PRPF31 MUTATIONASSOCIATED RETINAL DYSTROPHY

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

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Completed 1

New eye drug shows promise for rare Blindness-Causing mutation

Disease control Completed

This early-stage study tested a new drug called VP-001, given as an injection into the eye, in 17 adults with a rare inherited retinal disease caused by a PRPF31 gene mutation. The main goal was to check if the treatment is safe and tolerable. The study did not aim to cure the di…

Matched conditions: PRPF31 MUTATIONASSOCIATED RETINAL DYSTROPHY

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

Last updated May 22, 2026 13:52 UTC

PRPF31 MUTATIONASSOCIATED RETINAL DYSTROPHY

Get alerted when new PRPF31 MUTATIONASSOCIATED RETINAL DYSTROPHY trials appear

New eye drug shows promise for rare Blindness-Causing mutation