AFM-Telethon

What AFM-Telethon does

AFM-Téléthon (Association Française pour la Myopathie, founded 1958, headquartered in Évry, France) is a non-profit association recognized as being of public utility that operates as a patient-led organization fighting rare, genetic, and severely disabling diseases—primarily neuromuscular diseases such as Duchenne muscular dystrophy, spinal muscular atrophy, limb-girdle muscular dystrophies, myasthenia gravis, and Steinert disease. The organization serves three constituencies: patients and families affected by these diseases (3M people in France, 30M in Europe), the French general public as donors and volunteers (~3,030 permanent volunteers nationwide), and the biomedical research community. Governance is conducted exclusively by patients and parents of patients, with Laurence Tiennot-Herment presiding since 2003.

The association's core technical asset is the Institut des Biothérapies des Maladies Rares, which assembles three proprietary research laboratories across 15,000 m² in Paris and Évry: Généthon (gene therapy with ~260 collaborators, ~15 gene therapy products in clinical evaluation spanning neuromuscular, immune, blood, and liver diseases using AAV and lentiviral vectors); I-Stem (stem cell and cell therapy, 84 experts, with Inserm as co-founder since 2005); and the Institut de Myologie (300 experts, a European center of muscle expertise with I-Motion pediatric and adult clinical trial platforms at Trousseau and Pitié-Salpêtrière hospitals). Adjacent specialized entities include YposKesi (5,000 m² GMP viral vector bioproduction, Europe's leader, in which AFM-Téléthon retains a 17.4% stake via H-MRB); GenoSafe (45-staff service platform for quality and safety evaluation of innovative biologicals); the GenoTher biocluster (founded 2024 with €70M of Innovation Santé 2030 funding alongside Roche/Spark, Genopole, AP-HP, Inserm, YposKesi, Université Évry Paris-Saclay); and Ampleia, a startup studio created in 2023 to convert R&D-stage assets into drug candidates and biotech spinouts.

AFM-Téléthon's revenue model is donation-driven and transparent: the annual Téléthon televised campaign raised €100,548,314 in 2025, contributing 75.3% of €133.5M total revenue, with a 66% French income-tax deduction providing a structural incentive for giving. Supplemented by legacy gifts, public co-funding for specific facilities (e.g., the Yolaine de Kepper care home), and revenue from operating subsidiaries, the association deployed €106.5M to social missions plus €42.6M in advances and investments for the Guérir mission in 2025. Distribution of impact occurs through 115 research projects and 46 young researchers funded via annual calls for proposals, 91 funded multidisciplinary consultations across France, a nationwide referral network of 15 regional services and 68 departmental delegations, respite and assistive-technology facilities (Village Répit Familles, Maison d'Etiolles), and the Plateforme Maladies Rares consortium. Reported outcomes include 9 treatments already available to patients (Zolgensma, Skysona, Strimvelis, Zynteglo, Lumevoq, Firdapse, Cuprior, Namuscla, Metformin) and the generalization of SMA newborn screening in France from September 2025.

Name

AFM-Telethon

Legal name

AFM-Téléthon (Association Française pour la Myopathie)

Website

https://afm-telethon.fr

Company type

Private

Founded year

1958

Operating status

Operating

Headcount range

251–500 employees

Short description

AFM-Téléthon is a French non-profit patient-led association founded in 1958 that funds and conducts research into rare genetic and neuromuscular diseases through three in-house laboratories, a bioproduction platform, and a startup studio, while providing nationwide patient support, advocacy, and clinical care funded primarily by the annual Téléthon televised donation drive.

Ownership category

akta.pro rank

Product category

Nonprofit Rare Disease Research and Patient Support

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Religious, Grantmaking, Civic, Professional, and Similar Organizations (813), Individual and Family Services (6241)

SIC

Services-Medical Laboratories (8071), Services-Commercial Physical & Biological Research (8731), Services-Social Services (8300)

akta.pro primary industry

Disease-Specific Research & Support (e.g., Cancer, Diabetes, ALS) (BPAGACAA)

akta.pro secondary industry

Neurogenetics & Rare Neurologic Diseases (HLAKAIAO)

Keywords

• Rare disease research
• Gene therapy development
• Neuromuscular disease association
• Charitable fundraising campaigns
• Patient advocacy services

Headquarters

HQ city

Bas Évry

HQ country

France

HQ region

Europe

Offices10 records

Markets served

GTM type

B2C

Offering type

Services

Cost components

Personnel, Technology or R&D, Operations, Marketing or Sales, Infrastructure, Others

Revenue model

1. Public donations and Telethon fundraising: The primary revenue stream comes from public generosity — donations collected during the annual televised Téléthon event and year-round giving. In 2025, 100.5 M€ came from Telethon donations out of total revenue of 133.5 M€. Donors receive a 66% tax deduction in France, making donations highly incentivized.
2. Legs and donations (inheritance/transmission): AFM-Téléthon receives legacy donations and property bequests from donors, which are a significant component of long-term fundraising.
3. Government and public funding: Some activities, particularly the Yolaine de Kepper establishment, are financed by public funds. The Association also participates in calls for proposals co-funded by public bodies.

Pricing tiers

Go-to-market motion1 record

Distribution channels6 records

Marketing channels9 records

Core offering

AFM-Téléthon is a patient-led French non-profit association that fights rare genetic and neuromuscular diseases through three missions: Guérir (Cure) via its three research laboratories (Généthon for gene therapy, I-Stem for stem cell therapy, Institut de Myologie for muscle science), Aider (Help) via 15 regional services, multidisciplinary clinical consultations, respite care facilities and patient advocacy, and Communiquer (Communicate) via the annual Téléthon televised fundraising marathon and educational outreach. The association develops and funds gene and cell therapies, operates bioproduction infrastructure, and coordinates a nationwide network of patient support services.

Product overview

AFM-Téléthon is a patient-led association organized around three core missions: Guérir (Cure), Aider (Help), and Communiquer (Communicate). The organization operates primarily through its own research laboratories—Généthon (gene therapy), Institut de Myologie (muscle science), and I-Stem (stem cell therapy)—gathered within the Institut des biothérapies des maladies rares. Supporting infrastructure includes YposKesi (industrial gene therapy production), GenoSafe (biological product evaluation), GenoTher (biocluster), and Ampleia (startup studio). Patient support is delivered through 15 regional services with Health Pathway Referrers, a clinical network of 91 consultations, technical compensation services, and care facilities. Educational outreach is conducted via the 1000 chercheurs dans les écoles program. The annual Téléthon fundraising campaign generated 100.5 million euros in 2025. The organization also operates data infrastructure (health data warehouse) and publishes Myobase, a scientific database on rare diseases.

Differentiator

Problem solved

Functional benefit

Brands

• Généthon: Pioneering laboratory in gene therapy for rare diseases, one of the world leaders in the field with 260 collaborators and 15 gene therapy products in clinical evaluation.
• Institut de Myologie
• I-Stem
• GenoSafe
• YposKesi
• GenoTher
• Ampleia
• Institut des biothérapies des maladies rares
• Plateforme Maladies Rares
• Village Répit Familles (VRF)
• Yolaine de Kepper Center

Products and services

• Généthon Gene Therapy Research Laboratory

Quantifiable outcome

• FDA approval of KYGEVVI® (doxecitine and doxribtimine) — first and only treatment for TK2 deficiency, reducing risk of death by over 90% for early-onset cases
• +6 more outcomes

Named customers6 records

Segments5 records

Ideal customer profiles4 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature6 records

Partnerships

21 partnerships are on record, tiered minor and core.

• AEFE (Agence pour l'Enseignement Français à l'Étranger) and MLF (Mission Laïque Française)minorGTM or Marketing Partner · 1 January 2026AFM-Téléthon partners with AEFE and MLF to bring the '1000 chercheurs dans les écoles' program to French schools abroad. The operation takes place year-round for international French schools, with researchers from AFM-Téléthon labs visiting classes.
• FNHPA (Fédération Nationale de l'Hôtellerie de Plein Air)minorGTM or Marketing Partner · 1 January 2026AFM-Téléthon partners with FNHPA for #TéléthonCamping, now in its 7th edition (July-August 2026) across 200+ campsites in France. This summer initiative has collected over €500,000 for AFM-Téléthon through family-friendly activities at campsites.
• GenoTher Biocluster (Founding partners: Généthon, Spark/Roche, Genopole, AP-HP, Inserm, YposKesi, Université Évry Paris-Saclay)coreStrategic or Co-development Partner · 1 January 2024GenoTher is a biocluster dedicated to accelerating gene therapy drug development, launched in 2024. Founded by Généthon and six partners including Spark (Roche subsidiary), Genopole, AP-HP, Inserm, YposKesi, and Université Évry Paris-Saclay, it brings together 30 industrial, academic, investor, and grande école partners. Labelled under the Innovation Santé 2030 plan with €70 million in funding over five years. Objectives: 10s of new gene therapies for rare and common diseases within 10 years.
• IRDiRC (International Rare Diseases Research Consortium)minorStrategic or Co-development Partner · 1 January 2024AFM-Téléthon is a member of IRDiRC, a transatlantic consortium launched by the European Commission and NIH to promote therapies and genetic diagnostic tools for rare diseases.
• EURO-NMD (European Reference Network for Neuromuscular Diseases)minorStrategic or Co-development Partner · 1 January 2024AFM-Téléthon is a member of EURO-NMD, the European Reference Network for neuromuscular diseases, facilitating cross-border research and care coordination.
• Screen4Care (EU consortium for newborn screening)minorStrategic or Co-development Partner · 1 January 2024AFM-Téléthon participates in Screen4Care, a European consortium dedicated to rare disease diagnosis including neonatal screening pilot projects.
• REMEDi4ALL (EU consortium for drug repositioning)minorStrategic or Co-development Partner · 1 January 2024AFM-Téléthon is a member of REMEDi4ALL, a European consortium dedicated to drug repositioning for rare diseases.
• ERDERA (European Rare Disease Research Alliance)minorStrategic or Co-development Partner · 1 January 2024ERDERA is the European Alliance for Rare Disease Research launched in September 2024, supported by the EU and integrating more than 170 public and private sector organizations. AFM-Téléthon is a member.
• Plateforme Maladies Rares (founders: AFM-Téléthon, Alliance Maladies Rares, Eurordis, Orphanet, Maladies Rares Info Services, Fondation Maladies Rares)coreStrategic or Co-development Partner · 1 January 2024AFM-Téléthon is the principal founder and main funder of the Plateforme Maladies Rares, a unique European resource center grouping France's main rare disease actors: Alliance Maladies Rares (240 associations), Eurordis (1,000 patient associations from 74 countries), Orphanet (global rare disease portal), Maladies Rares Info Services, and Fondation Maladies Rares (400+ research projects funded).
• AmpleiacoreStrategic or Co-development Partner · 1 January 2023Ampleia is a startup studio created by AFM-Téléthon in 2023 to transform R&D-stage innovations into drug candidates, then into preclinical and clinical development. It audits projects, incubates them, and supports their development through biotech company creation or licensing. In 2024, Ampleia audited ~130 projects, incubated 5, and plans the launch of a first startup in 2025.
• FILNEMUS (Filière de Santé Maladies Neuromusculaires)coreStrategic or Co-development Partner · 1 January 2023AFM-Téléthon is a privileged partner of FILNEMUS, the national healthcare network for neuromuscular diseases, which coordinates scientists, physicians, and associations nationwide for research, diagnosis, care, and treatment. AFM-Téléthon actively contributes to its work on diagnostic wandering, newborn screening, and national diagnostic and care protocol writing.
• ALAN Maladies Rares LuxembourgminorGTM or Marketing Partner · 1 January 2023AFM-Téléthon provides financial support to ALAN Maladies Rares Luxembourg, which offers socio-therapeutic consultation services to people with neuromuscular or rare diseases in Luxembourg.
• FITIMA (Fondation Internationale Tierno et Mariam) and ROAMY NetworkminorGTM or Marketing Partner · 1 January 2023AFM-Téléthon supports FITIMA in Burkina Faso and Guinea Conakry and the ROAMY network (Mali, Senegal, Togo, Benin, Niger, Guinea) to facilitate access to quality care and improve quality of life for neuromuscular disease patients in West Africa through socio-educational support.
• APF France HandicapminorStrategic or Co-development Partner · 1 January 2023AFM-Téléthon and APF France Handicap jointly advocate for the rights of people with disabilities. In October 2023, they jointly filed a complaint with the Defender of Rights regarding endangerment of disabled persons requiring home assistance. The Defender of Rights issued a framework decision in their favor in 2025.
• YposKesicoreStrategic or Co-development Partner · 1 January 2016YposKesi is a GMP viral vector manufacturing platform created by AFM-Téléthon and Bpifrance in 2016. Europe's leader in GMP viral vector production, it has 5,000 m² of production laboratories and ~200 experts. In 2021, international group SK became the majority shareholder. AFM-Téléthon retains a 17.4% stake through H-MRB.
• PRO BTPminorGTM or Marketing Partner · 1 January 2016PRO BTP co-created with AFM-Téléthon the 'Village Répit Familles' concept and the Vacances Répit Familles association that operates the facilities, providing respite vacations for caregivers and their family members with disabilities.
• GenSight BiologicsminorStrategic or Co-development Partner · 1 January 2013GenSight Biologics is a biotechnology company that benefited from the Bpifrance/AFM-Téléthon seed fund. It is developing gene therapy for Leber hereditary optic neuropathy (Lumevoq), which received marketing authorization in England. The company is pursuing authorization in Europe and the US.
• Flamingo Therapeutics, Step Pharma, Sparing Vision, Orphalan, RibogenicsminorStrategic or Co-development Partner · 1 January 2013Five additional biotechnology companies that benefited from the AFM-Téléthon/Bpifrance seed fund for innovative rare disease therapeutics. Each develops approaches for specific rare disease indications.
• Atamyo TherapeuticsminorStrategic or Co-development Partner · 1 January 2013Atamyo Therapeutics is a spin-off from Généthon that launched in 2025 a Phase I/II clinical trial for ATA-200 gene therapy for gamma-sarcoglycanopathy (LGMD R5), and runs a Phase I/II trial for ATA-100 for FKRP-related LGMD R9.
• Inserm (I-Stem laboratory)coreStrategic or Co-development Partner · 1 January 2005Inserm co-created I-Stem laboratory with AFM-Téléthon in 2005 as a facility dedicated to stem cells and cell therapy for monogenic diseases. I-Stem has 84 experts working on muscle, motor neuron, skin, retina, and central nervous system diseases.
• GenoSafecoreStrategic or Co-development Partner · 1 January 2004GenoSafe was created in 2004 by AFM-Téléthon and Généthon as a service company evaluating quality, safety, and efficacy of innovative biological products (gene therapies, cell therapies, antibodies) from preclinical studies through human trials. It has 45 collaborators and 70% of its clients are international.

Scale indicators13 records

Recent moves6 records

Expansion highlights7 records

Direct peers

• Fondation Maladies Rares: Fondation Maladies Rares is a French research foundation specifically focused on rare diseases, created in 2011 during the second National Rare Disease Plan. Both organizations sit within the Plateforme Maladies Rares ecosystem, fund rare disease research through calls for proposals, and coordinate with French rare disease policy.
• Muscular Dystrophy Association (MDA): The MDA is a US patient-led nonprofit that funds research and provides care for neuromuscular diseases, the same disease space AFM-Téléthon was originally founded to address. Both organizations fund gene therapy research, run summer camp programs for affected families, and have played catalytic roles in advancing therapies like exon-skipping and gene replacement approaches.
• Parent Project Muscular Dystrophy (PPMD): PPMD is a US patient-led nonprofit specifically focused on Duchenne muscular dystrophy, the disease that drove AFM-Téléthon's founding. Both organizations are governed by affected parents, fund translational research, and advocate for newborn screening (PPMD led the US pilot that paralleled AFM's DEPISMA program).
• EURORDIS - Rare Diseases Europe: EURORDIS is the European umbrella organization for rare disease patient associations (1,000 member associations across 74 countries), of which AFM-Téléthon is a co-founder of the Plateforme Maladies Rares alongside. Both organizations advocate for rare disease policy at the EU level and represent patient communities in research governance.
• National Organization for Rare Disorders (NORD): NORD is the US national umbrella for rare disease patient organizations, functioning as the American analogue of EURORDIS. Both organizations provide advocacy, research funding, and patient services, and collaborate on international rare disease policy through IRDiRC and other consortia.
• Cystic Fibrosis Foundation: The CFF pioneered the patient-led venture philanthropy model that AFM-Téléthon also embodies, funding research that led to CFTR modulator therapies. Both organizations raise funds through mass mobilization, fund biotech R&D, and have shaped regulatory pathways for breakthrough rare disease treatments.

Emerging players

• GenSight Biologics: GenSight is a clinical-stage biotech that benefited from AFM-Téléthon/Bpifrance seed funding and is developing Lumevoq gene therapy for Leber hereditary optic neuropathy (received marketing authorization in England). It exemplifies AFM-Téléthon's translational pipeline and venture-style biotech creation model.
• Atamyo Therapeutics: Atamyo is a Généthon spin-off developing AAV gene therapies for limb-girdle muscular dystrophies, currently running Phase I/II trials for LGMD R9 (ATA-100) and LGMD R5 (ATA-200). As a direct spin-out of AFM-Téléthon's research, it represents the venture philanthropy model AFM-Téléthon has pioneered.

Broad incumbents

• Wellcome Trust: Wellcome is one of the world's largest research-funding charitable foundations, with a comparable model of patient/mission-driven research investment at scale. Both organizations combine disease-specific programs (Wellcome in mental health/infectious disease; AFM in rare neuromuscular) with broader support for biomedical infrastructure.
• Inserm (Institut national de la santé et de la recherche médicale): Inserm is France's national biomedical research agency, co-creator of I-Stem with AFM-Téléthon and a founding partner of GenoTher. Both organizations fund and conduct rare disease research in France, with AFM-Téléthon's disease-specific focus complementing Inserm's broader biomedical mandate.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat6 records

Key risks6 records

Key highlights7 records

Customer concentration

• Xx.com/Telethon_France
• YouTubeyoutube.com/c/afmtelethon
• Facebookfacebook.com/Telethon
• LinkedInlinkedin.com/company/afmtelethon
• Instagraminstagram.com/telethon_france
• TikToktiktok.com/@telethon_france

Compliance2 records

Revenue estimate

Valuation estimate

Number of profiles

Profiles4 records

Subsidiaries10 records

Funding overview

Funding rounds

Investors

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about AFM-Telethon