Following several proofs of concept, Atamyo Therapeutics, a spin-off from Geenethon, is 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.
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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.
