BioNeex Interviews

Gene Therapy

Interview with Co-Founder & CEO of Odylia Therapeutics, Scott Dorfman

Wed Mar 30 2022 18:36:33 GMT+0000 (Coordinated Universal Time)

Odylia Therapeutics is a Boston based independent nonprofit biotech organization focused on development of gene therapies for rare diseases. Odylia’s goal is to bring proven therapeutics to patients regardless of the number of people with the disease  Odylia is developing a gene therapy to treat vision loss caused by RPGRIP1 mutations. This gene therapy uses the novel Anc80 vector technology, which has a superior delivery profile compared to commonly used AAVs. RPGRIP1 mutations cause a spectrum of vision-related deficits most commonly diagnosed as LCA type 6 (LCA6). LCA6 patients are often completely blind from early childhood and have a rapid decline in photoreceptor function, but it is believed that the treatment window extends into adulthood and there is a potential for the regain of function in intact cells.

Tags and Keywords

First-in-ClassOphthalmologyOdylia TherapeuticsRare diseasesGene TherapiesEye disorder therapiesGene transferenceLeber congenital amaurosis