Skip to main content
Premium Trial:

Request an Annual Quote

Atamyo Therapeutics to Start Limb-Girdle Muscular Dystrophy Gene Therapy Trial in Italy, France

NEW YORK – Atamyo Therapeutics on Tuesday said that regulators in Italy and France have granted it permission to begin a clinical trial of the gene therapy ATA-200 in patients with gamma-sarcoglycan-related limb-girdle muscular dystrophy type 2C/R5 (LGMD-2C/R5).

The Italian Medicines Agency and the French Medicines Agency are the first regulators to authorize the Evry, France-based gene therapy developer's clinical trial application in Europe for a Phase Ib open-label, dose-escalation study of ATA-200. In the trial, Atamyo will evaluate the safety, pharmacodynamics, efficacy, and immunogenicity of the gene therapy candidate in children.

"This is a significant milestone for LGMD-2C/R5 patients and for Atamyo as ATA-200 is the first treatment targeting LGMD-2C/R5 to enter clinical trials," Atamyo CEO Stéphane Degove said in a statement. LGMD-2C/R5 is a rare disease caused by mutations in the SGCG gene and is characterized by progressive muscular weakness.

ATA-200 is a single-dose gene therapy administered to patients intravenously and uses an adeno-associated virus vector to deliver a functional copy of a SGCG transgene. 

The company expects to begin dosing patients with the gene therapy in the Phase Ib trial in Q3 2024.