation.

Marker Therapeutics, Inc., a clinical stage immuno-oncology company, engages in the development and commercialization of various T cell-based immunotherapies and peptide-based vaccines for the treatment of hematological malignancies and solid tumor indications. Its MultiTAA-specific T cell technology is based on the expansion of non-engineered tumor-specific T cells that recognize tumor associated antigens and kill tumor cells expressing those targets. The company’s MultiTAA-specific T cell therapies include autologous T cells for the treatment of lymphoma, multiple myeloma, and various solid tumors; and allogeneic T cells for the treatment of acute myeloid leukemia and acute lymphoblastic leukemia. It is also developing TPIV100/110, which is in Phase II clinical trial for the treatment of breast cancer; and TPIV200 that is in Phase II clinical trial for the treatment of breast and ovarian cancers. Marker Therapeutics, Inc. is headquartered in Houston, Texas.

Medigene AG (FSE: MDG1) is a leading immuno-oncology platform company dedicated to developing T-cell therapies to effectively eliminate cancer. Its end-to-end technology platform is built on multiple proprietary exclusive product development and product enhancement technologies, and allows Medigene to create best-in-class differentiated, T cell receptor engineered T cell (TCR-T) therapies for multiple solid tumor indications that are optimized for both safety and efficacy. This platform provides product candidates for both its in-house therapeutics pipeline and partnering.

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.

The METiS platform (AiTEM) combines state-of-the-art AI data-driven algorithms, mechanism- driven quantum mechanics and molecular dynamics simulations to calculate Active Pharmaceutical Ingredient (API) properties, elucidate API-target and API-excipient interactions, and predict chemical, physical and pharmacokinetic properties of small molecule and nucleic acid therapeutics in specific microenvironments. This enables efficient lead optimization, candidate selection and formulation design. Founded by a team of MIT researchers, serial entrepreneurs and biotech industry veterans, METiS develops and in-licenses novel assets with high therapeutic potential that could benefit from its data-driven platform.

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.

At Myeloid, we have applied our extensive knowledge of advanced gene and cell therapies to build novel platform technologies that harness the innate abilities of myeloid cells. We have translated the learnings from our backgrounds and novel platform to create a clinical-stage, mRNA-immunotherapy pipeline focused on breakthrough therapies for cancer.

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.

A global biotech company commited to developing new and effective therapies for psychiatric and neurological disorders affecting millions of patients in need. We design novel CNS-tropic AAV capsids and innovative gene expression cassettes to achieve efficient, effective, and safe gene therapies for severe CNS disorders.

Nkarta is a clinical-stage biotechnology company advancing the development of allogeneic natural killer (NK) cell therapies. By combining its cell expansion and cryopreservation platform with proprietary cell engineering technologies, Nkarta is building a pipeline of cell therapy candidates generated by efficient manufacturing processes, and engineered to enhance tumor targeting and improve persistence for sustained activity in the body. For more information, please visit the company’s website at www.nkartatx.com.

Our mission is to develop effective and affordable preventive vaccines for Alzheimer’s disease, Parkinson’s disease, and other neurodegenerative disorders. Using our universal vaccine platform for Alzheimer’s, our goal is to induce antibody titers high enough to penetrate the blood-brain barrier and prevent pathological plaques, fibrils, and oligomers to delay disease onset in cognitively unimpaired people at risk of Alzheimer’s.

Nurix Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of small molecule therapies for the treatment of cancer and immune disorders. The company develops NX-2127, an orally available Bruton’s tyrosine kinase (BTK) degrader with immunomodulatory drug (IMiD) activity for the treatment of relapsed or refractory B-cell malignancies; NX-5948, an orally bioavailable BTK degrader without IMiD activity for the treatment of relapsed or refractory B-cell malignancies; and NX-1607, an orally available Casitas B-lineage lymphoma proto-oncogene-B (CBL-B) inhibitor for immuno-oncology indications. It also develops NX-0255, a CBL-B inhibitor for ex vivo use to enhance adoptive T-cell therapy; DeTIL-0255 for tumor infiltrating lymphocytes therapy; KINASE-CTM3 to treat T cell malignancies and autoimmune diseases; and LIGASE-INH2 for immuno-oncology. In addition, the company develops programs, such as COVID-CTM1, COVID-CTM2, and COVID-CTM3 that are designed for protein degradation to SARs CoV2 targets. Nurix Therapeutics, Inc. has a strategic collaboration agreement with Gilead Sciences, Inc. for cancer and other challenging diseases patients; and Sanofi S.A. The company was formerly known as Nurix Inc. and changed its name to Nurix Therapeutics, Inc. in October 2018. Nurix Therapeutics, Inc. was incorporated in 2009 and is headquartered in San Francisco, California.

OXB (LSE: OXB) is a global quality and innovation-led contract development and manufacturing organisation (CDMO) in cell and gene therapy with a mission to enable its clients to deliver life changing therapies to patients around the world. One of the original pioneers in cell and gene therapy, OXB has 30 years of experience in viral vectors; the driving force behind the majority of cell and gene therapies. OXB collaborates with some of the world’s most innovative pharmaceutical and biotechnology companies, providing viral vector development and manufacturing expertise in lentivirus, adeno-associated virus (AAV), adenovirus and other viral vector types. OXB's world-class capabilities span from early-stage development to commercialisation. These capabilities are supported by robust quality-assurance systems, analytical methods and depth of regulatory expertise. OXB offers a vast number of unique technologies for viral vector manufacturing, including a 4th generation lentiviral vector system (the TetraVecta™ system), dual plasmid system for AAV production, suspension and perfusion process using process enhancers and stable producer and packaging cell lines.

Phio Pharmaceuticals Corp. (Nasdaq: PHIO) is a clinical stage biotechnology company whose proprietary INTASYL™ RNAi technology makes immune cells more effective in killing tumor cells. INTASYL is the only self-delivering RNAi technology focused on immuno-oncology therapeutics. INTASYL drugs precisely target specific proteins that reduce the body’s ability to fight cancer, without the need for specialized formulations or drug delivery systems. Phio was co-founded by Nobel Laureate Craig Mello, who was awarded the Nobel prize in 2006 for his discovery of RNAi, which has broad therapeutic applications. Our founders opened up a whole new field of biology when they discovered RNAi, and we continue their pioneering efforts today through our work with INTASYL. We’re committed to discovering and developing innovative cancer treatments for patients, creating new pathways for a cancer-free future. For additional information, visit the Company's website, www.phiopharma.com. #INTASYL #siRNA #RNAi #Phio #immunooncology

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

Our mission at Poseida Therapeutics is to create cell and gene therapies with the capacity to cure. We are developing novel products using our broad gene engineering platform technologies, with the goal of bringing new and better treatments to patients with serious diseases, first in the blood-based cancer Multiple Myeloma and next in solid tumors like prostate cancer.

Precision BioSciences, Inc. (Nasdaq: DTIL), is a clinical stage biotechnology company dedicated to improving life (DTIL) with its novel and proprietary ARCUS® genome editing platform. ARCUS is a highly specific and versatile genome editing platform designed with therapeutic safety, delivery, and control in mind. Using ARCUS, Precision is developing allogeneic CAR T and in vivo gene correction therapies for cancer and infectious diseases.

Ray Therapeutics Inc (RayTx) is a late-stage preclinical Bay Area biotechnology company pioneering the first-to-market precision optogenetic therapy for vision restoration that is highly active in ambient lighting (and will not require light-enhancing goggles). Our studies have shown for the first time that our proprietary ChRown optogenetic protein can restore high levels of visual acuity, contrast sensitivity and visual field. RayTx lead asset, RTx-015, is expected to address the huge unmet medical in people suffering from many forms of retinal blindness (independent of the underlying genetic cause), including those with advanced age-related macular degeneration. RayTx has partnered with a world-leading AAV gene therapy manufacturer is utilizing their proprietary commercial manufacturing process and analytics to maximally de-risk clinical development. RayTx anticipates clinical trials will begin by the end of 2023 in our initial indication, retinitis pigmentosa.

ReCode Therapeutics is a clinical-stage genetic medicines company using superior delivery to power the next wave of mRNA and gene correction therapeutics. ReCode’s selective organ targeting (SORT) lipid nanoparticle (LNP) platform is a next-generation, genetic medicines technology that enables precise delivery to target organs and cells beyond the liver.

ReiThera is a company devoted to developing and producing biopharmaceutical products based on gene delivery technologies doe advanced therapies, in order to prevent and treat several serious or life-threatening diseases.

Sana Biotechnology is focused on utilizing engineered cells as medicines for patients. The ability to modify genes and use cells as medicines will be one of the most important advances in healthcare over the next several decades. Sana is building differentiated capabilities across the spectrum of cell and gene therapy. Three aspirations drive Sana as we look to discover treatments for patients with poor outcomes or currently untreatable diseases. The first is the ability to repair and control the genes in any cell in the body. We are advancing novel delivery technologies with the goal of being able to deliver any payload to any cell in a specific, predictable, and repeatable manner, paving the way for next-generation in vivo gene therapy. Next is the ability to differentiate pluripotent stem cells ex vivo into immune-cloaked functional cells with the aspiration of being able to replace any missing or damaged cells in the body. Last is a belief we can enable broader access to our therapies through focusing on scalable manufacturing solutions, the cost of manufacturing, and aligning with key stakeholders. Sana launched in early 2019 and has over 350 employees in Seattle, Cambridge, and South San Francisco. Sana was founded with the long view – bringing together great people and the best technologies to deliver on the challenging promise of using engineered cells to meaningfully change the outcome of many human diseases.

Our mission is to create a new generation of smarter medicines that outmaneuver complex diseases in ways previously inconceivable. To accomplish this mission, we are building a synthetic biology platform that could enable us to program next-generation cell and gene therapies with what we refer to as “gene circuits”. These gene circuits, which are created from novel and proprietary combinations of DNA sequences, are intended to reprogram cells with biological logic to sense inputs, compute decisions and respond to their cellular environments. We are designing gene circuits to improve the “intelligence” of cell and gene therapies in order to enhance their therapeutic effectiveness against a broad range of diseases that conventional medicines do not readily address. For more information, please visit the Senti Bio website at https://www.sentibio.com.

Sonoma Biotherapeutics is developing engineered regulatory T cell (Treg) therapies to treat serious autoimmune and inflammatory diseases driven by an imbalanced immune system. Founded by pioneers in Treg biology and cell therapy, the company is employing proprietary platform technologies and approaches to develop a new generation of targeted Treg cell therapies designed to induce durable immune tolerance to treat and prevent autoimmune and inflammatory diseases. Sonoma Biotherapeutics is based in South San Francisco and Seattle.

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.

Synthekine is an engineered cytokine therapeutics company developing disease-optimized treatments. The company uses immunological insights to guide targeted protein engineering to generate transformative medicines for cancer and autoimmune disorders. Using the principles of cytokine partial agonism and immunological specificity, Synthekine designs differentiated therapeutics to be both safe and efficacious. Its lead programs have shown promising efficacy and tolerability in preclinical studies, and it is developing additional cytokine partial agonists that selectively modulate key pathways of the immune system.

TScan Therapeutics, Inc., a preclinical-stage biopharmaceutical company, focuses on developing a pipeline of T cell receptor-engineered T cell, or TCR-T, therapies for the treatment of patients with cancer. It is developing TSC-100 and TSC-101 for the treatment of patients with hematologic malignancies to eliminate residual leukemia and prevent relapse after hematopoietic stem cell transplantation; and TSC-200, TSC-201, TSC-202, and TSC-203 for the treatment of solid tumors. The company has a collaboration and license agreement with Novartis Institutes for Biomedical Research, Inc. to discover and develop novel TCR-T therapies. TScan Therapeutics, Inc. was incorporated in 2018 and is headquartered in Waltham, Massachusetts.

The vast majority of human disease is governed by the epigenome, the subtle machinery that controls the volume of gene expression in cells. Tune Therapeutics is pioneering the creation of epi-therapeutic medicines with its powerful and precise epigenomic control platform, TEMPO. Unlike genome editing, TEMPO dials the volume of gene expression up or down towards healthy levels, without breaking or permanently rewriting DNA – and it can do this in multiple genes at once. This genetic tuning approach allows us to change cell fate and function at will, unlocking the ability to reverse pathways of cancer, genetic disease and aging. Our approach effectively expands the potential reach of genetic medicine – from targeting a limited range of rare conditions, to overcoming thousands of complex and chronic diseases that currently have no curative treatment.

Turnstone Biologics is a clinical-stage biotechnology company developing new medicines to treat and cure solid tumors by pioneering a differentiated approach to TIL therapy. Our innovative TIL therapy is based upon the identification, selection, and expansion of the most potent tumor-reactive T cells, known as Selected TILs, and is designed to overcome the limitations of first-generation bulk TILs that have demonstrated objective responses only in limited tumor types. Our most advanced program, TIDAL-01, is currently being evaluated in two Phase 1 studies in patients with melanoma, breast cancer, and colorectal cancer. We are also actively advancing a pipeline of preclinical programs, including TIDAL-02, our next Selected TIL program, and our TIDAL-01 and viral immunotherapy combination program.

Umoja is developing a new approach to cancer therapy that retools a patient’s immune system in vivo, enhancing the body’s natural capacity to fight cancer. We envision a world where all patients with cancer have access to the most advanced immunotherapies and are free from the burdens of traditional cancer therapies.

Valo Therapeutics (ValoTx) is a developer of novel immunotherapies in cancer and infectious disease. Our unique approach to immunotherapy, based on oncolytic viruses and viral vectors combined with disease-specific peptides, delivers highly adaptable and immunogenic therapeutic vaccination platforms. A spin-out company from the University of Helsinki, Finland, Valo Tx has assembled a talented team of immunotherapy experts, who together with the founding scientists have the necessary expertise to take its patented technology through clinical development and make it available to patients.

Verismo Therapeutics is pioneering the development of novel chimeric antigen receptors (CARs) that enhance T-cell-based cellular immunotherapy for cancer, especially non-hematologic malignancies with few effective therapeutic options. Verismo Therapeutic’s core technology is built upon CARs that are uniquely engineered to more realistically reflect the natural multichain design of killer immunoglobulin-like receptors (KIRs), an important family of immunologic receptors used by T cells and NK cells. Preclinical studies show that SynKIR™-T cells, created by introducing these KIR-based CARs (KIR-CARs) into T cells, more effectively eliminate aggressive tumors in vivo when compared with T cells bearing traditional 2nd generation CAR designs found in current FDA approved CAR-T cell therapies.

Verve Therapeutics, Inc., a genetic medicines company, engages in developing gene editing medicines for patients to treat cardiovascular diseases. Its lead product candidate is VERVE-101, a single-course gene editing treatment that permanently turns off the PCSK9 gene in the liver. The company also engages in the development ANGPTL3 program to permanently turn off the ANGPTL3 gene in the liver. It has a collaboration and license agreement with Beam Therapeutics; a development and option agreement with Acuitas Therapeutics, Inc.; and a Cas9 license agreement with The Broad Institute and the President and Fellows of Harvard College. The company was formerly known as Endcadia, Inc. and changed its name to Verve Therapeutics, Inc. in January 2019. Verve Therapeutics, Inc. was incorporated in 2018 and is based in Cambridge, Massachusetts.

Xalud Therapeutics is a biotechnology company developing a non-viral gene therapy platform to treat pathologic inflammation through immune modulation. The company is harnessing the power of interleukin-10 (IL-10), a potent cytokine that acts as a master regulator for multiple inflammatory pathways, to address the root cause of inflammation and subsequently restore homeostasis in the immune system. Xalud’s lead product candidate, XT-150, is a locally injectable plasmid DNA gene therapy expressing IL-10 to addresses pathologic inflammation and pain.