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- Leber Congenital Amaurosis

Leber Congenital Amaurosis Newsletter

Opus Genetics Reports 1-Month Pediatric Results for OPGx-LCA5 Gene Therapy in Inherited Retinal Disease 09 Apr 2025 14:39 GMT
… therapy candidate, OPGx-LCA5, for Leber congenital amaurosis (LCA5)-related inherited retinal disease …
MeiraGTx Announces The Lancet Publication of Data Demonstrating the Efficacy of rAAV8.hRKp.AIPL1 for the Treatment of Leber Congenital Amaurosis 4 (LCA4) Retinal Dystrophy 21 Feb 2025 11:30 GMT
… the most severe forms of Leber congenital amaurosis 4 (LCA4) owing to genetic …
Opus Genetics issues 1-month pediatric gene therapy results 08 Apr 2025 21:13 GMT
… therapy candidate, OPGx-LCA5, for Leber congenital amaurosis (LCA) 5-related inherited retinal …
MeiraGTx Receives Rare Pediatric Disease Designation from FDA for AAV8-RK-RetGC for the Treatment of Patients with Leber Congenital Amaurosis due to GUCY2D Mutations 22 Jan 2025 13:30 GMT
… the treatment of patients with Leber congenital amaurosis due to GUCY2D mutations (LCA1 … of cases causing autosomal recessive Leber congenital amaurosis type 1 (LCA1) whilst … the most severe forms of Leber congenital amaurosis (LCA) owing to genetic …
Opus Genetics Announces One-Month Clinical Data from Pediatric Patient in Phase 1/2 Trial of OPGx-LCA5 Gene Therapy in Inherited Retinal Diseases 08 Apr 2025 12:00 GMT
… to address a form of Leber congenital amaurosis (LCA) due to biallelic mutations … cause different forms of bestrophinopathy, Leber congenital amaurosis (LCA) and retinitis pigmentosa. Our …
FDA grants rare pediatric disease designation for Leber congenital amaurosis gene therapy 21 Aug 2024 14:09 GMT
… patients with a form of Leber congenital amaurosis, according to a press release … patients with a form of Leber congenital amaurosis, according to a press release …
Atsena Therapeutics Secures $150 Million Series C Funding to Advance Ocular Gene Therapy Pipeline 04 Apr 2025 21:04 GMT
… for Leber Congenital Amaurosis type 1 (LCA1).” Continued Development of ATSN-101 for Leber Congenital Amaurosis … gene therapy candidate targeting LCA1 (Leber Congenital Amaurosis type 1). The therapy has …
Atsena closes $150m in Series C round to progress gene therapy 03 Apr 2025 17:22 GMT
… -201. ATSN-101, which targets Leber Congenital Amaurosis type 1 (LCA1), has also …
Breakthrough research reveals gut-eye connection in vision loss 02 Apr 2025 18:35 GMT
… , including retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA). Recent research has unveiled …
Atsena Therapeutics Announces Oversubscribed $150 Million Series C Financing to Further Advance Ocular Gene Therapy Programs 02 Apr 2025 11:30 GMT
… a global pivotal trial for Leber Congenital Amaurosis type 1 (LCA1) and initiating … -class, investigational gene therapy for Leber congenital amaurosis type 1 (LCA1) has completed …

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