Leber Congenital Amaurosis
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CLINICAL_TRIALS research · 2026-03-17Clinical Trial Update: Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION) [Phase 3]
The document describes an ongoing Phase 3 clinical trial evaluating Sepofarsen for Leber congenital amaurosis (LCA) type 10, which is curren…
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EMA approval · 2026-02-16EMA Authorised: Luxturna (voretigene neparvovec) — Novartis Europharm Limited
The European Medicines Agency authorised Luxturna (voretigene neparvovec) from Novartis Europharm Limited for the treatment of inherited ret…
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CLINICAL_TRIALS research · 2025-08-06Clinical Trial Update: Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis [Phase 1, Phase 2]
Ocugen's OCU400 is being studied in a Phase 1/2 trial to assess safety and efficacy in patients with Retinitis pigmentosa and Leber congenit…
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CLINICAL_TRIALS trial_result · 2025-08-05Clinical Trial Update: Phase I Trial of Gene Vector to Patients With Retinal Disease Due to RPE65 Mutations [Phase 1]
Phase I trial update of an AAV2-based gene therapy delivering RPE65 for Leber congenital amaurosis due to RPE65 mutations; the study remains…
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CLINICAL_TRIALS research · 2025-06-18Clinical Trial Update: Psychotherapy Group for Parents of Children With LCA [NA]
This clinical trial update notes a psychotherapy group for parents of children with Leber congenital amaurosis, and indicates the study is n…
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CLINICAL_TRIALS trial_result · 2025-04-29Clinical Trial Update: Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2 [Phase 1, Phase 2]
A Phase 1/2 follow-on study of the AAV2-hRPE65v2 gene therapy vector for Leber congenital amaurosis (LCA2) is ongoing. The sponsor Spark The…
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CLINICAL_TRIALS research · 2025-03-27Clinical Trial Update: Inherited Retinal Degenerative Disease Registry
A Foundation Fighting Blindness–sponsored inherited retinal degenerative disease registry is currently recruiting participants to collect in…
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CLINICAL_TRIALS research · 2024-09-19Clinical Trial Update: Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR) [Phase 1, Phase 2]
A Phase 1/2 clinical trial evaluating HG004 for Leber congenital amaurosis due to RPE65 mutations is currently recruiting.
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CLINICAL_TRIALS research · 2024-09-19Clinical Trial Update: Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial(LIGHT) [EARLY_Phase 1]
Early phase 1 clinical trial update (LIGHT) investigates a gene therapy for Leber congenital amaurosis, conducted by Xinhua Hospital and Sha…
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CLINICAL_TRIALS trial_result · 2024-02-20Clinical Trial Update: Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D [Phase 1, Phase 2]
A phase 1/2 clinical trial evaluating subretinal ATSN-101 for Leber congenital amaurosis caused by biallelic GUCY2D mutations is currently a…
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CLINICAL_TRIALS trial_result · 2023-01-04Clinical Trial Update: Extension Study to Study PQ-110-001 (NCT03140969) [Phase 1, Phase 2]
An extension study update for PQ-110-001 in Leber congenital amaurosis; the study (NCT03140969) has been terminated with results posted on 2…
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CLINICAL_TRIALS trial_result · 2022-12-27Clinical Trial Update: Study to Evaluate QR-110 in Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene [Phase 1, Phase 2]
The document reports the completion of a Phase 1/2 clinical trial evaluating QR-110 for Leber congenital amaurosis due to CEP290 mutation, w…
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CLINICAL_TRIALS trial_result · 2021-04-08Clinical Trial Update: Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA) [Phase 1, Phase 2]
Clinical trial update for a gene therapy aimed at treating Leber Congenital Amaurosis (LCA) across Phase 1/2, with completion and results po…
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CLINICAL_TRIALS trial_result · 2018-04-10Clinical Trial Update: Clinical Trial of Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations [Phase 1]
A Phase 1 clinical trial update for a gene therapy targeting Leber Congenital Amaurosis caused by RPE65 mutations; the study status is compl…
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CLINICAL_TRIALS trial_result · 2018-03-26Clinical Trial Update: Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis [Phase 3]
A Phase 3 study evaluating safety and efficacy in subjects with Leber congenital amaurosis due to RPE65 mutations has posted results.
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CLINICAL_TRIALS trial_result · 2016-11-21Clinical Trial Update: Genetic Decryption of Leber Congenital Amaurosis (LCA) in a Large Cohort of Independent Families.
The completed clinical trial reports genetic analysis identifying variants associated with Leber Congenital Amaurosis in a large cohort of i…
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CLINICAL_TRIALS trial_result · 2016-04-29Clinical Trial Update: Natural History Study in Inherited Retinal Disease Subjects Caused by Mutations in RPE65 or LRAT
A natural history study in inherited retinal diseases caused by mutations in RPE65 or LRAT has completed.
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CLINICAL_TRIALS trial_result · 2016-04-08Clinical Trial Update: Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis [Phase 1, Phase 2]
Phase 1/2 study of an AAV-RPE65 gene therapy for Leber congenital amaurosis completed, with results posted in 2016.
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CLINICAL_TRIALS trial_result · 2015-12-07Clinical Trial Update: Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis [Phase 1, Phase 2]
Phase 1/2 safety study of RPE65 gene therapy for Leber congenital amaurosis has completed, according to the trial update.
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CLINICAL_TRIALS research · 2015-10-07Clinical Trial Update: Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65 [Phase 1, Phase 2]
This clinical trial update describes a Phase 1/Phase 2 gene therapy protocol for retinal dystrophy due to RPE65 defects in Leber congenital …
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CLINICAL_TRIALS trial_result · 2014-07-28Clinical Trial Update: Repeated Treatments of QLT091001 in Subjects With Leber Congenital Amaurosis or Retinitis Pigmentosa (Extension of Study RET IRD 01) [Phase 1]
Phase 1 extension study update on repeated treatments with QLT091001 in people with Leber congenital amaurosis or retinitis pigmentosa; the …
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CLINICAL_TRIALS trial_result · 2013-05-14Clinical Trial Update: Safety/Proof of Concept Study of Oral QLT091001 in Subjects With Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) Mutations [Phase 1]
A Phase 1 safety and proof-of-concept trial evaluating oral QLT091001 in individuals with Leber congenital amaurosis or retinitis pigmentosa…
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CLINICAL_TRIALS research · 2011-11-24Clinical Trial Update: Genetic Study of Patients Suffering From Congenital Amaurosis of Leber or From an Early Severe Retinal Dystrophy [NA]
A completed genetic study investigating patients with Leber congenital amaurosis and early severe retinal dystrophy.