The first European gene therapy trial in limb-girdle muscular dystrophy linked to the FKRP gene (LGMD2I/R9) has just started. This is a multicentre phase 1-2 study evaluating the safety, pharmacodynamics, efficacy and immunogenicity of GNT0006, an adeno-associated virus (AAV) vector carrying the human FKRP transgene. This study will consist of two phases: an open-label dose escalation phase (Phase 1) and a randomised double-blind placebo-controlled phase (Phase 2), both with a long-term follow-up period (LTFU).
The trial will take place in three countries, Denmark, Great Britain and France, where patients will receive the drug candidate and be monitored at the Institute of Myology by Dr Tanya Stojkovick.
Conducted by Atamyo Therapeutics, a biotechnology company created by Genethon to accelerate the development of gene therapy for limb-girdle muscular dystrophy, this trial is the result of nearly 30 years of excellent research conducted by the team of Isabelle Richard, research director at Genethon.
Further details on this trial: “GNT0006 Gene Therapy Trial in Patients With LGMDR9” NCT05224505
