PRPF31 MUTATIONASSOCIATED RETINAL DYSTROPHY

Clinical trials for PRPF31 MUTATIONASSOCIATED RETINAL DYSTROPHY explained in plain language.

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Completed 1

New eye injection shows promise for rare Blindness-Causing gene mutation

Disease control Completed

This early-stage study tested a new drug, VP-001, given as an injection into the eye to see if it is safe for people with a rare inherited eye disease caused by a PRPF31 gene mutation. Seventeen adults took part, and the main goal was to check for any side effects. The study did …

Matched conditions: PRPF31 MUTATIONASSOCIATED RETINAL DYSTROPHY

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

Last updated May 13, 2026 15:58 UTC

PRPF31 MUTATIONASSOCIATED RETINAL DYSTROPHY

Get alerted when new PRPF31 MUTATIONASSOCIATED RETINAL DYSTROPHY trials appear

New eye injection shows promise for rare Blindness-Causing gene mutation