Drug Profile

OPGx 001

Alternative Names: AAV8.hLCA5; LCA5-LCA; OPGx-001; OPGX-LCA5

Latest Information Update: 14 May 2025

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At a glance

Originator University of Pennsylvania
Developer Opus Genetics
Class Eye disorder therapies; Gene therapies
Mechanism of Action Gene transference; LCA5 protein replacements

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

Highest Development Phases

Phase I/II Leber congenital amaurosis

Most Recent Events

06 May 2025 OPGx 001 receives Regenerative Medicine Advanced Therapy (RMAT) status for Leber congenital amaurosis in USA
05 May 2025 Efficacy and adverse event data from a phase I/II trial in Leber congenital amaurosis released by Opus Genetics
08 Apr 2025 Opus Genetics announces intention to submit BLA for Leber congenital amaurosis

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