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Atamyo Therapeutics Seeks OK to Start Limb-Girdle Muscular Dystrophy Gene Therapy Trial in Europe

NEW YORK – Atamyo Therapeutics on Tuesday said it has filed a clinical trial application in Europe for ATA-200, its gene therapy candidate for gamma-sarcoglycan related limb-girdle muscular dystrophy type 2C/R5 (LGMD2C/R5).

LGMD2C/R5 is a rare genetic disease that leads to progressive muscular weakness and is caused by mutations in the SGCG gene, which produces the protein gamma-sarcoglycan.

Evry, France-based Atamyo is planning a multicenter, open-label, dose-escalation Phase Ib clinical trial to evaluate the safety, pharmacodynamics, and efficacy of ATA-200, which delivers a functional copy of a SGCG transgene. The single-dose gene therapy is administered intravenously and delivered with an adeno-associated virus vector.

Atamyo hopes to start dosing patients in the first half of next year.

"No curative treatment exists for this disease," said Sophie Olivier, Atamyo's chief medical officer, in a statement. "LGMD-R5 is the most rapidly progressive and debilitating form of LGMDs with onset of symptoms in early childhood, a loss of ambulation before adulthood, and frequent cardiac impairment."

Atamyo on Tuesday also announced that it is eligible to receive up to €8 million ($8.6 million) of non-dilutive financing from France 2030, a public program managed by the French investment bank Bpifrance, to support the company's clinical trials and manufacturing development programs for ATA-200.

ATA-200 would be the second of Atamyo's gene therapies to enter the clinic. The first, ATA-100, a candidate for LGMD type 2I/R9, was cleared to start US clinical trials earlier this month.