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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Early gene therapy trial aims to slow vision loss in rare eye disease
Disease control OngoingThis is an early-stage study testing a one-time gene therapy called LX101 for people with a rare inherited eye disease that causes progressive vision loss. The goal is to see if the treatment is safe and if it can improve or stabilize vision. Nine participants, aged 6 and older w…
Matched conditions: INHERITED RETINAL DYSTROPHY ASSOCIATED WITH RPE65 MUTATIONS
Phase: PHASE1 • Sponsor: Innostellar Biotherapeutics Co.,Ltd • Aim: Disease control
Last updated Apr 01, 2026 14:42 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