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 injection aims to restore vision in rare blindness
Disease control OngoingThis early-stage study is testing a new gene therapy, called LX101, for people with a rare inherited eye disease that causes vision loss. The therapy involves a single injection into the eye to deliver a working copy of a faulty gene. Researchers are primarily checking if the tre…
Matched conditions: INHERITED RETINAL DYSTROPHY ASSOCIATED WITH RPE65 MUTATIONS
Phase: PHASE1 • Sponsor: Innostellar Biotherapeutics Co.,Ltd • Aim: Disease control
Last updated Apr 03, 2026 14:43 UTC
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Gene injection aims to restore vision in inherited blindness trial
Disease control OngoingThis study is testing whether a one-time gene therapy injection (called LX101) can improve vision in people with a specific inherited retinal disease that causes blindness. The trial involves 30 participants, aged 6 and older, who have vision loss due to mutations in the RPE65 ge…
Matched conditions: INHERITED RETINAL DYSTROPHY ASSOCIATED WITH RPE65 MUTATIONS
Phase: PHASE3 • Sponsor: Innostellar Biotherapeutics Co.,Ltd • Aim: Disease control
Last updated Apr 01, 2026 14:41 UTC