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Getting the latest healthcare news for you
Atsena Therapeutics' gene therapy candidates ATSN-201 and ATSN-101 are showing encouraging results for two rare inherited retinal diseases — X-linked retinoschisis and Leber congenital amaurosis, respectively. Both therapies demonstrated meaningful improvements in retinal structure and function with strong safety profiles. The European Medicines Agency also granted orphan drug designation to both candidates this summer.
Atsena Therapeutics is making strides in the fight against rare inherited retinal diseases. At the Association for Research in Vision and Ophthalmology (ARVO) meeting in Denver, researchers presented promising data on two gene therapy candidates — ATSN-201 for X-linked retinoschisis (XLRS) and ATSN-101 for Leber congenital amaurosis (LCA) — both of which recently received orphan drug designation from the European Medicines Agency.
ATSN-201, which uses a novel laterally spreading capsid (AAV.SPR) to target central retinal photoreceptors, showed schisis closure and measurable improvements in retinal function, with no dose-limiting toxicities reported. Meanwhile, ATSN-101 demonstrated durable gains in retinal sensitivity over three years, with no serious drug-related adverse events and five of six patients achieving the maximum score on mobility testing.
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Why it matters: Both conditions currently have no approved treatments, leaving patients with progressive vision loss and few options. These results bring Atsena closer to delivering the first therapies for these diseases, with pivotal trials already underway or imminent in the U.S. and Europe.
