These companies have a known or expressed interest in LGMD2I.
Atamyo
As the first spin-off of Généthon in developing gene therapy, Atamyo’s most advanced programs address different forms of limb-girdle muscular dystrophies (LGMD) based on the pioneering research of Pr. Isabelle Richard in identifying both the genes and the mechanisms responsible for many of these diseases.
- Gene therapy (ATA-100): Atamyo’s most advanced program is in LGMD2I. A clinical trial (Phase I/II) was started in 2022 in 3 European clinical centers (ClinicalTrials.gov: NCT05224505).
ML Bio
ML BIO is a subsidiary of BridgeBio, a company whose mission is to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases.
- Sugar-based therapy (BBP-418): FORTIFY (ClinicalTrials.gov: NCT05775848) is a global, randomized, double‑blind, placebo‑controlled Phase 3 registrational study evaluating the safety and efficacy of BBP‑418 in people with LGMD2I/R9. The trial is active in the US, Europe, and Australia.
AskBio
The mission of AskBio is to advance genetic technology and life-saving gene therapy, with the goal to erase genetic diseases. It was founded over 20 years ago based on the pioneering work of the scientific co-founder, Jude Samulski, PhD, who was the first to demonstrate that adeno-associated virus (AAV) could be cloned for therapeutic purposes.
- Gene therapy (AB-1003): A clinical trial (Phase I/II) in LGMD2I was started in 2024 in US and European clinical centers (ClinicalTrials.gov: NCT05230459).
Edgewise Therapeutics
Its mission is to bring innovative novel therapeutics for serious skeletal and cardiac muscle diseases.
- Protein-based therapy (EDG-5506): A multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of sevasemten in adults with Becker (ClinicalTrials.gov: NCT05291091).