What Atamyo Therapeutics does

Atamyo Therapeutics is a clinical-stage biopharmaceutical company founded in 2020 as a spin-off from Genethon, focused on developing AAV-based gene replacement therapies for limb-girdle muscular dystrophies (LGMD), a group of rare genetic neuromuscular diseases for which no curative treatments exist. The company is headquartered in Évry Courcouronnes, in the Paris region of France, and operates with a small team (1–10 employees) under the leadership of co-founders Stéphane Degove (CEO) and Isabelle Richard (CSO), with Sophie Olivier as Chief Medical Officer and Catherine Cancian as Chief Technology Officer. Gene therapy pioneer Roger J. Hajjar, MD, joined the Board of Directors in 2023.

The company's pipeline consists of two clinical-stage programs: ATA-100, a single-administration AAV gene therapy delivering a normal copy of the FKRP gene for the treatment of LGMD type 2I/R9 (currently in Phase 1b/2b with a selected pivotal dose of 9E12 vg/kg), and ATA-200, a single-administration AAV gene therapy delivering the human γ-sarcoglycan (SGCG) transgene for LGMD type 2C/R5 (currently in Phase 1b/2). Both candidates are built on proprietary AAV vectors developed through Genethon's 30+ years of muscle-directed gene therapy research and are administered as single intravenous injections designed to produce durable, potentially lifelong expression of the therapeutic protein. Early clinical data show over 90% muscle fiber expression of therapeutic protein, significant reductions in muscle damage biomarkers, and clinical functional improvements (e.g., +5% sitting FVC, +19% 10-meter walk velocity, +16–23% quality-of-life scores at 12 months for ATA-100) with no serious adverse events reported across the four ATA-200 patients dosed to date.

Atamyo is pre-revenue and finances operations through non-dilutive public funding (Bpifrance, France 2030, European Innovation Council Accelerator) and patient advocacy partnerships (The Dion Foundation for Children with Rare Diseases, CureSCG), supplemented by academic clinical collaborations with Copenhagen Neuromuscular Center, Newcastle University's John Walton Muscular Dystrophy Research Centre, and the University of Florida Powell Gene Therapy Center. Its target patient populations are small but well-defined ultra-rare disease groups — approximately 5,000 LGMD-R9 patients and 2,000 LGMD-R5 patients in Europe and the US. Both programs have received Orphan Drug Designation from the FDA and EMA, and ATA-100 additionally holds Rare Pediatric Disease and Fast Track Designations from the FDA. The company's commercial revenue model will likely rely on premium one-time pricing for gene therapy upon regulatory approval, distributed through specialty pharmacy and hospital-based channels.

Name

Atamyo Therapeutics

Legal name

Atamyo Therapeutics SAS

Website

https://atamyo.com

Company type

Private

Founded year

2020

Operating status

Operating

Headcount range

1–10 employees

Short description

Atamyo Therapeutics is a clinical-stage biopharmaceutical company developing single-administration AAV-based gene replacement therapies for limb-girdle muscular dystrophies (LGMD-R9 and LGMD-R5), spun off from Genethon and headquartered near Paris, France.

Ownership category

akta.pro rank

Product category

Gene Therapy

NAICS

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

SIC

Services-Commercial Physical & Biological Research (8731)

akta.pro primary industry

AAV Vector Gene Therapy Developers (HLAAACAA)

akta.pro secondary industry

Gene Therapy for Rare/Monogenic Diseases (HLAAACAE)

Keywords

• Gene therapy development
• Neuromuscular disease treatment
• AAV gene delivery
• Muscular dystrophy therapy
• Rare disease biopharma

Headquarters

HQ city

Paris

HQ country

France

HQ region

Europe

Offices1 record

Markets served

GTM type

B2C

Offering type

Hardware or Manufacturing

Cost components

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

Revenue model

1. Gene Therapy Commercialization: Pre-revenue clinical-stage company. Currently has no approved products. Revenue will be generated from commercialization of gene therapy candidates (ATA-100 and ATA-200) upon regulatory approval. Gene therapies for rare diseases typically command premium pricing as one-time treatments.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels4 records

Core offering

Atamyo Therapeutics develops single-administration AAV-based gene replacement therapies for limb-girdle muscular dystrophies (LGMD). Its two clinical-stage candidates — ATA-100 (delivering the FKRP gene for LGMD-R9) and ATA-200 (delivering the SGCG gene for LGMD-R5) — are administered as single intravenous injections designed to provide potentially lifelong, curative treatment for ultra-rare neuromuscular diseases. The platform leverages proprietary AAV vectors developed by parent organization Genethon.

Product overview

Atamyo Therapeutics is a clinical-stage biopharma company developing a new generation of AAV-based gene replacement therapies for limb-girdle muscular dystrophies (LGMDs). The company's portfolio consists of two clinical-stage gene therapy products: ATA-100 for LGMD-R9 (FKRP-related) and ATA-200 for LGMD-R5 (gamma-sarcoglycanopathy), both delivered as single intravenous injections using AAV vectors. The technology platform is built on expertise from spin-off parent Genethon, leveraging over 30 years of research in muscular dystrophies and AAV gene therapy development.

Differentiator

Problem solved

Functional benefit

Products and services

• ATA-100 Single-administration AAV gene therapy delivering a normal copy of the FKRP gene to produce fukutin-related protein for treatment of limb-girdle muscular dystrophy type 2I/R9 (LGMD-R9). Administered as a single intravenous injection at 9E12 vg/kg dose. Currently in Phase 1b/2b clinical development with demonstrated 5% improvement in sitting FVC, 19% velocity improvement in 10M Walk Test, and 16-23% quality-of-life score increases at 12 months.
• ATA-200 Single-administration AAV gene therapy delivering a normal copy of the SGCG gene to produce gamma-sarcoglycan protein for treatment of limb-girdle muscular dystrophy type 2C/R5 (LGMD-R5 / gamma-sarcoglycanopathy). Administered as a single intravenous injection at 1.0E+14 vg/kg dose. Currently in Phase 1b/2 clinical development with over 90% muscle fiber expression of therapeutic SGCG protein (90.2% and 92.1%) demonstrated in first patients and no serious side effects observed across four patients dosed.

Quantifiable outcome

• Over 90% muscle fiber expression of therapeutic SGCG protein (90.2% and 92.1% for first two patients)
• +4 more outcomes

Named customers1 record

Segments1 record

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

Six partnerships are on record, tiered core and supporting.

• GenethoncoreStrategic or Co-development PartnerAtamyo is a spin-off of Genethon, the pioneer and world leader in gene therapy R&D. Leverages Genethon's 30+ years of expertise in AAV-based gene therapy and muscular dystrophies from the Progressive Muscular Dystrophies Laboratory. Isabelle Richard, Atamyo's Chief Scientific Officer, leads the Progressive Muscular Dystrophies Laboratory at Genethon. Atamyo benefits from Genethon's breakthrough technologies and manufacturing capabilities.
• Powell Gene Therapy Center, University of FloridacoreStrategic or Co-development PartnerClinical trial site for ATA-200 LGMD-R5 Phase 1b/2 study. Dr. Barry Byrne, Associate Chair of Pediatrics and Director of the Powell Gene Therapy Center, serves as principal investigator. First patients treated at this center in Gainesville, Florida.
• Copenhagen Neuromuscular Center, Rigshospitalet (Denmark)coreStrategic or Co-development PartnerClinical trial site for ATA-100 LGMD-R9 Phase 1/2 study. Pr. John Vissing, Director of Copenhagen Neuromuscular Center, serves as principal investigator. First European patient treated at this site.
• John Walton Muscular Dystrophy Research Centre, Newcastle University (UK)coreStrategic or Co-development PartnerClinical trial site for ATA-100 LGMD-R9 study. Pr. Volker Straub, Professor of Medicine and Director of the center, involved in clinical trial execution.
• European Medicines Agency (EMA)supportingOthersRegulatory authority that granted Orphan Drug Designation to both ATA-100 and ATA-200. Clinical Trial Applications reviewed and approved in multiple European countries.
• US Food and Drug Administration (FDA)supportingOthersUS regulatory authority that granted Orphan Drug Designation, Rare Pediatric Disease Designation, and Fast Track Designation to ATA-100 and ATA-200. IND for ATA-200 cleared to proceed in November 2024.

Scale indicators4 records

Recent moves6 records

Expansion highlights6 records

Emerging players

• Taysha Gene Therapies: Clinical-stage AAV gene therapy developer focused on rare monogenic CNS and neuromuscular diseases. Comparable on AAV platform, rare disease strategy, and clinical-stage profile targeting small patient populations.
• LEXEO Therapeutics: Clinical-stage gene therapy company with programs in cardiac and neuromuscular diseases using AAV vectors. Comparable on AAV gene therapy modality, focus on rare monogenic muscle/cardiac targets, and similar development stage.
• Abeona Therapeutics: Clinical-stage gene therapy company with AAV and other modalities targeting rare diseases including neuromuscular indications. Comparable on small-cap gene therapy profile and rare disease development strategy.

Direct peers

• Sarepta Therapeutics: Commercial-stage leader in gene therapy for Duchenne muscular dystrophy (Elevidys) and other neuromuscular diseases. Most directly comparable as a muscular dystrophy AAV gene therapy developer pursuing premium-priced, one-time treatments via similar regulatory pathways (Orphan, Fast Track).
• Solid Biosciences: Clinical-stage gene therapy developer focused on Duchenne muscular dystrophy and other neuromuscular disorders using AAV vectors. Directly comparable as a small-cap AAV gene therapy biotech targeting rare muscular dystrophies.
• Kinea Bio: Early-stage gene therapy developer focused on LGMD and other rare neuromuscular diseases using AAV vectors. Most directly comparable as a small, clinical-stage AAV gene therapy peer competing in the same LGMD space.

Broad incumbents

• Regenxbio: Clinical and commercial-stage AAV gene therapy platform company with internal and partnered programs in neuromuscular and metabolic diseases (including DMD and LGMD via partnerships). Comparable on AAV delivery technology and platform approach to rare disease gene therapy.
• Pfizer: Large pharma with a DMD gene therapy franchise (Beqvez) and broader AAV gene therapy R&D. Comparable on neuromuscular gene therapy indication overlap and large-pharma strategic acquirer profile relevant to Atamyo.
• Ultragenyx Pharmaceutical: Commercial-stage rare disease company with a growing gene therapy pipeline (including AAV-based programs). Comparable on rare-disease focus, AAV modality, and strategy of pursuing premium-priced treatments for ultra-rare indications.

Others

• Genethon: Atamyo's parent organization and a global leader in AAV gene therapy R&D for neuromuscular diseases. Comparable on AAV platform and LGMD scientific leadership, while also serving as technology and manufacturing partner.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks5 records

Key highlights7 records

Customer concentration

• Xx.com/AtamyoTX
• LinkedInlinkedin.com/company/atamyo-therapeutics

Compliance7 records

Revenue estimate

Valuation estimate

Number of profiles

Profiles5 records

Funding overview

Funding rounds2 records

Investors1 record

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M&A

Investments

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

Frequently asked questions about Atamyo Therapeutics