Atsena Therapeutics Announces 12-Month Safety and Efficacy Data from Phase I/II Clinical ...
Atsena Therapeutics announced 12-month safety and efficacy data from its Phase I/II trial of ATSN-101, a gene therapy for Leber congenital amaurosis caused by biallelic mutations in GUCY2D, published in The Lancet. ATSN-101 demonstrated durable, clinically significant improvements in vision at the high dose and was well-tolerated. This marks the first gene therapy treatment for LCA1, with potential for a Phase III trial.
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Gene therapy, ATSN-101, improved vision 100 to 10,000 times in 15 LCA1 patients, reversing Leber congenital amaurosis caused by GUCY2D gene mutations. Vision improvements were rapid and lasting, with high-dose recipients experiencing significant enhancements. Side effects were minor and related to the injection procedure. A larger trial is planned for FDA approval.
UF scientists developed a gene therapy restoring vision in LCA1 patients, with a 10,000-fold light sensitivity improvement and successful navigation in a maze. The treatment showed a clean safety profile, paving the way for phase 3 trials and potential commercialization.
UF researchers developed a gene therapy for LCA1, improving vision in a trial. High-dose recipients saw a 10,000-fold increase in light sensitivity. The therapy showed minor side effects and is set for phase 3 trials. Atsena Therapeutics aims to commercialize the treatment.
Atsena Therapeutics announced 12-month safety and efficacy data from its Phase I/II trial of ATSN-101, a gene therapy for Leber congenital amaurosis caused by biallelic mutations in GUCY2D, published in The Lancet. ATSN-101 demonstrated durable, clinically significant improvements in vision at the high dose and was well-tolerated. This marks the first gene therapy treatment for LCA1, with potential for a Phase III trial.
Gene therapy trial ATSN-101 showed rapid, substantial vision improvement in LCA1 patients, with 10,000-fold enhancement in 2 high-dose cases.
Gene therapy for Leber congenital amaurosis (LCA1) caused by mutations in the GUCY2D gene resulted in 100 times better vision, with some patients experiencing a 10,000-fold improvement. The Phase 1/2 trial involved 15 patients, including 3 pediatric cases, with rapid and lasting improvements noted. Safety concerns were addressed, with no serious side effects reported. The therapy, ATSN-101, shows potential for future randomized controlled trials.
New results show promise in treating Leber congenital amaurosis 1 (LCA1) with ATSN-101, an AAV5 gene therapy. Some patients experienced a 10,000-fold improvement in vision, with rapid improvements lasting at least 12 months. Published in *The Lancet*, the study highlights the potential of gene therapy for inherited retinal diseases.