First clinical results of a Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy presented at ESGCT Congress

Genethon announced today the first positive clinical results obtained with the on-going ATA-001 Phase 1b/2b gene therapy clinical trial for the treatment of fukutin-related protein (FKRP) limb-girdle muscular dystrophy (LGMD2I/R9). These results were presented on October 27 at the 30th ESGCT congress in Brussels by Atamyo Therapeutics, a biotechnology company created by Genethon to accelerate the development of gene therapy for limb-girdle muscular dystrophy.
This new milestone is the result of 30 years of pioneering research conducted by Isabelle Richard, Ph.D, head of our Progressive Muscular Dystrophies team at Genethon.

ATA-100 is a one-time gene therapy for the treatment of fukutin-realted protein (FKRP) limb-girdle muscular dystrophy . ATA-100 is being evaluated in a multi-center phase 1b/2b in Denmark, France and UK. The aim of the trial is to assess the safety and efficacy of a gene therapy combining an AAV vector and the FKRP gene.
Preliminary results in the first cohort (3 patients treated) show:
• No unexpected safety signal identified,
• Marked decline in levels of creatine kinase (a serum biomarker of muscular dystrophy) in all three patients,
• Improved velocity, sustained at one year,
• Disappearance of symptoms (cramps, muscle pain) and improved quality of life,
• Correction of centronucleation (a histological feature of muscular dystrophy) and evidence of transgene expression on the 3-month muscle biopsy.

Pr. John Vissing, principal investigator of the study, noted that “the preliminary results from cohort 1 with the first dose tested already show encouraging results from a safety and efficacy perspective. ATA-100 treatment has a life-changing potential in an indication where there is no approved treatment’.

Atamyo also announced that Data Safety Monitoring Board (DSMB) authorized the enrollment of the second dose cohort of the ATA-001 clinical trial.
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