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What will be the outcome of the phase 1/2 trial for ATSN-101 by December 31, 2024?
2
Resolution / Starting Odds
Successful with no major adverse effects • 25%
Successful with minor adverse effects • 25%
Unsuccessful due to lack of efficacy • 25%
Unsuccessful due to major adverse effects • 25%
Clinical trial reports, Atsena Therapeutics press releases
Story
AAV Gene Therapy Shows Promise for LCA1 Caused by GUCY2D Mutations
Sep 10, 2024, 07:31 AM
Recent advancements in AAV gene therapy have shown promising results for patients with Leber congenital amaurosis 1 (LCA1), a rare inherited eye disorder caused by mutations in the GUCY2D gene. AAV gene therapy has demonstrated dramatic improvements in vision for affected patients, with benefits sustained for over a year. The safety and efficacy of this therapy, specifically ATSN-101, were evaluated in a phase 1/2, multicenter, open-label, unilateral dose escalation study. Atsena Therapeutics is at the forefront of this research, highlighting the potential of gene therapy in treating inherited blindness.
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