A Multiple-Site Phase 1/2 Safety and Efficacy Trial of AGTC 402 a Recombinant Adeno-associated Virus Vector Expressing CNGA3 in Patients with Congenital Achromatopsia Caused by Mutations in the CNGA3 Gene

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Study Overview

Achromatopsia is an inherited retinal disorder characterized by markedly reduced visual acuity, nystagmus, photoaversion (intolerance to bright light), a small central scotoma, eccentric viewing, and complete or severe loss of color discrimination. The genetic mutations of the disease can be established in the majority of individuals. Approximately 25% are caused by mutations in the cone-specific cyclic nucleotide gated channel alpha subunit (CNGA3) gene.

  • Study Identifier: 826563

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