Research and Development

Gene Therapy Development Pipeline

Odylia Gene Therapy 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.

Below is more information about our active pipeline programs, RPGRIP1 and USH1C.

Learn about the Odylia gene therapy pipeline

RPGRIP1 Gene Therapy, LCA6

Partner: PTC Therapeutics

Leber congenital amaurosis (LCA) is one of the most severe retinal diseases that leads to vision loss, accounting for 20% of blindness in school-age children. LCA symptoms can develop as early as birth beginning with profound vision loss and involuntary eye movements, also known as nystagmus. Mutations in both copies of the RPGRIP1 gene cause a spectrum of vision-related deficits most commonly diagnosed as LCA type 6 (LCA6). Vision loss caused by RPGRIP1 mutations is particularly devastating because of the rapid onset and loss of vision. LCA6 patients are often completely blind from early childhood. There are currently no treatments for LCA6. In 2019 PTC Therapeutics licensed from Odylia an investigational gene therapy treatment for RPGRIP1 patients. 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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