List of Gene Therapy Companies in France - 41

🚀 Aenitis designs, develops, and markets sole-in-class acoustofluidic technology that makes cell and gene therapy manufacturing better, safer, and less expensive. By precisely controlling levitated cells with sound waves, our systems handle cells in full sterile conditions and radically increase productivity while preserving the native state of cells at every step of the engineering process. This technology enables gentle, non-invasive manipulation of cells at the millifluidic scale, allowing for the handling of tens of milliliters per minute. Such capabilities are critical for scaling applications from research to clinical and industrial use. 💡Today, we have achieved proof-of-concepts for our contact-free & label-free proprietary technology across multiple cell types & processes. Mitis™, our flagship product, is already used for a wide range of applications, including cell sorting, isolation, concentration & washing, from research to clinical and industrial scale. 🌍Co-founded by pioneers of acoustic levitation & fluid mechanics and a biotech entrepreneur, the company benefits from the support of the European Commission & the French Government and from the commitment of leading investors, notably the European Investment Bank and Seventure Partners.

We build high tech equipment supporting end-to-end development and manufacture of cell and gene therapies.

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

Brink Therapeutics is developing next-generation recombinases — ultra-precise and efficient enzymes that surpass CRISPR-Cas9. We aim to revolutionize Cell and Gene therapies, enabling large-scale production to treat cancer, genetic disorders, and other diseases.

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.

CERES BRAIN Therapeutics is dedicated to treat cerebral diseases with a first focus on rescuing suffering neurons with creatine through an innovative "creatine-to-brain" concept. The first indications are Creatine Transporter Deficiency syndrome, a disabling condition with no treatment affecting children and Amyotrophic Lateral Sclerosis. The lead compound CBT101 demonstrated efficacy in a preclinical models models as well as a large cerebral and neuronal distribution in primate. The CMC reached noticeable milestones with several GMP batches produced. Other mitochondriopathies are in the Ceres's pipeline. CERES BRAIN Therapeutics has already raised up to 12M€ to move towards the clinical program.

Clean Cells is a GMP-licensed Contract Development and Manufacturing Organization providing R&D, production and quality control testing services to the biopharmaceutical industry. Our two-part site located in Western France holds an innovative process development platform, BSL2 and BSL3 GMP manufacturing laboratories and analytical areas to develop bespoke assays and perform GMP testing. Clean Cells is part of the Clean Biologics group and benefits from the additional experience of sister company Naobios, a CDMO with considerable experience in the development and manufacture of virus-based products.

Clean Cells is a GMP-licensed Contract Development and Manufacturing Organization providing R&D, production and quality control testing services to the biopharmaceutical industry. Our two-part site located in Western France holds an innovative process development platform, BSL2 and BSL3 GMP manufacturing laboratories and analytical areas to develop bespoke assays and perform GMP testing. Clean Cells is part of the Clean Biologics group and benefits from the additional experience of sister company Naobios, a CDMO with considerable experience in the development and manufacture of virus-based products.

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.

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

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.

Innate Pharma S.A. is a commercial stage oncology-focused biotech company dedicated to improving treatment and clinical outcomes for patients through therapeutic antibodies that harness the immune system to fight cancer. Innate Pharma’s commercial-stage product, Lumoxiti, in-licensed from AstraZeneca, was approved by the FDA in September 2018. Lumoxiti is a first-in class specialty oncology product for hairy cell leukemia (HCL). Innate Pharma’s broad pipeline of antibodies includes several potentially first-in-class clinical and preclinical candidates in cancers with high unmet medical need. Pioneers in the biology of Natural Killer cell, Innate Pharma has expanded its expertise in the tumor microenvironment and tumor-antigens, as well as antibody engineering. This innovative approach has resulted in a diversified proprietary portfolio and major alliances with leaders in the biopharmaceutical industry including Bristol-Myers Squibb, Novo Nordisk A/S, Sanofi, and a multi-products collaboration with AstraZeneca.

Créé en 1964, l’Institut national de la santé et de la recherche médicale (Inserm) est un établissement public à caractère scientifique et technologique, placé sous la double tutelle du Ministère de l’Enseignement supérieur et de la recherche et du ministère de la Santé. Ses chercheurs ont pour vocation l’étude de toutes les maladies, des plus fréquentes aux plus rares, à travers leurs travaux de recherches biologiques, médicales et en santé des populations. L’Inserm, chercheur en santé depuis 1964 Avec un budget 2013 de 927 M€, l’Inserm soutient près de 300 laboratoires répartis sur le territoire français. L’ensemble de ses 1200 équipes regroupe près de 15 000 chercheurs, ingénieurs, techniciens, gestionnaires, hospitalo-universitaires, post-doctorants…Il est actuellement dirigé par Yves Levy. L’Inserm est membre de l’Alliance nationale pour les sciences de la vie et de la santé, fondée en avril 2009 avec le CNRS, le CEA, l’Inra, l’Inria, l’IRD, l’Institut Pasteur, la Conférence des Présidents d’Université (CPU) et la Conférence des directeurs généraux de centres hospitaliers régionaux et universitaires. Cette alliance s’inscrit dans la politique de réforme du système de recherche visant à mieux coordonner le rôle des différents acteurs et à renforcer la position de la recherche française dans ce secteur par une programmation concertée. L’Inserm célèbre ses 50 ans en 2014.

Construit au cœur de l'Hôpital national des 15-20, l’Institut de la Vision est l’un des plus importants centres de recherche intégrée sur les maladies de la vision en Europe. Conçu comme un lieu de rassemblement et d’échanges, il réunit sur un même site la recherche fondamentale, clinique et industrielle, favorisant ainsi le partage des concepts et des techniques, la rencontre de compétences et d’expertises complémentaires et l’émergence de nouvelles pistes de recherche. Chercheurs, médecins et industriels y travaillent de concert pour découvrir et valider ensemble de nouvelles thérapeutiques, des solutions préventives, ainsi que des technologies compensatrices des atteintes visuelles.

Situé à Paris au cœur du plus grand centre hospitalier européen, l'hôpital de la Pitié-Salpêtrière, l’Institut de Myologie est né en 1996 sous l’impulsion d’une association de malades et parents de malades, l’AFM-Téléthon. Son objectif : favoriser l’existence, la reconnaissance et l’essor de la Myologie, science et médecine du Muscle, en tant que discipline à part entière. Qu’il soit sain, malade, accidenté, sportif, vieillissant… le muscle, dont dépend notre activité et nos fonctions vitales, est devenu un véritable modèle d’innovation pour la recherche scientifique et médicale et un enjeu de santé publique. L’Institut de Myologie coordonne, autour du malade, la prise en charge médicale, la recherche fondamentale, la recherche appliquée, la recherche clinique et l’enseignement. C’est un centre de référence international qui participe à de nombreux essais et études cliniques concernant principalement les maladies neuromusculaires mais également les lésions musculaires liées au sport de haut niveau ou au vieillissement. Depuis 2005, l’Institut de Myologie est une association régie par la loi du 1er juillet 1901 dont les membres fondateurs sont l’AFM-Téléthon et Généthon. L’Association Institut de Myologie a pour mission de faciliter la coordination des activités du site, en partenariat avec cinq tutelles publiques : AP-HP, CEA, Inserm, Sorbonne Université (ex-Université Pierre et Marie Curie) et CNRS.

The Institut Pasteur, a non-profit foundation with recognized charitable status set up by Louis Pasteur in 1887, is today an internationally renowned center for biomedical research with a network of 33 members worldwide. In the pursuit of its mission to tackle diseases in France and throughout the world, the Institut Pasteur operates in four main areas: research, public health, training, and development of research applications. The Institut Pasteur is a globally recognized leader in infectious diseases, microbiology, and immunology, with research focusing on the biology of living systems. Among its areas of investigation are emerging infectious diseases, antimicrobial resistance, certain cancers and brain connectivity diseases. The Institut Pasteur's outstanding research is facilitated by the development of a technological environment of the highest standard, with core facilities for nanoimaging, computational biology and artificial intelligence. Since its inception, 10 Institut Pasteur scientists have been awarded the Nobel Prize for Medicine, including two in 2008 for the 1983 discovery of the human immunodeficiency virus (HIV) that causes AIDS. www.pasteur.fr/en

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

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.

Jalon Therapeutics aims to develop innovative therapies that inactivate yet untargeted vital tumor signaling pathways. Our mission is to develop medicines that will transform the lives of people fighting cancer. For that purpose, we leverage our deep understanding of stress-related protein-protein interaction networks that sustain the malignant phenotype while not being necessary for normal cells. This mechanism, defined as “non-oncogene addiction” (NOA), offers potential novel, safer and more effective therapeutic strategies. Jalon Therapeutics is rooted in pioneering fundamental research and clinical investigations from INSERM laboratories and Saint-Louis Hospital in Paris. Jalon Therapeutics was cofounded in 2021 by Jean-Luc Poyet, Prof. Martine Bagot, Prof. Hervé Dombret, Jérôme Tiollier, Jean-Christophe Rain and Philippe Salphati. Among the proteins involved in non-oncogene addiction, the scaffold protein AAC-11 (Anti-Apoptosis Clone-11) is a cornerstone component of the signaling networks essential for cancer cell survival, adaptation to stress, resistance to therapies, immune evasion and metastatic potential. Derived from AAC-11, JRT39 is the first-in-class lead candidate developed for the treatment of hard-to-treat cancers. JRT39 properties combine the broad tissue distribution and cell permeability of small molecules with the excellent specificity and target-engagement potency of antibodies, together with unique modes of action. As such, JRT39 stands as breakthrough discovery to treat advanced and refractory cancers, alone or in combination with other treatment modalities. Furthermore, Jalon Therapeutics is building a drug discovery platform to develop other candidates derived from AAC-11 and AAC-11 partners for the treatment of cancer.

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

Odimma is a French biotech company with a unique approach in active personalized cancer immunotherapy. Immunotherapy has revolutionized the treatment of many advanced cancers. Despite this amazing achievement, significant clinical benefits are still limited to 10 to 30% of patients. Odimma believes in the future of precision medicine, and leverages recent advances in the field of immuno-oncology to amplify the efficacy of current therapies, improving lives and making each patient unique. By harnessing the ability of the patient’s own immune system to specifically recognize non-self-targets displayed by the tumor, also called neoantigens, Odimma has designed a next-generation personalized immunization platform that brings a sustainable therapeutic solution to fight the most hard-to-treat cancers. Carried by a team of experts and a supportive environment, we are now preparing our upcoming entry in the clinic

OSE Immunotherapeutics is an integrated biotech company focused on developing first-in-class assets targeting cancer and inflammatory diseases. A current well-balanced first-in-class clinical pipeline including: • Tedopi® (T-cell specific immunotherapy): Company’s most advanced product; positive results for Ph 3 in NSCLC in secondary resistance after CKI failure. Other sponsored combo Ph 2 trials in solid tumors ongoing. • OSE-279 (anti-PD1) advanced preclinical stage • OSE-127 (humanized mAb antagonist of IL-7R); ongoing Ph 2 in Ulcerative Colitis; ongoing preclinical research in leukemia • VEL-101/FR104 (anti-CD28 mAb) developed in partnership with Veloxis in transplantation; ongoing Ph 1/2 in renal transplant (sponsor Nantes University Hospital); Ph 1 ongoing in the US (sponsor Veloxis). • BI 765063 (anti-SIRPα mAb on CD47/SIRPα pathway) developed in partnership with Boehringer Ingelheim in advanced solid tumors; positive Ph 1 dose escalation results in mono and in combo, in particular with anti-PD-1 antibody ezabenlimab; BI sponsored international Ph 1b clinical trial ongoing in combo with ezabenlimab alone or with other drugs in recurrent/metastatic HNSCC and HCC. OSE Immunotherapeutics expects to generate further significant value from its two proprietary drug discovery platforms, which are central to its ambitious goal to deliver next-generation first-in-class immunotherapeutics: • BiCKI® platform focused on immuno-oncology (IO) is a bispecific fusion protein platform built on the key backbone component of anti-PD1 combined with a new immunotherapy target to increase anti-tumor efficacy. Most advanced BiCKI® candidate is targeting anti-PD1xIL-7. • Myeloid platform focused on optimizing the therapeutic potential of myeloid cells in IO & immuno-inflammation (I&I). OSE-230 (ChemR23 agonist mAb), most advanced candidate generated by the platform, with the potential to resolve chronic inflammation.

PEP-Therapy is a clinical-stage biotechnology company developing first-in-class peptides as targeted therapies in oncology. PEP-010, first drug candidate, is a pro-apoptotic agent which has demonstrated anti-tumor efficacy in a number of pre-clinical models and a good safety profile. PEP-010 is currently evaluated in a Phase Ia/b clinical trial in patients with recurrent and/or metastatic solid tumors. PEP-Therapy was founded in 2014 and builds on research results from Institut Curie and Sorbonne University. The company is backed by international investors.

Pierre Fabre Group held by the Pierre Fabre Foundation, is a worldwide company with a unique positioning : the alliance of pharmaceutical and dermocosmetics expertise. This makes Pierre Fabre Group : 💊 A pharmaceutical group with a strong positioning : medical and natural 🥈 The second largest dermo-cosmetics laboratory in the world 🥈 The second largest private French pharmaceutical group 🥇 The market leader in France for products sold over the counter in pharmacies. Our portfolio includes several medical franchises and international brands including ; Pharmaceutical Care Pierre Fabre Oncologie Pierre Fabre Dermatologie Naturactive Eau Thermale Avène Klorane Ducray René Furterer A-Derma Pierre Fabre Oral Care Glytone (US) Darrow (Brasil) Established in the Occitanie region since its creation, we manufacture over 95% of our products in France. We keep innovating passionately with our teams in 2 innovation centers in Brasil and Japan as well as in 6 R&D centers in France. Thanks to our 9,600 employees in 44 subsidiaries and our distribution activities in 120 countries, our group generated €2.7 billion in revenues in 2022, 69% of which is from international business. Pierre Fabre Group has a unique company structure. 86% of the Pierre Fabre Group is held by the Pierre Fabre Foundation, a government-recognized public-interest foundation, while a smaller share is owned by its employees via an employee stock ownership plan. In 2020, the independent organization ECOCERT Environment awarded Pierre Fabre's CSR policy at "excellence" level : - Excellence being the highest maturity level of the ECOCERT 26000 standard.

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.

PulseSight is an ophthalmology drug development company creating novel, non-viral gene therapies with minimally invasive delivery technology to address the unmet need for treatments against severe retinal diseases leading to blindness.

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).

Smart Immune is a clinical-stage biotechnology company developing ProTcell, a thymus-empowered T-cell therapy platform to fully and rapidly re-arm the immune system, enabling next-generation allogeneic T-cell therapies for all. The company is a spin-off from the Imagine Institute of Genetic Diseases, and was founded in 2017 by Karine Rossignol, PharmD-HEC, Marina Cavazzana, MD-PhD and Isabelle André, PhD to transform outcomes for patients with life-threatening diseases such as high-risk blood cancers and primary immunodeficiencies. Smart Immune has clinical partnerships with leading institutions in the US and Europe. The ProTcell platform, already in Phase I/II clinical trials, enables the accelerated recovery of a complete immune repertoire in patients fighting cancer and infection. The ProTcell platform introduces potent, allogeneic T-cell progenitors which are then differentiated by the thymus into fully functional T-cells, to ultimately develop an ‘off the shelf’ T-cell medicine. The company is headquartered in Paris, France, at Paris Biotech Santé.

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.

Stilla Technologies is a leading global biotechnology company that focuses on accelerating development of next-generation genetic testing by providing researchers and clinicians with a flexible Digital PCR solution for high-resolution genetic analysis and assay development. Stilla’s next-generation Multiplex Crystal Digital PCR™ technology employs cutting-edge microfluidic innovations and integrates the entire dPCR process on a single chip. The naica® system is highly sensitive, fast, and features an easy workflow, higher levels of multiplexing, and intuitive software. Stilla developed the world's first 6-color Digital PCR Platform, providing High Multiplexing and Sensitivity for Advancing Cancer & Liquid Biopsy Studies, Cell & Gene Therapies, Infectious Disease & COVID-19 Research, and Wastewater and Environmental Testing and many more applications. Stilla aims to make Digital PCR a lab commodity in all life sciences areas including research, therapeutics, and the omics. Visit the website and connect on Twitter @StillaTech

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

TreeFrog Therapeutics is a French-based biotech company aiming to unlock access to cell therapies for millions of patients. TreeFrog Therapeutics is developing a pipeline of therapeutic candidates using proprietary C-Stem technology, allowing for the mass production of induced pluripotent stem cells and their differentiation into ready-to-transplant microtissues with unprecedented scalability and cell quality. Bringing together over 150 biophysicists, cell biologists and bioproduction engineers, TreeFrog Therapeutics raised $82M in 2021 to advance its pipeline of stem cell-based therapies in the field of regenerative medicine with the lead program in Parkinson's Disease. In 2022, the company opened a technological hub in Boston, USA to drive the adoption of C-Stem and initiate co-development partnerships with leading academic, biotech and industry players in the field of cell therapy.

Université de Bordeaux is a higher education institution offering a wide range of academic programs and conducting research.

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.