OPA1 GENE MUTATION

Clinical trials for OPA1 GENE MUTATION explained in plain language.

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First patients receive experimental gene therapy injection for rare inherited blindness

Disease control Ongoing

This is the first study in people to test the safety of an experimental drug called PYC-001 for a rare, inherited eye disease that causes progressive vision loss. Up to 18 adults with a confirmed OPA1 gene mutation will receive a single injection of the drug into one eye. The mai…

Matched conditions: OPA1 GENE MUTATION

Phase: PHASE1 • Sponsor: PYC Therapeutics • Aim: Disease control

Last updated Mar 30, 2026 14:29 UTC

OPA1 GENE MUTATION

Get alerted when new OPA1 GENE MUTATION trials appear

First patients receive experimental gene therapy injection for rare inherited blindness