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

  • STATUS
    Enrolling By Invitation
  • participants needed
    24
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Updated on 19 February 2024

Summary

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.

Details
Condition Achromatopsia
Age 99years or below
Clinical Study IdentifierTBD
Last Modified on19 February 2024

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