The RPGRIP1 Project
to Prevent Vision Loss
What is LCA & RPGRIP1?
Leber Congenital Amaurosis (LCA), is a rare inherited eye disorder that affects the retina, and can cause severe vision loss starting from birth. It is the most common cause of childhood vision loss and accounts for 20% of blindness in school-age children. At least 27 different genes are associated with LCA. Mutations in both copies of the RPGRIP1 gene cause a spectrum of vision-related deficits most commonly diagnosed as LCA type 6 (LCA6). LCA6 patients are often completely blind from early childhood and have a rapid decline in function of the photoreceptor cells (rods and cones) of the retina. The loss of the RPGRIP1 protein causes vision loss in patients and onset is often rapid and progressive
There is currently no treatment for LCA, but Odylia Therapeutics is working on a gene therapy
that could treat vision loss caused by RPGRIP1 mutations.
The Path Forward
Gene therapy, or the replacement of a functional gene, can be an extremely effective treatment for genetic disorders like LCA6. But, development of a gene therapy is a long and expensive process and not all treatments are commercially viable. When science is no longer the barrier, profit should not be the reason a treatment is abandoned. That's where Odylia Therapeutics comes in - we're a nonprofit biotech working to bring lifesaving and life-altering treatments to those with rare diseases regardless of prevalence or commercial interest.
Odylia is developing a gene therapy to treat vision loss caused by RPGRIP1 mutations that builds upon proof-of-concept data generated at Massachusetts Eye and Ear in the labs of Eric Pierce and Luk Vandenberghe. Odylia is currently conducting late-stage preclinical experiments and is preparing for IND submission for use of the gene therapy in clinical trials. In 2021, the FDA granted Orphan Drug and Rare Pediatric Disease Designations to the RPGRIP1 gene therapy. Progress is being made!
What Can I Do?
To move this program forward, we need YOU. Odylia is committed to manufacturing the gene therapy and completing the final studies required to begin clinical trials. Donations are vital to making this happen. We hope to raise $950,000 by September 1 toward the overall goal of $4.75 million over the next two years. Money raised will be used directly for the RPGRIP1 Program. Make a donation, sign up to be a fundraiser, join a team, or host an event.
You Can Help Prevent Vision Loss
Gather friends and family to form a team or challenge yourself to raise funds. Either way, your efforts to spread awareness about RPGRIP1 and to inspire donations will impact patients. Once you register, you can create a personal fundraising page to send out to your network to gain support and gather donations.
Sign in to your personal page following this link.
Odylia Therapeutics is a nonproﬁt (501c3) biotech working to accelerate the development of treatments for people with rare diseases. We work to change the way therapies are brought from the lab to patients regardless of prevalence or commercial interest.
Odylia can change the lives of those with rare diseases by de-risking the most promising therapeutic approaches and streamlining drug development through strategic partnerships.