List of Adeno-associated viral vector (AAV) Companies with Phase 3 Active Clinical Trial - 30

4DMT is a clinical stage biopharma company inventing and developing innovative products to unlock the full potential of genetic medicine to treat large market and rare diseases. Our vector discovery platform Therapeutic Vector Evolution harnesses the power of directed evolution, a Nobel Prize-winning technology, with approximately one billion synthetic AAV capsid-derived sequences to invent customized and evolved vectors with improved therapeutic profiles compared to wild-type vectors. Our product design, development and manufacturing engine empowers us to efficiently create our valuable and diverse product pipeline. This combination of bold innovation and relentless execution gives 4DMT the capability to revolutionize genetic medicines and to strive for potential curative therapies. We have built a deep portfolio of genetic medicine product candidates, with five product candidates in clinical trials for seven patient populations. We are taking genetic medicine to new heights and into new disease areas to overcome challenges not addressed by existing medicines. This incredible challenge drives our relentless and talented team. Our people are the essence of our inspiring, innovative and collaborative environment that encourages individual contributions and provides opportunities for career development. We seek the best in the industry who identify with our mission and our bold and innovative results-driven approach. Our 4 Guiding Principles drive the way we work together: - Dare to Cure - Break Boundaries - Beyond Yourself - Prepare & Execute Relentlessly Join us in creating the future of genetic medicine.

Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing cell and gene therapies for serious diseases. Prademagene zamikeracel (pz-cel) is Abeona’s investigational autologous, COL7A1 gene-corrected epidermal sheets currently in development for recessive dystrophic epidermolysis bullosa. The Company’s fully integrated cell and gene therapy cGMP manufacturing facility served as the manufacturing site for pz-cel used in its Phase 3 VIITAL™ trial, and is capable of supporting commercial production of pz-cel upon FDA approval. The Company’s development portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical need. Abeona’s novel, next-generation AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases.

Adverum Biotechnologies, Inc., a clinical-stage gene therapy company, engages in developing gene therapy product candidates to treat ocular and rare diseases. Its pipeline of product candidates includes ADVM-022, an adeno- associated virus (AAV). 7m8-aflibercept for the treatment of wet age-related macular degeneration; ADVM-043, an investigational gene therapy candidate for the treatment of alpha-1 antitrypsin deficiency; and ADVM-053, a preclinical gene therapy product candidate for the treatment of hereditary angioedema. The company has collaboration agreements with Editas Medicine, Inc. to leverage its proprietary AAV vectors for genome editing technologies to treat up to five inherited retinal diseases; and Regeneron Pharmaceuticals, Inc. for the development of up to eight distinct ocular therapeutic targets. The company was formerly known as Avalanche Biotechnologies, Inc. and changed its name to Adverum Biotechnologies, Inc. in May 2016. Adverum Biotechnologies, Inc. was founded in 2006 and is headquartered in Redwood City, California.

Amicus Therapeutics, Inc., a biotechnology company, engages in the discovery, development, and commercialization of therapies to treat a range of rare and orphan diseases. The company offers Galafold, an orally administered small molecule pharmacological chaperone for the treatment of Fabry disease. It is also conducting Phase 3 (ATB200-03) clinical study of AT-GAA for Pompe disease. The company has collaboration and license agreements with Nationwide Children's Hospital and University of Pennsylvania. Amicus Therapeutics, Inc. was founded in 2002 and is headquartered in Philadelphia, Pennsylvania.

Beacon Therapeutics is an ophthalmic gene therapy company founded in 2023 to restore and improve the vision of patients with a range of prevalent and rare retinal diseases that result in blindness. The Company has an established scientific foundation that combines a late-stage development candidate to treat X-linked retinitis pigmentosa (XLRP), as well as two preclinical programs, one targeting dry age-related macular degeneration (AMD) and another in-licensed from the University of Oxford targeting cone-rod dystrophy (CRD), an inherited retinal disease. Lead development candidate AGTC-501, is a gene therapy program in Phase II clinical trials for the treatment of XLRP, an inherited monogenic recessive disorder that causes progressive vision loss in boys and young men. XLRP is predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. AGTC-501 expresses the full length RPGR protein, thereby addressing the full complement of photoreceptor damage caused by XLRP, including both rod and cone loss. Beacon Therapeutics also has access to a target generation technology platform that will identify, screen, and search secreted proteins in the ophthalmology space. The Company is supported by funds from Syncona and additional investors including Oxford Science Enterprises.

BioNTech is a global next generation immunotherapy company aspiring to translate science into survival. Around 6.100 pioneers currently work with us, employing a wide array of computational discovery and therapeutic drug platforms for the rapid development of novel biopharmaceuticals. Our broad portfolio of oncology product candidates includes individualized and off-the-shelf mRNA-based therapies, innovative chimeric antigen receptor (CAR) T cells, several protein-based therapeutics, including bispecific immune checkpoint modulators, targeted cancer antibodies and antibody-drug conjugate (ADC) therapeutics, as well as small molecules.   Based on our deep expertise in mRNA vaccine development and in-house manufacturing capabilities, we are developing multiple mRNA vaccine candidates for a range of infectious diseases alongside its diverse oncology pipeline. To learn more about us, please visit www.BioNTech.com. Our privacy statement can be found here:  https://www.biontech.com/int/en/home/data-privacy-statement.html Our imprint can be found: https://www.biontech.com/int/en/home/imprint.html

At Candel Therapeutics, we are developing immunotherapies that induce immunogenic cell death in cancer cells at the site of injection, unmasking tumor neo-antigens within an activated microenvironment. This process leads to a systemic, durable immune response against the tumor, with the potential to change disease outcomes across a variety of indications. Candel’s products are designed to improve survival while maintaining quality of life – from early- to late-stage disease.

Cartesian Therapeutics is a fully integrated, clinical-stage biopharmaceutical company developing novel cell and gene immunotherapies to treat cancer and autoimmune diseases.

CG Oncology, Inc. (Nasdaq: CGON), is a late-stage clinical biopharmaceutical company focused on developing and commercializing a potential backbone bladder-sparing therapeutic for patients afflicted with bladder cancer. We see a world where urologic cancer patients can benefit from our innovative oncolytic immunotherapies to live with dignity and have an enhanced quality of life. Our lead candidate, cretostimogene grenadenorepvec, is an intravesically delivered oncolytic immunotherapy agent in a Phase 3 trial for the treatment of BCG-unresponsive non-muscle invasive bladder cancer. Cretostimogene grenadenorepvec is also in a Phase 2 study in combination with KEYTRUDA® (pembrolizumab) in the same indication. Other types of bladder cancer are being evaluated with cretostimogene grenadenorepvec in combination with OPDIVO® (nivolumab).

Complement Therapeutics 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.

CRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom.

Elpida Therapeutics is a biopharmaceutical company that focuses on developing treatments for rare diseases, offering hope to children and families affected.

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

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.

Gritgen Therapeutics (here in after designated as "Gritgen") was founded in China and is a biotechnology company focused on gene therapy. They have launched a commercial GMP facility in China and have opened a good manufacturing practice (GMP) facility in Suzhou Industrial Park. Gritgen provides an important vector foundation for the company to realize gene therapy for hemophilia A. The company has also launched a Phase 1 clinical trial to assess the safety of gene therapy for hemophilia A.

ImmunityBio (previously NantKwest (NASDAQ: NK)) is a pioneering, next generation, clinical-stage immunotherapy company focused on harnessing the unique power of our immune system using natural killer (NK) cells to treat cancer, infectious diseases and inflammatory diseases. NK cells are the body’s first line of defense due to the innate ability of NK cells to rapidly identify and destroy cells under stress, such as cancer or virally-infected cells. ImmunityBio is uniquely positioned to implement precision cancer medicine, with the potential to change the current paradigm of cancer care. Natural Killer cells are ancient cells in the human body designed to recognize and detect cells under stress or infected. Our “off-the-shelf” activated Natural Killer (NK) platform have the capacity to destroy cancer and virally infected cells from the body. The safety of our NK cells as well as their activity against a broad range of cancers have been tested in multiple phase 1 clinical trials in the United States, Canada and Europe. In addition to our NK cells capability to be administered in the outpatient setting as an “off-the-shelf” living drug, it serves as a universal cell-based therapy without need for individualized patient matching. Moreover, our NK cell based platform has been bioengineered to incorporate chimeric antigen receptors (CARs) and antibody receptors to further optimize targeting and potency in the therapeutic disease. Our targeted therapeutic areas include: (1) cancer, focusing on bulky hematological cancers and solid tumors as well as cancer stem cells, (2) infectious diseases, including viral, fungal and bacterial infections, and (3) inflammatory diseases, ranging from rare inherited diseases to more prevalent autoimmune disorders.

Intellia Therapeutics is a leading genome editing company whose mission is to develop potentially curative gene editing treatments that can positively transform the lives of people living with severe and life-threatening diseases. We are focused on the development of proprietary therapeutics using a recently developed biological tool known as the CRISPR/Cas9 system. The promise of the CRISPR/Cas9 system is the driving force behind the creation of Intellia. Our founders have a shared belief that the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course. This technology offers the potential for us to develop curative therapeutic options for patients with chronic diseases by addressing the underlying cause of the disease. It can bring new hope and cures to people who now have nowhere to turn for help. Our combination of deep scientific expertise and clinical development experience, along with our leading intellectual property portfolio, puts Intellia in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Intellia has the right people, assets and ambitious vision needed to take full advantage of the CRISPR/Cas9 technology’s attributes – high potency, specificity, simplicity of use, broad applicability, and multifunctional programmability – and accelerate the advancement of curative products into the clinic. View our social media community guidelines at https://www.intelliatx.com/terms-and-conditions/ .

Ionis Pharmaceuticals, Inc. discovers and develops RNA-targeted therapeutics in the United States. The company offers SPINRAZA for spinal muscular atrophy (SMA) in pediatric and adult patients; TEGSEDI, an injection for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults; and WAYLIVRA, a treatment for familial chylomicronemia syndrome and familial partial lipodystrophy. It is involved in developing neurology products that include Tominersen for Huntington’s diseases; Tofersen for amyotrophic lateral sclerosis (ALS); IONIS-MAPTRx for Alzheimer's diseases; IONIS-C9Rx for genetic form of ALS; ION859 for Parkinson’s disease; ION464 for multiple system atrophy and Parkinson’s disease; ION541 for amyotrophic lateral sclerosis; and IONIS-DNM2-2.5Rx for centronuclear myopathy. The company is also developing products for cardiometabolic disease, such as IONIS-TTR-LRx for transthyretin amyloidosis and IONIS-GHR-LRx for acromegaly; IONIS-APOCIII-LRx for familial chylomicronemia syndrome and TG- driven diseases; IONIS-APOCIII-LRx for treatment resistant hypertension; Pelacarsen for Lp(a)cardiovascular disease risk reduction; Vupanorsen for sHTG/CVDRR; IONIS-FXI-LRx for clotting disorders; ION449 for cardiovascular diseases; and IONIS-GCGRRx for diabetics. In addition, it develops IONIS-ENAC-2.5Rx for cystic fibrosis and chronic obstructive pulmonary disease; IONIS-PKK-LRx for hereditary angioedema; Danvatirsen for cancer; IONIS-TMPRSS6-LRx for beta-thalassemia and iron toxicity; IONIS-HBVRx for hepatitis B virus; IONIS-AR-2.5Rx for prostate cancer; IONIS-FB-LRx for age-related macular degeneration and IgA nephropathy; and ION357 for retinitis pigmentosa. It has a collaboration agreement with AstraZeneca; GlaxoSmithKline plc; Janssen Biotech, Inc.; Bayer AG; Novartis AG; Pfizer, Inc.; Biogen Inc.; and Roche. Ionis Pharmaceuticals, Inc. was founded in 1989 and is based in Carlsbad, California.

At Kite, a Gilead Company, our singular focus is cell therapy to treat and potentially cure cancer. Our goal is to bring the promise of cell therapy to as many eligible patients as possible who may benefit. Since 2009, Kite has been at the forefront of cancer immunotherapy. Today, we are proud to be the leader in engineered T cell therapy for hematologic malignancies, but we recognize that this is just the beginning. Patients are at the heart of our focus on the cure, which is why we are committed to pursuing cell therapy as a transformative treatment option for as many people with cancer as possible who may benefit. Cancer is personal to us. Unlike traditional pharmaceutical manufacturing, our therapies are not created on an assembly line. Cell therapy specialists at our manufacturing facilities reengineer a patient’s own immune cells to attack and fight their cancer cells — one patient at a time. That sense of connection to the people we serve motivates us daily at Kite. Our corporate headquarters are located in Santa Monica, California, and we have dedicated cell therapy manufacturing facilities in California, Maryland and the Netherlands. If you’re passionate about changing the future of cancer treatment, let’s talk.

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.

Ocugen, Inc. is a biopharmaceutical company focused on discovering, developing, and commercializing gene therapies to cure blindness diseases and developing a vaccine to save lives from COVID-19. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with one drug – “one to many” and our novel biologic product candidate aims to offer better therapy to patients with underserved diseases such as wet age-related macular degeneration, diabetic macular edema, and diabetic retinopathy. We are co-developing Bharat Biotech’s COVAXIN™ vaccine candidate for COVID-19 in the U.S. market.

Pacira BioSciences Inc. is a leading provider of non-opioid pain management and regenerative health solutions. We are dedicated to offering patients—and the clinicians who treat them—cutting-edge therapies to treat pain with the potential to reduce or eliminate the need for opioids. Founded in 2006 and headquartered in Tampa, FL, Pacira has offices in Parsippany, NJ as well as San Diego, CA. With more than 700 employees who share a passion for delivering improved patient care and reducing the need for opioids, Pacira is here to challenge and transform the way we think about – and treat – pain. We are committed to providing solutions that address unmet medical need and improve clinical results, and empowering patients and healthcare providers to choose non-opioid options. Our non-opioid product portfolio is aimed at opioid minimization, long-lasting pain control, and enhanced recovery. Find out why we aim for change at Pacira.com/commitments

Precigen (Nasdaq: PGEN) is a dedicated discovery and clinical stage biopharmaceutical company advancing the next generation of gene and cell therapies using precision technology to target the most urgent and intractable diseases in immuno-oncology, autoimmune disorders, and infectious diseases. Our technologies enable us to find innovative solutions for affordable biotherapeutics in a controlled manner. Precigen operates as an innovation engine progressing a preclinical and clinical pipeline of well-differentiated unique therapies toward clinical proof-of-concept and commercialization.

Priothera - We are dedicated to improving the lives of patients suffering from hematological malignancies by delivering an innovative immune modulator to enhance the curative potential of allogeneic Hematopoietic Stem Cell Transplantation (HSCT)

PTC Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of medicines for the treatment of rare disorders. The company offers Translarna (ataluren) and Emflaza (deflazacort) for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients. It is developing Translarna, which is in Phase 2 clinical study for the treatment of nonsense mutation aniridia and nonsense mutation Dravet syndrome/CDKL5; and RG7916 and RO7034067 for the treatment of patients with spinal muscular atrophy, as well as PTC596 and PTC299, a small molecule dihydrooratate dehydrogenase (DHODH) inhibitor that inhibits de novo pyrimidine nucleotide synthesis, which is in Phase 1 clinical development stage to treat cancer patients. The company is also developing gene therapy product candidate that include PTC-AADC for the treatment of Aromatic L-amino acid decarboxylase deficiency. It has collaborations with F. Hoffman-La Roche Ltd and Hoffman-La Roche Inc., and the Spinal Muscular Atrophy Foundation to develop and commercialize compounds identified under its spinal muscular atrophy sponsored research program; research collaboration with Massachusetts General Hospital for the treatment of rare genetic disorders resulting from pre-mRNA; and Akcea Therapeutics, Inc. to commercialize Tegsedi and Waylivra for the treatment of rare diseases in Latin America and the Caribbean. PTC Therapeutics, Inc. was founded in 1998 and is headquartered in South Plainfield, New Jersey.

Sarepta Therapeutics, headquartered in Cambridge, Massachusetts, is a global biotechnology company on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development. Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing. For information on our Community Guidelines, please visit sarepta.com/community-guidelines. We want to share a reminder with all job seekers and candidates regarding the persistence of recruiting fraud. Please read a message about recruiting fraud and steps you can take to protect yourself here: https://www.sarepta.com/recruiting-fraud

Thermo Fisher Scientific Inc. is the world leader in serving science, with annual revenue of approximately $40 billion. Our Mission is to enable our customers to make the world healthier, cleaner and safer. Whether our customers are accelerating life sciences research, solving complex analytical challenges, increasing productivity in their laboratories, improving patient health through diagnostics or the development and manufacture of life-changing therapies, we are here to support them. Our global team delivers an unrivaled combination of innovative technologies, purchasing convenience and pharmaceutical services through our industry-leading brands, including Thermo Scientific, Applied Biosystems, Invitrogen, Fisher Scientific, Unity Lab Services, Patheon and PPD.

Leading with purpose. Every day. “Ultragenyx was founded to advance innovative medicines for rare and ultrarare diseases that have never been treated before. We are delivering transformative therapies across multiple indications, and we have one of the most robust and diverse clinical pipelines in rare disease. Our focus is on doing the right things for patients both during development and commercialization to deliver on the promise of these therapies in a way that's meaningful for rare disease communities.” Emil D. Kakkis, M.D., Ph.D. Chief Executive Officer and President We have an inclusive culture of value and respect. Ultragenyx brings novel products to those living with rare and ultrarare diseases with a focus on debilitating genetic diseases. Founded in 2010, we have rapidly built a diverse portfolio of approved therapies and products aimed at diseases with a high unmet medical need for which many have no approved therapies. It takes courage, care, talent, and dedication to make a meaningful impact for those living with rare diseases, their families and the rare disease community. We put our people first, so they can take care of the rare disease community. We purposefully cultivate and nurture a dynamic, supportive work environment where we encourage every team member to share their new ideas and help us unlock more possibilities. Our team is made up of exceptional and diverse people with a strong passion and commitment to helping those living with rare diseases. By embracing generosity, curiosity, inclusion, and humility we are constantly learning together—fostering an environment that supports profound growth and fulfillment. If you want to have a meaningful impact, do the best work of your career while having fun and growing professionally and personally, come join our team! Visit our community guidelines at: https://ultragenyx.co/community