Two Partners Come Together to Manufacture Gene Therapy for RPGRIP1-Related Vision Disorders

According to a story from World Pharma Today, Andelyn Biosciences, Inc., which is a gene therapy Contract Development and Manufacturing Organization (CDMO), has recently struck a partnership with Odylia Therapeutics, which is a nonprofit biotech dedicated to the acceleration of rare disease drug development. The groups have partnered in order to manufacture a gene therapy called OT-004, which is intended to treat disorders of vision loss resulting from mutations to the RPGRIP1 gene. This gene has been linked to the rare eye disease Leber congenital amaurosis type 6, as well as cone rod dystrophy type 13 and a juvenile form of retinitis pigmentosa

About Leber Congenital Amaurosis

Leber congenital amaurosis is a genetic disorder which causes vision loss and often affects children and newborns. There are many different variants of this disorder which are distinguished from one another by which mutation is the cause. Symptoms include involuntary eye movements, severe vision loss and blindness, light sensitivity, and slow pupil response. Early clinical trials conducted in 2008 were the first which signaled the potential of gene therapy as a treatment for Leber congenital amaurosis and other eye diseases which were linked to the RPE65 gene. A gene therapy for cases caused by mutations of this gene was FDA approved in 2017. Leber congenital amaurosis occurs at an estimated rate of one in 40,000 births. To learn more about Leber congenital amaurosis, click here.

Hope for Inherited Retinal Disorders

Andelyn has established itself as a player in the field of rare genetic diseases, and Odylia was able to recognize that Andelyn had the capability to produce the innovative AAV Anc80 capsid, which will allow OT-004 to be studied in clinical trials. 

Currently, there are no treatments for vision loss disorders related to 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.” – Ashley, President, CSO, Odylia Therapeutics


“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.” – Wade Macedone, COO, Andelyn Biosciences

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