INHERITED RETINAL DYSTROPHY ASSOCIATED WITH RPE65 MUTATIONS
Clinical trials for INHERITED RETINAL DYSTROPHY ASSOCIATED WITH RPE65 MUTATIONS explained in plain language.
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One-Time gene injection aims to restore sight in rare eye disease
Disease control OngoingThis phase 3 trial tests a gene therapy called LX101 in 30 people with inherited retinal dystrophy caused by RPE65 mutations. Participants receive a single injection of LX101 into the eye, while a control group gets no treatment. The study measures changes in functional vision an…
Matched conditions: INHERITED RETINAL DYSTROPHY ASSOCIATED WITH RPE65 MUTATIONS
Phase: PHASE3 • Sponsor: Innostellar Biotherapeutics Co.,Ltd • Aim: Disease control
Last updated Jun 27, 2026 09:10 UTC
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Experimental gene therapy aims to restore sight in rare eye disease
Disease control OngoingThis early study tests a gene therapy called LX101 in 9 people with a rare inherited eye disease caused by RPE65 gene mutations. The therapy delivers a working copy of the gene into the retina via a single injection. The main goals are to check safety and see if it can improve vi…
Matched conditions: INHERITED RETINAL DYSTROPHY ASSOCIATED WITH RPE65 MUTATIONS
Phase: PHASE1 • Sponsor: Innostellar Biotherapeutics Co.,Ltd • Aim: Disease control
Last updated Jun 27, 2026 09:10 UTC