2023 Fall
As the vibrant colors of fall begin to paint the world around us, we are delighted to share the latest news and developments from the Center for Hereditary Retinal Degenerations (CHRD) at the Scheie Eye Institute in Philadelphia. We have been diligently working to advance our mission of bringing hope and healing to those affected by inherited retinal diseases, and we have some exciting updates to share with you.
New CHRD Website! Here you will find information about
- our groundbreaking research
- ongoing trials
- latest updates and resources
We’ve been busy! Some key highlights include:
Gene Therapy Trial for LCA5 Patients Our commitment to innovation continues with the commencement of a cutting-edge gene therapy trial for LCA5 patients with Lebercilin mutations. You can find more details about the trial at ClinicalTrials.gov. If you have any questions or wish to get involved, please reach out to us.
Promising Preliminary Results for LCA1 Patients We are elated to report that our gene therapy trial for LCA1 patients with GUCY2D mutations has yielded encouraging preliminary results. While our Phase 1/2 trial is currently ongoing and closed to new participants, a future Phase 3 trial is in planning stages in the quest for an eventual cure.
Upcoming Clinical Trials In the coming months, we are embarking on two additional clinical trials that hold great promise. The first is an international, multi-center Phase 3 clinical trial evaluating the safety and efficacy of oral N-acetylcysteine (NAC) in patients with RP. We invite you to learn more about this groundbreaking study led by Johns Hopkins University, aptly dubbed the "NAC Attack" trial. Our second exciting endeavor is a subretinal gene therapy clinical trial for patients with X-linked retinoschisis (XLRS) caused by RS1 mutations. You can find comprehensive information about this trial on ClinicalTrials.gov.