Drug Profile

AAV8 RK RetGC

Alternative Names: AAV-RetGC; AAV8-RK-RetGC

Latest Information Update: 11 Mar 2025

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At a glance

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

No
New Molecular Entity No

22 Jan 2025 Investigation in Cone-rod-dystrophies in USA (Subretinal), prior to January 2025
22 Jan 2025 US FDA grants Rare Pediatric Disease Designation (RPDD) to AAV8 RK RetGC for Leber congenital amaurosis in USA

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