Odylia Therapeutics, a nonprofit in Atlanta, has received a $249,719 grant from Critical Path Institute (C-Path). The money will help support a gene therapy for Usher Syndrome Type 1C (USH1C). Gene therapy is a treatment that delivers a healthy copy of a gene to cells to restore the function of that gene. People with USH1C are born deaf and often begin to lose their vision in their teen years. This vision loss happens because of retinitis pigmentosa (RP), an eye disease that slowly damages the retina, the part of the eye that senses light. Currently, there is no treatment to stop or slow this vision loss.
Odylia’s therapy uses a small, altered virus called an adeno-associated virus (AAV) to deliver a functional copy of the USH1C gene into retinal cells. The virus is used like a delivery tool. Early studies suggest the gene is reaching the right cells and doing its job. This grant will pay for safety tests and help the team create a pathway towards the therapy. These steps are needed before the treatment can be tested in people.
What this means for the Usher syndrome community: This grant is a positive step for people and families affected by USH1C. Research for rare diseases can be hard to fund, so support like this matters. Human testing has not started yet, but the project is moving forward. Each step brings researchers closer to a treatment that could one day help protect vision in people with USH1C.
Link to article
