Atsena Therapeutics’ gene therapy takes aim at childhood blindness

May 23, 2022 -- Ophthalmology-based gene therapy company Atsena Therapeutics is focused on developing novel treatments for inherited forms of blindness, with an ongoing Phase I/II clinical trial evaluating a potential therapy for patients with Leber congenital amaurosis (LCA) 1.

"Gene therapy, broadly, we're learning a lot. The field is still relatively young. Within the eye itself, we're working on the approach to solve the delivery issue and getting the vector to the right cell types," Atsena Therapeutics CEO Patrick Ritschel told at the American Society of Gene & Cell Therapy (ASGCT) 2022 annual meeting in Washington, DC.

Ritschel pointed to Atsena's laterally-spreading vector that can be administered "in one location in the eye and it will spread and cover the entire retina" to help solve the delivery issue.

LCA is the most common cause of blindness in children, impacting two to three per 100,000. LCA1 is caused by mutations in the guanylate cyclase 2D (GUCY2D) gene and results in early and severe vision impairment or blindness. LCA1-GUCY2D is one of the most common forms of LCA, affecting roughly 20% of patients who live with this inherited retinal disease.

Watch the video below to learn more.

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