List of Adeno-associated viral vector (AAV) Companies with Phase 1 Active Clinical Trial - 26

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.

Akamis is a clinical-stage oncology company whose mission is to leverage its groundbreaking T-SIGn® platform to positively impact the lives of people living with cancer. We are developing a portfolio of solid tumor-targeted T-SIGn® therapeutics which aim to enable a patient’s own immune system to recognize, attack, and clear their cancer. Akamis’ growing pipeline of T-SIGn® therapeutics is anchored by its lead clinical-stage program, NG-350A, which is being investigated in an ongoing Phase 1 clinical study in patients with metastatic or advanced epithelial tumors. Akamis has a number of T-SIGn® platform-focused collaborations with leaders in the immuno-oncology field including BMS, Merck, and the Parker Institute for Cancer Immunotherapy (PICI)

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).

Caribou Biosciences, Inc., a clinical-stage biopharmaceutical company, engages in the development of genome-edited allogeneic cell therapies for the treatment of hematologic malignancies and solid tumors in the United States and internationally. Its lead product candidates are CB-010, an allogeneic anti-CD19 CAR-T cell therapy that is in phase 1 clinical trial to treat relapsed or refractory B cell non-Hodgkin lymphoma; and CB-011, an allogeneic anti-BCMA CAR-T cell therapy for the treatment of relapsed or refractory multiple myeloma. The company also develops CB-012, an allogeneic anti-CD371 CAR-T cell therapy for the treatment of relapsed or refractory acute myeloid leukemia; and CB-020, an allogeneic CAR-NK cell therapy for the treatment of solid tumors. Caribou Biosciences has collaboration with AbbVie Manufacturing Management Unlimited Company to develop CAR-T cell therapies. The company was incorporated in 2011 and is headquartered in Berkeley, California.

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.

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.

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

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.

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.

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.

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.

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.

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

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.

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.

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.

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.

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.

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.

At Virion Therapeutics, We Believe a Future Free of Cancer and Chronic Infectious Diseases Is Within Reach​ Driven by science, our mission is to revolutionize the immunotherapy treatment landscape, focusing on the development of novel, adaptable, and accessible CD8+ T cell-based technologies.​ Our transformative platform is first in its class, offering a unique methodology that combines innovative and established approaches to elicit a superior CD8+ T cell response. Novel genetically encoded checkpoint modifiers are delivered alongside optimized target-specific antigens via an immunogenic and adaptable viral vector platform that promotes potent, prolonged, and broad CD8+ T cell responses. With potential applications across a range of cancers and chronic infectious diseases, our proprietary technology provides us with a robust pipeline. Our lead product, VRON-0200, is under investigation as a potential functional cure for the treatment of chronic hepatitis B virus (HBV) – a first-in-human clinical study is planned to initiate in late 2022. Founded in 2018 to advance technology licensed from The Wistar Institute, we have assembled a world-class management team and an industry-leading global Board of Directors, with proven experience in the biotechnology sector, as well as extensive preclinical and clinical expertise in antiviral, vaccine, oncology, and immuno-oncology.​