What Genethon does

Genethon is a French nonprofit biotech founded in 1990 by the patient organization AFM-Téléthon, dedicated to developing gene therapies for rare genetic diseases that are commercially unattractive to for-profit pharmaceutical companies. The organization operates a 15-candidate pipeline spanning neuromuscular diseases (Duchenne muscular dystrophy, limb-girdle muscular dystrophies, myotubular myopathy, SMA), liver metabolic disorders (Crigler-Najjar syndrome, Pompe disease, GSD1a, Cori disease), blood disorders (sickle cell disease, Fanconi anemia), and immune deficiencies (Wiskott-Aldrich syndrome, SCID, chronic septic granulomatosis).

Its core technology stack centers on AAV gene therapy vectors (notably AAV8 for GNT0004 in DMD), CRISPR/Cas9 gene editing (including a mutation-independent utrophin upregulation approach for DMD), microdystrophin transgenes with muscle-specific promoters, lentiviral vectors with embedded microRNAs, and dual-AAV split-intein approaches. Genethon complements these with proprietary platform capabilities including AI-powered AAV capsid engineering via its Ampersand Biomedicines partnership, cardiotoxicity mitigation strategies for high-dose AAV, imlifidase pretreatment to overcome AAV seropositivity (applicable to ~1 in 3 patients), and an ISO 20387-certified DNA Bank. Clinical-stage lead asset GNT0004 has demonstrated +9 NSAA points, 173m improvement in 6-minute walk test, and 70% sustained CPK reduction at 2 years at a notably low therapeutic dose of 3×10¹³ vg/kg.

Genethon's revenue model is hybrid and nonprofit: ~€800 million in cumulative AFM-Téléthon funding since 1990 supports R&D operations, complemented by licensing royalties (e.g., GAA transgene to AskBio), patent licensing to biotech companies, and research services leveraging its DNA bank and imaging cytometry platform. Distribution occurs exclusively through academic clinical trial sites in France, the UK, and the US, with eventual commercialization routed through spin-off Atamyo Therapeutics and the GenoTher biocluster. The organization employs 200+ R&D staff at its Évry-Courcouronnes headquarters within the Genopole biocluster and is led by CEO Frédéric Revah (since 2010) under President Laurence Tiennot-Herment.

Name

Genethon

Legal name

Généthon

Website

https://genethon.fr

Company type

Private

Founded year

1990

Operating status

Operating

Headcount range

101–250 employees

Short description

Genethon is a French nonprofit biotech founded in 1990 by AFM-Téléthon that develops AAV, CRISPR, and lentiviral gene therapies for ultra-rare genetic diseases, with 15 candidates in its pipeline and lead program GNT0004 in Phase III for Duchenne muscular dystrophy.

Ownership category

akta.pro rank

Product category

Gene Therapy / Rare Disease Biopharmaceuticals

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Scientific Research and Development Services (5417), Medical Laboratories (621511)

SIC

Services-Commercial Physical & Biological Research (8731), Services-Medical Laboratories (8071), Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

Gene Therapy for Rare/Monogenic Diseases (HLAAACAE)

akta.pro secondary industries

Gene Therapy CMC/Process Development Platforms (capsid engineering, producer cell lines, plasmids, upstream/downstream) (HLAAACAN), Genome Editing Gene Therapies (CRISPR/TALEN/ZFN; in vivo/ex vivo) (HLAAACAD), Neurology/CNS Gene Therapies (HLAAACAH), Molecular & Genetic Testing (PCR/NGS/qPCR) (HLAGADAF)

Keywords

• Gene therapy research
• Rare disease therapeutics
• AAV vector development
• Nonprofit biotech
• CRISPR gene editing

Headquarters

HQ city

Évry

HQ country

France

HQ region

Europe

Offices2 records

Markets served

GTM type

B2B

Offering type

Services

Cost components

Technology or R&D, Personnel, Infrastructure, Operations, Marketing or Sales, Supply Chain

Revenue model

1. Nonprofit Research Funding and Charitable Donations: Genethon is a French nonprofit biotech funded primarily by patient organization AFM-Téléthon, which has provided nearly €800m in funding since Genethon's founding in 1990. The nonprofit structure enables greater scientific and strategic risk-taking for rare diseases commercially unattractive to for-profit pharmaceutical companies. Revenue comes from grants, donations, and potentially future therapeutic product revenues upon regulatory approval.
2. Licensing Royalties and Technology Transfer: Genethon licenses its proprietary technologies and gene therapy components to commercial partners. In January 2026, it concluded an exclusive worldwide licensing agreement with AskBio (a Bayer subsidiary) for a GAA transgene for Pompe disease treatment, and holds patents licensed to biotechnology companies.
3. Gene Therapy Clinical Development Services: Through its partnership with academic medical centers, Genethon conducts clinical trials as the trial sponsor, with costs covered by its nonprofit funding model. The organization may also provide research services and access to its DNA bank and imaging cytometry platforms to external academic and industrial users.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels5 records

Core offering

Genethon is a nonprofit French biotech research laboratory that develops gene therapies for rare genetic diseases, leveraging AAV vectors, CRISPR/Cas9 gene editing, and lentiviral platforms. It operates a pipeline of 15 gene therapy candidates covering Duchenne muscular dystrophy, Crigler-Najjar syndrome, limb-girdle muscular dystrophies, sickle cell disease, and other monogenic conditions, with its lead candidate GNT0004 in pivotal Phase III clinical trials. The organization also provides certified DNA banking, imaging cytometry services, and licenses proprietary gene therapy technologies to commercial partners.

Product overview

Genethon is a nonprofit French biotech specializing in gene therapy research and development for rare genetic diseases. The organization operates as a research laboratory developing multiple clinical-stage gene therapy candidates, with GNT0004 for Duchenne muscular dystrophy being the most advanced (pivotal Phase III). The pipeline includes 15 gene therapy candidates across diverse rare diseases. Genethon also provides research platforms including a certified DNA Bank and Imaging Cytometry Platform. The organization leverages AAV vector technology, CRISPR gene editing, and collaborates with partners for bioproduction and clinical development.

Differentiator

Problem solved

Functional benefit

Products and services

• GNT0004 AAV8-based microdystrophin gene therapy for Duchenne muscular dystrophy, administered as a single intravenous injection at therapeutic dose of 3×10¹³ vg/kg. Currently in pivotal Phase III clinical trials in Europe (France, UK).
• GNT0003 Gene therapy for Crigler-Najjar syndrome using AAV8 vector, developed with imlifidase pretreatment approach to enable administration in patients with pre-existing AAV antibodies.
• ATA-200 Gene therapy for LGMD-R5 (limb-girdle muscular dystrophy type R5/sarcoglycanopathy), being developed in partnership with Atamyo Therapeutics, with clinical trial sites including the University of Florida Powell Gene Therapy Center.
• Gene Therapy Pipeline (15 candidates) Portfolio of 15 gene therapy candidates for rare diseases covering Duchenne muscular dystrophy, Spinal muscular atrophy, SMA-PME and Farber's disease, Myotubular myopathy, Limb girdle muscular dystrophies, Wiskott-Aldrich syndrome, Fanconi's anemia, Sickle cell disease, Chronic septic granulomatosis, SCID, Cori Forbes disease, GSD1a, Crigler-Najjar syndrome, Pompe disease, and Leber Hereditary Optic Neuropathy.
• DNA Bank European leading DNA and cell bank for genetic diseases, ISO 20387 certified, providing high-quality biological samples and services to the scientific and medical community.
• Imaging Cytometry Platform (ImCy) High-technology platform providing imaging and flow cytometry support for Genethon research, labeled by Génopole, also open to external users.
• DREPAMIR Clinical Trial (sickle cell disease gene therapy) Phase I/II clinical trial for sickle cell disease using an innovative bifunctional lentiviral vector developed by Mario Amendola's team at Genethon, conducted in collaboration with Institut Imagine, AP-HP, Inserm, and Université Paris Cité.

Quantifiable outcome

• +9 NSAA points at 2 years vs untreated controls in DMD patients treated with GNT0004 at therapeutic dose (3×10¹³ vg/kg)
• +6 more outcomes

Named customers1 record

Segments2 records

Ideal customer profiles3 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature6 records

Partnerships

13 partnerships are on record, tiered core and minor.

• Ampersand BiomedicinescoreTechnology or Integration · 21 May 2026Exclusive collaboration to engineer new AAV vectors with enhanced tissue specificity for gene therapy, leveraging Ampersand's AI-powered Address, Navigate, Determine (AND) platform and Genethon's AAV expertise. Initially focused on skeletal muscle targeting with plans to expand to other tissues. Combines precision targeting technology with gene therapy development to improve therapeutic outcomes and reduce required doses.
• Institut Imagine / AP-HP / Inserm / Université Paris CitécoreStrategic or Co-development Partner · 27 March 2026Collaboration led by Prof. Marina Cavazzana and Dr. Anne Galy with Mario Amendola's team at Genethon contributing the bifunctional lentiviral vector for the DREPAMIR sickle cell disease clinical trial (Phase I/II). ANSM authorized the trial in March 2026.
• The Dion FoundationminorStrategic or Co-development Partner · 3 February 2026Partnership extension with The Dion Foundation supporting the ATA-200 gene therapy program for LGMD-R5 limb-girdle muscular dystrophy. First three children treated in ongoing clinical trial; 90%+ muscle fiber expression of therapeutic SGCG protein demonstrated.
• AskBio (Bayer AG subsidiary)coreOEM/ Whitelabel/ Licensing Partner · 14 January 2026Exclusive worldwide licensing agreement for Genethon's proprietary GAA transgene for Pompe disease gene therapy. AskBio plans to initiate clinical trial in early 2026. Genethon's Immunology and Liver Diseases team developed and demonstrated preclinical efficacy of the therapy.
• Hansa BiopharmacoreStrategic or Co-development Partner · 10 October 2025Collaboration on the GNT-018-IDES clinical trial testing imlifidase (IgG-degrading enzyme) as pretreatment to enable gene therapy in Crigler-Najjar syndrome patients with natural AAV immunity. Successfully demonstrated feasibility and safety, with bilirubin reduction enabling discontinuation of phototherapy 16 weeks post-injection in first patient.
• GenoTher BioclustercoreStrategic or Co-development Partner · 15 May 2025Co-founder of GenoTher, a France 2030-labeled biocluster exclusively dedicated to gene therapy. Partners include Genethon, Genopole, AP-HP, INSERM, University of Evry Paris-Saclay, Spark Therapeutics, and Yposkesi. Aims to structure, accelerate, and industrialize gene therapy innovation from research to commercialization.
• University of Florida Powell Gene Therapy CenterminorStrategic or Co-development PartnerClinical trial site for ATA-200 LGMD-R5 gene therapy Phase 1b/2 study; Dr. Barry Byrne presented preliminary safety and efficacy data at ASGCT 2026.
• INSERMcoreStrategic or Co-development PartnerGenethon operates its main research unit (UMR_S951 Integrare) as a joint unit with INSERM and University of Evry Paris-Saclay. Multiple collaborative research programs and co-authored publications.
• University of London, Royal Holloway (Pr Dickson)coreStrategic or Co-development PartnerCollaborative development of GNT0004 microdystrophin gene therapy for Duchenne muscular dystrophy, including preclinical research and transgene optimization.
• Institute of Myology (Paris)coreStrategic or Co-development PartnerPartnership with the Institute of Myology for DMD gene therapy development, immunology center creation (led by Klaudia Kuranda), and clinical trial execution. Both organizations are part of the AFM-Téléthon ecosystem.
• I-StemminorStrategic or Co-development PartnerPart of the Biotherapies Institute for Rare Diseases (Institut des biothérapies des maladies rares) created by AFM-Téléthon, alongside Genethon, Institute of Myology, and I-Stem (stem cell research center).
• Yposkesi (CDMO)minorImplementation/ SI/ Consulting PartnerYposkesi is a CDMO (Contract Development and Manufacturing Organization) spin-off of Genethon, specialized in gene therapy manufacturing. Frédéric Revah serves on its Board.
• Atamyo TherapeuticsminorStrategic or Co-development PartnerAtamyo is a biotech spin-off of Genethon dedicated to neuromuscular gene therapies. Frédéric Revah serves as President of Atamyo's Strategic Committee. Collaboration on limb-girdle muscular dystrophy programs including ATA-200.

Scale indicators8 records

Recent moves10 records

Expansion highlights6 records

Broad incumbents

• BioMarin Pharmaceutical: Rare-disease biotech with AAV gene therapy (Roctavian for hemophilia A) and broad rare-disease portfolio. Comparable in AAV modality and rare-disease commercial positioning.
• AveXis (Novartis Gene Therapies): Novartis's gene therapy unit commercializing Zolgensma (onasemnogene abeparvovec) for spinal muscular atrophy — a neuromuscular AAV gene therapy directly comparable to Genethon's neuromuscular pipeline focus.
• Pfizer: Global pharma with approved DMD gene therapy Beqvez (fordadistrogene movaparvovec). Comparable as a competing developer of AAV microdystrophin therapies for DMD and a major participant in rare-disease gene therapy.

Others

• Yposkesi: CDMO spin-off of Genethon specialized in gene therapy manufacturing. Comparable as the bioproduction backbone serving Genethon's pipeline and external gene therapy clients.

Direct peers

• Spark Therapeutics: AAV-based gene therapy developer (Luxturna) now a Roche subsidiary and co-founder of the GenoTher biocluster with Genethon. Directly comparable as an AAV gene therapy company targeting rare monogenic diseases.
• Bluebird Bio: Gene therapy company developing lentiviral vector therapies for rare genetic diseases (sickle cell, beta-thalassemia, cerebral adrenoleukodystrophy). Comparable modality and rare-disease focus to Genethon's lentiviral and AAV programs.
• AskBio: AAV gene therapy developer and Bayer subsidiary — Genethon's licensing partner for the Pompe disease GAA transgene. Comparable in AAV modality and rare-disease pipeline focus.
• Sarepta Therapeutics: Approved Elevidys (delandistrogene moxeparvovec) for Duchenne muscular dystrophy — the most direct competitor to Genethon's GNT0004 in the AAV microdystrophin space, with shared indication and modality.
• uniQure: AAV gene therapy company targeting rare diseases (hemophilia B with etranacogene dezaparvovec). Comparable to Genethon in modality, rare-disease focus, and European clinical pipeline.

Emerging players

• Atamyo Therapeutics: Biotech spin-off from Genethon focused on gene therapy for limb-girdle muscular dystrophies (ATA-200, ATA-100). Comparable as a clinical-stage AAV gene therapy company operating inside the AFM-Téléthon ecosystem.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat6 records

Key risks6 records

Key highlights7 records

Customer concentration

• LinkedInlinkedin.com/company/genethon

Compliance1 record

Revenue estimate

Valuation estimate

Number of profiles

Profiles12 records

Subsidiaries1 record

Funding overview

Funding rounds

Investors

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M&A

Investments

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Frequently asked questions about Genethon