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.

Andelyn and Odylia’s partnership grew out of a long-standing relationship. Following a review of the gene therapy manufacturing marketplace and impressed by Andelyn’s work within the rare disease space, Odylia recognized that Andelyn had the capabilities to manufacture this novel AAV capsid Anc80 and bring the treatment to clinical trials.

“At Andelyn, we are committed to making an impact on the lives of patients affected by rare diseases,” said Wade Macedone, chief operations officer at Andelyn. “We have the expertise and are determined to support Odylia in bringing this new therapy to clinical trials to give hope to patients with RPGRIP1-associated vision loss.”

Ashley Winslow, president and chief scientific officer at Odylia Therapeutics, said, “There is currently no treatment for vision loss caused by RPGRIP1 mutations. Finding the right partner to manufacture our gene therapy was critical to the success of the program. Andelyn is well respected in the rare disease space and recognizes where we are, where we came from, and where we are headed.”

Andelyn recently expanded and now operates in three facilities for plasmids, preclinical, and GMP clinical and commercial manufacturing. The opening of its new Andelyn Corporate Center completes a network of flexible and scalable sites that support end-to-end gene therapy contract development and manufacturing.