The initial results of the ATA-100 gene therapy clinical trial for the treatment of limb-girdle muscular dystrophy linked to the FKRP gene (LGMD2I/R9) were presented at the ESGCT congress in Brussels on 27 October.
This multicentre Phase 1b/2b trial is designed to assess the safety and efficacy of a gene therapy combining an AAV vector and the FKRP gene, administered intravenously as a single injection. It is being conducted in Denmark, France and the UK by Atamyo Therapeutics, a biotechnology company set up by Genethon to accelerate the development of gene therapy for limb-girdle muscular dystrophy.
In France, the patients included in the trial are being monitored by Dr Tanya Stojkovic at I-Motion Adultes, the Institute of Myology’s adult clinical research platform directed by Dr Giorgia Querin.
Preliminary results from the first cohort (3 patients treated) show :
- an absence of unexpected side effects,
- a marked drop in creatine kinase levels (serum biomarker of muscular dystrophy) in all patients,
- an improvement in walking speed, maintained after one year,
- the disappearance of symptoms (cramps, muscle pain) and an improvement in quality of life,
- correction of centronucleation (a histological feature of muscular dystrophy) and evidence of transgene expression on muscle biopsy at 3 months.
Recruitment of the 2nd cohort, which will receive a treatment dose three times higher, has begun.
Find out more about the LGMD trials conducted at the Institute
Read Genethon news First clinical results of a Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy presented at ESGCT Congress
Download Atamyo Therapeutics press release, 27 October 2023, First clinical results of ATA-100, a Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9), presented at ESGCT
