List of Adeno-associated viral vector (AAV) Companies in France - 17

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

BrainEver is a biopharmaceutical company developing a pipeline of novel therapies for patients with age-related neurodegenerative diseases, such as Charcot disease, Parkinson’s, and glaucoma, the second leading cause of blindness. Our mission is to develop recombinant human Homeoproteins (HPs) to halt disease progression by stopping neuronal loss and durably restoring the physiology and metabolism of the remaining neurons. BrainEver was co-founded by Bernard Gilly (iBionext) and Alain Prochiantz (Collège de France). Our innovative therapeutic approach stems from the pioneering work of Alain Prochiantz and his team on brain development and physiology. Our lead project, BREN02, focuses on developing recombinant human Engrailed-1 (hEN1) for the treatment of patients with Amyotrophic Lateral Sclerosis (ALS). Building on the highly promising results of our preclinical studies, we are preparing to launch clinical trials by 2025. To fund this next phase and secure market approval by 2027, we are opening our capital to those eager to join a unique human and scientific journey. We are supported by prominent investors such as Bpifrance, Ibionext, and Turenne Capital. Now, we aim to raise €35M, with €5M available for participation through the crowdfunding platform Tudigo. Become a BrainEver shareholder 👉 https://tudigo.typeform.com/brainever

CAPACITÉS is the subsidiary of University of Nantes, dedicated to the development of research. Specialized in engineering of innovative projects, CAPACITÉS exploits the latest scientific discoveries necessary for the performance of its customers. Springboard to differentiate in the ever-changing markets, areas of activity extend to technological and engineering sciences, human and social sciences and health. The activities of CAPACITÉS have advanced expertise on specific and cross-cutting themes. From idea generation to their implementation, CAPACITÉS impulse with its customers a strong value creation process through the development of R&D of high level dedicated to nine economic sectors.

Cellectis is a global clinical-stage biopharmaceutical company. Pioneers and innovators in our field, our mission is to develop innovative treatments for patients with unmet medical needs. With 25 years of expertise, we have the best-in-class gene editing platform focusing on immuno-oncology, and gene therapy. Through our efficient and precise TALEN® technology, we create allogeneic CAR-T cells capable of recognizing and combating cancer cells. Today, our three clinical programs target patients with B-cell acute lymphoblastic leukemia (B-ALL), non-Hodgkin lymphoma (NHL) and acute myeloid leukemia (AML). We are fully integrated and a leader in end-to-end gene editing, allogeneic CAR T-cell companies. With our in-house manufacturing, we control our gene and cell therapy process from start to finish with starting materials produced in Paris (France) and CAR-T therapy products created in Raleigh, NC (USA). We also have several ongoing strong collaborations, based on our TALEN® technology, with leading cell & gene therapy companies, including our recent partnership with AstraZeneca, to develop new product candidates in oncology, immunology, and treatment of rare diseases. At Cellectis, we are committed to a cure. Cellectis headquarters are in Paris, France, with additional locations in New York, New York and Raleigh, North Carolina. Cellectis is listed on the Nasdaq Global Market (ticker: CLLS) and on Euronext Growth (ticker: ALCLS). To find out more about us, visit our website: www.cellectis.com. To learn more about our community guidelines, visit: http://www.cellectis.com/en/social-media-guidelines/ Follow our other social media accounts: @cellectis on LinkedIn on X (formerly Twitter) . TALEN® is a registered trademark owned by the Cellectis Group.

Coave Therapeutics is a clinical stage biotechnology company focused on developing life changing gene therapies in rare Ocular and CNS (Central Nervous System) diseases. We are pushing the boundaries of gene therapy through application of our AAV-Ligand Conjugates platform (ALIGATER) that enables targeted delivery, enhanced gene transduction and tissue distribution to improve the effectiveness of advanced gene therapies for rare diseases. Our strategy is to advance a pipeline of novel therapies targeting rare ocular and CNS diseases where targeted gene therapy has the potential to be most effective, where there is a clear unmet need and where success will provide rapid validation of our ALIGATER platform.

Created in 1990 and funded by AFM-TELETHON, Genethon, a pioneer in deciphering the human genome and identifying the genes associated with genetic diseases is today fully dedicated to the design and development of gene therapy treatments for rare diseases. At Genethon more than 180 scientists, clinicians and engineers specialized in gene therapy drug development, from research to the clinic, have joined forces in order to provide these innovative treatments to patients affected with rare disorders. These treatments also pave the way for new therapeutic approaches for frequent disorders.

GenoSafe is a CRO providing analytical testing services for the evaluation of quality, safety and efficacy of Gene and Cell therapy candidates including : - GLP biodistribution, shedding and immunogenicity studies ; - QC testing, such as viral titration, safety and potency/efficacy testing ; - GCP bioanalysis of patient samples, including immunogenicity, shedding and gene expression studies. GenoSafe brings more than 15 years of experience in the development, qualification and validation of key analytical methods for product and sample testing.

GenSight Biologics is a clinical-stage biotechnology company discovering and developing novel therapies for neurodegenerative retinal diseases and diseases of the central nervous system. GenSight Biologics’ pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics for retinitis pigmentosa, to help preserve or restore vision in patients suffering from severe degenerative retinal diseases. GenSight Biologics’ lead product candidate, GS010, is in Phase III trials in Leber’s Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible low vision and legal blindness in teens and young adults. Using its gene therapy-based approach, GenSight Biologics’ product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery.

Created in 2005 through a collaboration between Inserm – Institut National Health and Medical Research – and AFM-Telethon – French Association against Myopathies – I-Stem is the largest French laboratory research and development dedicated to human pluripotent stem cells, embryonic origin or obtained by reprogramming gene. I-Stem is part of the Biotherapy Institute for Rare Diseases, which includes so far the four centers of research and development funded directly by the AFM Telethon. The specific vocation of I-Stem is to explore all the therapeutic potential of human pluripotent stem cells for applications in patients affected by rare diseases of genetic origin. In this context, our teams are developing two major areas of research. The first one is cell therapy, which aims to replace lost or diseased cells to other cells with the same characteristics, produced in the laboratory from pluripotent stem cells. The second area is pharmacology based on automated screening of large libraries of compounds with therapeutic potential, following modeling of molecular mechanisms associated with diseases, as revealed by the study of pluripotent stem cells from affected donors. I-Stem teams are currently working on a dozen genetic diseases that affect different organs. The Institute also hosts every year many researchers interested in other diseases and provides training and technology support.

Nantes Université is a Higher Education institution that offers academic programs and conducts research.

Polyplus, part of Sartorius, is a leading upstream solutions provider for advanced biologic and cell and gene therapy production from research to commercial scale. An innovator in nucleic acid delivery, the legacy portfolio features process-centric transfection reagents, kits, and support services for viral and non-viral delivery. Custom plasmid vector design and plasmid and protein manufacturing was integrated into the offer in 2022 to expand the products and services portfolio to help the industry optimize process economics while meeting strict scientific and regulatory standards.

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)

Sanofi, together with its subsidiaries, engages in the research, development, manufacture, and marketing of therapeutic solutions in the United States, Europe, and internationally. It operates through three segments: Pharmaceuticals, Vaccines, and Consumer Healthcare. The company provides specialty care products, including human monoclonal antibodies; products for multiple sclerosis, neurology, other inflammatory diseases, immunology, rare diseases, oncology, and rare blood disorders; medicines for diabetes; and cardiovascular and established prescription products. It also supplies poliomyelitis, pertussis, and hib pediatric vaccines; and influenza, adult booster, meningitis, and travel and endemic vaccines. In addition, the company offers allergy, cough and cold, pain, liver care, physical and mental wellness, probiotics, digestive, and nutritional products; and other products, such as daily body lotions, anti-itch products, moisturizing and soothing lotions, and body and foot creams, as well as powders for eczema. Further, it has various pharmaceutical products and vaccines in development stage. Sanofi has collaboration agreement with GlaxoSmithKline to develop a recombinant Covid-19 vaccine; and a research collaboration with Stanford University School of Medicine to advance the understanding of immunology and inflammation through open scientific exchange. It also has a collaboration and license option agreement with Prellis Biologics, Inc. The company was formerly known as Sanofi-Aventis and changed its name to Sanofi in May 2011. Sanofi was founded in 1973 and is headquartered in Paris, France.

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

SparingVision is a genomic medicines company, translating pioneering science into vision-saving treatments. Founded to advance over 20 years of world-leading ophthalmic research from its scientific founders at the Paris Vision Institute, SparingVision is leading a step shift in how ocular diseases are treated, moving beyond single gene correction therapies. At the heart of this is a pipeline of gene independent treatments for rod-cone dystrophies. Lead products, SPVN06 and SPVN20, address mid and late stages of retinitis pigmentosa (RP)respectively. RP is the most common inherited retinal disease affecting two million people worldwide. These novel medicines could form the basis of a suite of new sight-saving treatments with potential applications across many other retinal diseases, regardless of genetic cause. The Company is supported by a strong, internationally renowned team who aim to harness the potential of genomic medicine to deliver new treatments to all ocular disease patients as quickly as possible. SparingVision has raised €60 million to date and its investors include 4BIO Capital, Advent France Biotechnology, Bpifrance, Foundation Fighting Blindness (US), Fondation Voir & Entendre, UPMC Enterprises, Jeito Capital and Ysios Capital. For more information, please visit www.sparingvision.com.

Vivet Therapeutics is dedicated to developing innovative gene therapy treatments for orphan diseases. Vivet Therapeutics is focused on optimizing gene therapy through collaborating with the Fundacion para la Investigacion Medica Aplicada (CIMA, Universidad de Navarra) to develop new AAV vectors specifically targeting the liver and generating new technologies to optimize gene delivery and long term expression.

Founded in 2019, backed by Y-Combinator, WhiteLab Genomics stands at the convergence of computer sciences and biology, pioneering the accelerated development of genomic medicines. By leveraging their proprietary technology, WhiteLab Genomics analyzes complex biological data powered by AI to significantly reduce development timelines and mitigate associated risks. Based on exhaustive datasets, the platform provides in-silico simulations to discover, and design optimized payloads and vectors. WhiteLab aims to expedite the drug development process, cut costs, and accelerate the delivery of life-saving therapies to the market. The company collaborates with leading pharmaceutical companies such as Sanofi, academic institutions, and innovative biotechnology companies. Recognized for their contribution to advancing the field of gene and cell therapy, WhiteLab Genomics is part of the prestigious French government supported by French Tech 2030 program and recently joined the Bayer Co.Lab in Cambridge, MA, and Ginkgo Bioworks' Technology Network, marking major milestones in our international expansion.