First clinical results of a gene therapy for the treatment of FKRP-related LGMD

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« adeno-associated virus » , ou virus adéno-associé est un petit virus à ADN simple brin. Il fait partie de la famille des Parvoviridae et appartient au genre des Dependovirus. La particule virale est constituée d’un brin d’ADN de polarité positive ou négative protégé par une capside. La taille moyenne d’une particule d’AAV et de 18 à 22 nm. Les AAV sont les seuls parvovirus non autonomes. Lorsqu’on emploie « rAAV » , il s’agit du virus AAV recombinant, c’est-à-dire qu’il a été modifié pour devenir un vecteur (et n’est donc plus virulent). 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