LEBER CONGENITAL AMAUROSIS
Clinical trials for LEBER CONGENITAL AMAUROSIS explained in plain language.
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Can a single injection restore sight in a rare childhood blindness?
⭐️ CURE ⭐️ OngoingThis trial tests a gene therapy called ATSN-101, given as a one-time injection under the retina, for people with Leber congenital amaurosis caused by GUCY2D gene mutations. The goal is to see if the treatment is safe and can improve vision. Participants receive the therapy in one…
Matched conditions: LEBER CONGENITAL AMAUROSIS
Phase: PHASE1, PHASE2 • Sponsor: Atsena Therapeutics Inc. • Aim: ⭐️ CURE ⭐️
Last updated Aug 05, 2026 00:00 UTC
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Can a single eye injection restore sight in genetic blindness?
⭐️ CURE ⭐️ OngoingThis trial tests an experimental gene therapy called OCU400 in people with retinitis pigmentosa or Leber congenital amaurosis, which are inherited conditions that cause progressive vision loss and can lead to blindness. The therapy is given as a single injection into the eye, wit…
Matched conditions: LEBER CONGENITAL AMAUROSIS
Phase: PHASE1, PHASE2 • Sponsor: Ocugen • Aim: ⭐️ CURE ⭐️
Last updated Aug 02, 2026 00:00 UTC