AGTC-501 Gene Therapy Offers Vision Gains for X-Linked RP (Ophthalmology Times)

AGTC-501 Gene Therapy Offers Vision Gains for X-Linked RP

AGTC-501 (Applied Genetic Technologies Corporation), a novel gene therapy in development for the potential treatment of X-linked retinitis pigmentosa (RP), showed an acceptable safety profile and visual function improvements when administered in the study’s higher dose groups, according to Robert A. Sisk, MD, FACS, from the Cincinnati Eye Institute in Ohio.

Patients with X-linked RP, an inherited retinal disease that begins in childhood, are characterized by night blindness, progressive loss of peripheral vision, and eventual loss of central vision. Affected patients have access to only 1 approved treatment, voretigene neparvovec-rzyl (Luxturna; Spark Therapeutics) for RPE65, which represents about 1% of RP.

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