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MeiraGTx receives FDA Rare Pediatric Disease Designation for AAV8-RK-RetGC to treat Leber congenital amaurosis due to GUCY2D mutations. MeiraGTx Holdings Plc announced that the FDA has granted its ...

GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative ...

Credit: Getty Images. Visomitin is a cardiolipin peroxidation inhibitor designed to sustain and restore mitochondrial function and interrupt apoptosis in mitochondrial conditions. The Food and Drug ...

Please provide your email address to receive an email when new articles are posted on . Opus Genetics received rare pediatric disease designation from the FDA for its ocular gene therapy designed to ...

A research team has successfully demonstrated the world's first gene-editing treatment for Leber's hereditary optic neuropathy (LHON). The study was conducted in collaboration with the Seoul National ...

Please provide your email address to receive an email when new articles are posted on . The first cohort has finished dosing in the phase 1/2 clinical trial of OPGx-LCA5. The gene therapy, under ...

A Prescription Drug User Fee Act target date of February 28, 2026 has been set for the application. The Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) for ...

Researchers at the Eye Genetics Research Unit at Children's Medical Research Institute (CMRI) are the first in the world to use stem cells to study one of the genetic causes of Leber Congenital ...

PARIS--(BUSINESS WIRE)--Regulatory News: GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene ...

Researchers from the National Eye Institute (NEI) have developed a gene therapy that rescues cilia defects in retinal cells affected by a type of Leber congenital amaurosis (LCA), a disease that ...