Andelyn Biosciences, a gene therapy contract development and manufacturing organization (CDMO), has partnered with Odylia Therapeutics, a nonprofit 501(c)(3) biotech focused on accelerating rare disease drug development, to manufacture a novel gene therapy (OT-004) to treat vision loss caused by mutations in the RPGRIP1 gene.
Mutations in the RPGRIP1 gene cause rapid decline in function of the photoreceptor cells of the retina, resulting in a rare type of vision loss that can begin as early as infancy. Patients are usually diagnosed with one of three forms of retinal dystrophy: Leber Congenital Amaurosis 6, Cone Rod dystrophy 13, or juvenile Retinitis Pigmentosa. OT-004, is an investigational AAV gene therapy that utilizes the Anc80 AAV vector for efficient gene transfer of RPGRIP1 to treat vision loss in patients lacking a functional copy of the gene.
