Fall 2026
November 6th, 2026
Fall brings new beginnings. As a season that represents harvest and growth, we persist in our fight for transformative research in inherited retinal diseases for our patients and families, the most tenacious and inspiring individuals we have known.
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Support for our Research!
Pictured Dr. Tomas Aleman, Mrs. Marisa Langford, and Mrs. Nikki Kurland
Over the decades, we have received support from many of our patients, families, and supporters. Recently, Eyes on the Cure, under the leadership of Mrs. Langford and Mrs. Kurland raised $67,000 for research and we are very thankful! Eyes on the Cure is preparing for next year’s event which is scheduled for May 12th, 2027. Support is pivotal for us as we use these funds to drive advances in better understanding and better measuring vision loss, and using this knowledge towards designing better clinical trial outcomes.
If you are interested in donating, click here → https://giving.apps.upenn.edu/fund?program=MED&fund=605604
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Since our last newsletter, we have expanded our clinical trials and have had discussions involving future gene therapy trials. Currently, CHRD has nine active clinical trials with four of them enrolling new participants. Below are a few highlights:
LCA5 Gene Therapy – Restoration of Day Vision
We had previously shown that adult patients with LCA5 could rapidly gain vision despite advanced disease. With funding from the US FDA, we have extended this work to adolescent patients who demonstrated robust and consistent restoration of day vision. We are currently working hard to share the latest results with the scientific/medical community and preparing for the start of Phase 3 trial surgeries.
LCA1 Gene Therapy – Improved Navigation under Dim Light Conditions
Our earlier results established that gene therapy can substantially improve dim light vision in LCA1. A Phase 3 trial is to start soon. In the meantime, we have been designing outcomes that evaluate the impact in patients daily living by measuring navigation under dim light conditions.
XLRS Gene Therapy – At the Children’s Hospital of Philadelphia
We are part of the multicenter LIGHTHOUSE trial for gene therapy ongoing at CHOP with Dr. Drew H. Scoles as surgeon, and Dr. Erin O’Neil co-principal investigator.
BEST1 Gene Therapy – Translation from Laboratory to Clinic
Inventions made at the Penn Veterinary School in collaboration with CHRD investigators to treat Best disease were recently translated to the clinic with the start of a Phase 1/2 clinical trial. Preliminary evidence of vision gains and anatomical improvements have been reported.
International Conference
CHRD members attended this year’s ARVO conference at Denver, CO, making a record 18 presentations with the latest results in our clinical trials, natural histories of retinal disease, developments of novel specialized vision tests, and more.
CHRD Website
To stay updated, with our clinical trials, hundreds of publications, and press releases, make sure to visit our website.
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Please remember, if a disease of interest is not listed in the newsletter, it does not mean that we are not working on it! For updates or questions for our investigators, you are more than welcome to reach out to us via email or call (215) 662-9981. We are always happy to hear from you!
Warm Regards,
Tomas S. Aleman, MD and Artur V. Cideciyan, PhD
Co-Directors, Center for Hereditary Retinal Degenerations
University of Pennsylvania Scheie Eye Institute,
Philadelphia, PA
(215) 662-9981