Atamyo Therapeutics, a biotechnology company focused on the development of new-generation gene therapies targeting neuromuscular diseases, announced multiple major milestones for ATA-100 and ATA-200, its one-time gene-replacement therapies for the treatment of limb-girdle muscular dystrophy types 2I/R9 and 2C/R5 (LGMD2I/R9 and LGMD2C/R5 respectively), as well as the reinforcement of its management team.
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Authorization of Clinical Trial Application in France for ATA-100
A third authorization of a Clinical Trial Application (CTA) in Europe was granted by the French National Medicines Health Agency (ANSM) for ATA-100. Two CTA approvals had previously been granted respectively by the United Kingdom Medicines & Healthcare products Regulatory Agency (MHRA) and by the Denmark Danish Medicines Agency (DKMA). ATA-100, a single-administration gene therapy candidate for LGMD2I/R9, delivers a normal copy of the gene for production of FKRP proteins. LGMD2I/R9 is a rare genetic disease caused by mutations in the gene that produces fukutin-related protein (FKRP) and affects an estimated 5,000 people in the US and Europe.
Orphan Drug Designation awarded from European EMA for ATA-200
ATA-200, Atamyo’s one-time gene replacement therapy for the treatment of LGMD 2C/R5, has been granted Orphan Drug Designation by the European Medicines Agency (EMA) for the treatment of LGMD. Orphan Drug Designations by the EMA grants a ten-year market exclusivity in Europe and provides with other benefits such as tax credits, protocol assistance and research grants.
The therapy is based on the research of Atamyo Chief Scientific Officer Isabelle Richard, Ph.D., Research Director at CNRS who heads the Progressive Muscular Dystrophies Laboratory at Genethon.
“We are thrilled by this additional approval for our first-in-human trial with ATA-100 and by the Orphan Drug Designation in Europe for ATA-200. These single-administration treatments bring hope to patients with LGMD2I/R9 and LGMD2C/R5,” said Stéphane Degove, CEO of Atamyo Therapeutics.