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Gene therapy for Leber congenital amaurosis has positive phase 1/2 results at 12 months
26 Jun 2025 21:06 GMT
… -LCA5, a gene therapy for Leber congenital amaurosis.
She presented 12-month results … -LCA5 for the treatment of Leber congenital amaurosis, as Healio previously reported.
Sources …
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Global Leber Congenital Amaurosis Market 2025-2034: Growth, Share, Segments & Emerging Trends Analysis
13 May 2025 17:14 GMT
… the Growth of the Leber Congenital Amaurosis Market?
The leber congenital amaurosis market is expected to … ;leber-congenital-amaurosis-global-market-report
How Is the Leber Congenital Amaurosis Market Segmented?
The leber congenital amaurosis …
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FDA grants RMAT designation to gene therapy for Leber congenital amaurosis
08 May 2025 01:12 GMT
… -LCA5 for the treatment of Leber congenital amaurosis due to genetic variations in …
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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 …
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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 …
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[Interview] Doctors said he’d go blind. So he launched a biotech startup to cure it.
04 Jul 2025 11:21 GMT
… woman in her twenties with Leber congenital amaurosis. She had gone blind as … on our first case of Leber congenital amaurosis three years ago.”
Still, money …
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Opus Genetics Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)
03 Jul 2025 20:30 GMT
… targeting inherited retinal diseases including Leber congenital amaurosis (LCA), bestrophinopathy, and retinitis pigmentosa …
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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 …
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Novel Gene Therapy Improves Vision in Children with Leber Congenital Amaurosis
07 May 2024 13:06 GMT
… 17. These participants suffer from Leber Congenital Amaurosis (LCA), a blindness caused by … Mark Pennesi, MD, PhD.
About Leber Congenital Amaurosis
Leber Congenital Amaurosis (LCA) is a severe genetic …
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Opus Genetics completes dosing in first cohort of Leber congenital amaurosis trial
28 Mar 2024 18:13 GMT
… therapy, under investigation for Leber congenital amaurosis, demonstrated signs of biological … for the treatment of Leber congenital amaurosis, according to a press … administration in patients with Leber congenital amaurosis resulting from biallelic mutations …