Following several proofs of concept, Atamyo Therapeutics, a spin-off from Geenethon, has obtained authorisation to start a new gene therapy trial in patients suffering from limb-girdle muscular dystrophy (LGMD) R5 linked to SGCG (gamma-sarcoglycanopathy). Its aim is to test the safety, pharmacodynamics and efficacy of ATA-200, a product resulting from the work of Isabelle Richard, a researcher at Genethon. Approved to take place in France (at I-motion) and Italy, this new multicentre phase I/II trial will include :
- six walking children aged between 6 and 12 with a clinically and genetically confirmed diagnosis of LGMD R5 ;
- two cohorts, each receiving a single intravenous injection of ATA-200, the first at 1 x 1014 vg/Kg and the second at a dose three times higher;
- an assessment of the effects of the product at six months;
- follow-up of patients for four and a half years after the evaluation phase.
Recruitment is due to begin in May this year, with the first injections scheduled for the third quarter of 2024.
Atamyo Therapeutics obtient les autorisations réglementaires en Europe pour lancer l’essai clinique de ATA-200, sa thérapie génique pour traiter la myopathie des ceintures LGMD-2C/R5. Atamyo Therapeutics. 26 March 2024 press release.
On Clinicaltrials.gov website: NCT05973630.
