Research and Development

Gene Therapy Development Pipeline

Odylia is continuously evaluating new opportunities to add to our rare disease pipeline. If you have a therapeutic already in development that you believe would be a great match for Odylia, please reach out to us. We can evaluate the potential for integrating your therapeutic or gene target into the Odylia pipeline.

Learn about the Odylia gene therapy pipeline

RPGRIP1 Gene Therapy, LCA6

Looking for new partners

Leber congenital amaurosis (LCA) is a rare eye disorder that primarily affects the retina, which is the specialized tissue at the back of the eye that detects light and color. People with this disorder typically have severe visual impairment beginning in infancy. There are currently no treatments for LCA6. Odylia is developing an investigational gene therapy to treat vision loss caused by RPGRIP1 mutations. This gene therapy uses the novel Anc80 vector technology developed by Odylia co-founder, Luk Vandenberghe, and builds upon proof-of-concept data generated at Massachusetts Eye and Ear in the labs of Eric Pierce and Luk Vandenberghe.

USH1C Gene Therapy for the treatment of vision loss, Usher Syndrome Type 1

Partners: Usher 2020 Foundation, FAUN Foundation

Usher syndrome accounts for 50% of all inherited deaf-blindness disorders. It is characterized by profound deafness, progressive vision loss, and vestibular dysfunction with varying ages of initial symptom onset and severity. Usher Syndrome Type 1 is the most severe form of Usher Syndrome. Type 1 patients are born profoundly deaf, often have delayed motor skills, and have early-onset vision loss caused by retinitis pigmentosa. Inheritance of mutations in both copies of the USH1C gene cause Usher Syndrome Type 1. Oftentimes vision loss associated with USH1C mutations begins around 10 years of age with initial onset of night-blindness and a loss of side (peripheral) vision due to the dysfunction and eventual death of the light sensing cells of the retina, the photoreceptors. Vision loss progresses to tunnel vision, or loss of all but the most central portion of the visual field.  There are currently no treatments for the vision loss in Usher syndrome caused by USH1C mutations. Odylia has partnered with two patient advocacy groups, the Usher2020 Foundation and the FAUN Foundation, to develop a novel USH1C gene therapy. We are collaborating with scientific experts around the world, including Germany, the Cech Republic, and the United States, to streamline development of this gene therapy.

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