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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Gene therapy offers hope for rare blindness
Disease control OngoingThis study tests a gene therapy called LX101 for people with a rare inherited eye disease that causes blindness. 30 participants aged 6 and older were randomly assigned to receive the therapy or no treatment. The goal is to see if the treatment improves functional vision and ligh…
Matched conditions: INHERITED RETINAL DYSTROPHY ASSOCIATED WITH RPE65 MUTATIONS
Phase: PHASE3 • Sponsor: Innostellar Biotherapeutics Co.,Ltd • Aim: Disease control
Last updated May 17, 2026 13:29 UTC
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Gene therapy offers hope for rare eye disease
Disease control OngoingThis early-stage study tests a gene therapy called LX101 for people with a rare inherited eye disease that causes vision loss. The therapy uses a harmless virus to deliver a working copy of the RPE65 gene to the retina. Nine participants aged 6 and older will be monitored for saf…
Matched conditions: INHERITED RETINAL DYSTROPHY ASSOCIATED WITH RPE65 MUTATIONS
Phase: PHASE1 • Sponsor: Innostellar Biotherapeutics Co.,Ltd • Aim: Disease control
Last updated May 17, 2026 13:26 UTC