NEW YORK – Atamyo Therapeutics on Wednesday announced it has received permission from the US Food and Drug Administration to begin studying the gene therapy candidate ATA-100 in patients with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9).
The FDA cleared the company's investigational new drug application for ATA-100, previously known as GNT0006, which will allow investigators to proceed with a Phase Ib/IIb trial in the US. ATA-100 is already being evaluated as a treatment for LGMD2I/R9 in a multicenter Phase Ib/IIb trial underway in Denmark, France, and the UK.
There aren't any approved curative treatments for LGMD2I/R9. The rare muscular dystrophy disorder is caused by mutations in the FKRP gene, which produces the fukutin-related protein (FKRP). ATA-100 is designed to deliver a normal copy of that gene.
"This IND clearance [is] an important step to bring ATA-100 to US patients suffering from this highly debilitating LGMD-R9 disease," Atamyo Chief Medical Officer Sophie Olivier said in a statement. "We are looking forward to opening US centers in the near future for this clinical trial."
Evry, France-based Atamyo is a spinoff of Genethon, a company focused on developing gene therapies for rare diseases.