Atamyo Therapeutics' ATA-200 Gene Therapy Receives FDA Clearance for US Clinical Trial in LGMD2C/R5
• The FDA has cleared Atamyo Therapeutics' IND application for ATA-200, a gene therapy for Limb-Girdle Muscular Dystrophy Type 2C/R5 (LGMD2C/R5).
• ATA-200 delivers a functional copy of the SGCG gene and has shown promise in preclinical studies, correcting symptoms and disease biomarkers in mice.
• The Phase 1b dose-escalation trial (NCT05973630) will now expand to the US, supported by funding from The Dion Foundation, with plans to open the first US center before year-end.
• ATA-200 has also been granted orphan drug designation by the FDA, highlighting the unmet need for treatments for this rare pediatric disease.
FDA Clears Atamyo's ATA-200 Gene Therapy Trial for LGMD2C/R5 in Children
• The FDA has approved Atamyo Therapeutics' Investigational New Drug application for ATA-200, a gene therapy for limb-girdle muscular dystrophy type 2C/R5 (LGMD2C/R5).
• ATA-200 is the first potential treatment for LGMD2C/R5 to enter clinical development in the U.S., offering hope for children with this debilitating condition.
• The Phase 1b trial will enroll approximately six children aged 6-13 across sites in the U.S., France and Italy, evaluating the safety and tolerability of ATA-200.
• ATA-200 delivers a healthy copy of the _SGCG_ gene to muscle cells, aiming to restore gamma-sarcoglycan protein production and improve muscle function in affected individuals.
Atamyo Therapeutics Advances Gene Therapy Programs for Limb-Girdle Muscular Dystrophy
• Atamyo Therapeutics completed recruitment for the dose escalation phase of the Phase 1b trial of ATA-100 for LGMD2I/R9.
• The company submitted an IND application to the FDA for ATA-200, a gene therapy for LGMD2C/R5, expanding its clinical trial to the U.S.
• Initial results from the ATA-100 trial show promising functional improvements and a marked decline in creatine kinase levels in treated patients.
• The FDA granted rare pediatric disease designation to ATA-200, highlighting the unmet need for treatments for these rare muscular dystrophies.
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