AFM-Telethon
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.
- Company typePrivate
- Founded1958
- HeadquartersBas Évry, France
- Headcount251–500
- GTM typeB2C
- OfferingServices
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.
AFM-Telethon firmographics
Firmographics- 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
AFM-Telethon industry classification
Industry- 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
Where AFM-Telethon is headquartered
LocationHeadquarters
- HQ city
- Bas Évry
- HQ country
- France
- HQ region
- Europe
Offices10 records
Markets served
AFM-Telethon business model
Business model- GTM type
- B2C
- Offering type
- Services
- Cost components
- Personnel, Technology or R&D, Operations, Marketing or Sales, Infrastructure, Others
Revenue model
- 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.
- Legs and donations (inheritance/transmission): AFM-Téléthon receives legacy donations and property bequests from donors, which are a significant component of long-term fundraising.
- 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
| Model | Billing | Price |
|---|---|---|
| Subscription | Monthly | Donation tiers with tax incentive |
Go-to-market motion1 record
Distribution channels6 records
Marketing channels9 records
AFM-Telethon product offering
Product offeringCore 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
Companies that use AFM-Telethon
Customer profileNamed customers6 records
Segments5 records
Ideal customer profiles4 records
AFM-Telethon technology and API
TechnologyTechnology focussed Yes
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature6 records
AFM-Telethon partnerships and signals
Strategic signalPartnerships
21 partnerships are on record, tiered minor and core.
- AEFE (Agence pour l'Enseignement Français à l'Étranger) and MLF (Mission Laïque Française)minorAFM-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)minorAFM-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)coreGenoTher 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)minorAFM-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)minorAFM-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)minorAFM-Téléthon participates in Screen4Care, a European consortium dedicated to rare disease diagnosis including neonatal screening pilot projects.
- REMEDi4ALL (EU consortium for drug repositioning)minorAFM-Téléthon is a member of REMEDi4ALL, a European consortium dedicated to drug repositioning for rare diseases.
- ERDERA (European Rare Disease Research Alliance)minorERDERA 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)coreAFM-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).
- AmpleiacoreAmpleia 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)coreAFM-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 LuxembourgminorAFM-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 NetworkminorAFM-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 HandicapminorAFM-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.
- YposKesicoreYposKesi 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 BTPminorPRO 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 BiologicsminorGenSight 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, RibogenicsminorFive 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 TherapeuticsminorAtamyo 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)coreInserm 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.
- GenoSafecoreGenoSafe 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
AFM-Telethon competitors and assessment
Company assessmentDirect 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
AFM-Telethon social profiles
Digital presenceAFM-Telethon compliance and trust
Trust signalCompliance2 records
AFM-Telethon financial estimates
Financial estimateRevenue estimate
Valuation estimate
AFM-Telethon leadership team
Management profileNumber of profiles
Profiles4 records
AFM-Telethon subsidiaries and ownership
Company hierarchySubsidiaries10 records
AFM-Telethon funding detail
Funding detailFunding overview
Funding rounds
Investors
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
AFM-Telethon M&A and investment
M&A and investmentM&A
Investments
M&A and investment is available on the Subscription and Enterprise plan.Contact sales →
Frequently asked questions about AFM-Telethon
What does AFM-Telethon do?
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.
Is AFM-Telethon a public or private company?
AFM-Telethon is a private company. It is classified as nonprofit foundation owned and is currently operating.
When was AFM-Telethon founded?
AFM-Telethon was founded in 1958. It employs 251 to 500 people.
Where is AFM-Telethon based?
AFM-Telethon is headquartered in Bas Évry, France, in the Europe region.
How does AFM-Telethon make money?
Three revenue lines are on record. Public donations and Telethon fundraising is the primary driver. The others are legs and donations (inheritance/transmission) and government and public funding.
Who are AFM-Telethon's main competitors?
Direct peers on record are Fondation Maladies Rares, Muscular Dystrophy Association (MDA), Parent Project Muscular Dystrophy (PPMD), EURORDIS - Rare Diseases Europe, National Organization for Rare Disorders (NORD) and Cystic Fibrosis Foundation. Emerging players are GenSight Biologics and Atamyo Therapeutics. Broad incumbents are Wellcome Trust and Inserm (Institut national de la santé et de la recherche médicale).
Does AFM-Telethon have an API?
No public API is recorded for AFM-Telethon.
What industry is AFM-Telethon in?
AFM-Telethon's product category is Nonprofit Rare Disease Research and Patient Support. Its primary akta.pro industry code is BPAGACAA, Disease-Specific Research & Support (e.g., Cancer, Diabetes, ALS), with a secondary code of HLAKAIAO, Neurogenetics & Rare Neurologic Diseases. Its NAICS code is 541714 and its SIC code is 8071.