Apr 9, 2021

Foundation Invests $5.5 Million in Seven New Translational Research Projects

Eye On the Cure Research News

Projects target a variety of conditions including: age-related macular degeneration, Stargardt disease, retinitis pigmentosa, and Usher syndrome type 3A

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Through its Translational Research Acceleration Program (TRAP), the Foundation Fighting Blindness is funding seven new projects aimed at advancing potential therapies toward clinical trials.

To launch an authorized clinical trial, a therapy developer must submit an Investigational New Drug (IND) application to the US Food & Drug Administration (FDA) – a process that requires expensive preclinical studies, regulatory knowledge, and manufacturing expertise. TRAP-funded grants support these efforts and guides projects toward an IND submission.

“Our TRAP grantees are exceptional scientists developing therapies that have strong potential to reach the patients with retinal diseases who need them,” says Chad Jackson, PhD, director of the Foundation’s TRAP program. “Translational research is challenging and expensive, but our program gives these scientists critical resources to help them succeed.”

Summaries of the new TRAP grants:

Dry AMD Gene Therapy

Bärbel Rohrer, PhD, Medical University of South Carolina, is conducting an animal study of a gene therapy designed to selectively deliver a component of complement factor H (CFH) to temper the overactive innate immune system in age-related macular degeneration (AMD). The approach is designed to mitigate retinal degeneration caused by the immune response, targeting the damage where it is most likely to occur.

Pharmaceutical for RP

Paul Yang, MD, PhD, is evaluating the drug mycophenolate as a therapy for multiple forms of RP and related conditions. Already approved by the FDA for inflammatory conditions, mycophenolate has been shown to reduce the accumulation of a molecule called cyclic guanosine monophosphate (cGMP). While cGMP is an important messenger molecule for converting light into electrical signals in the retina, too much of it is toxic and causes retinal degeneration. 

Small-Molecule for Usher Syndrome 3A (USH3A)

Mahdi Farhan, MD, Usher 3 Initiative, is completing pre-IND toxicity studies to advance a novel small-molecule therapy for USH3A into a Phase 1 clinical trial. The emerging drug works by stabilizing the misfolded USH3A protein (clarin-1) and enabling it to better move to its target location in retinal cells, thereby striving to preserve structure and function.

Enabling the Retina to Generate New Photoreceptors

Tom Reh, PhD, University of Washington, is developing a process to enable the human retina to grow its own new photoreceptors. Thus far, he has used a small molecule to sprout photoreceptors from Muller glia in mice. The TRAP project is for evaluating the approach in a large animal.

Cross-Cutting Gene Therapy for RP

Stephen Tsang, MD, PhD, Columbia University, is developing a gene therapy to increase aerobic glycolysis – a process that generates energy -- in cone photoreceptors of those affected by retinitis pigmentosa (RP). He believes the approach may preserve cones for RP patients and would do so independent of the mutated gene causing the disease.

RNA Therapies for Stargardt Disease

Rob Collin, PhD, Radboud University, is developing antisense oligonucleotides (AON) – tiny pieces of DNA – to mask splicing mutations in ABCA4, the affected gene in people with Stargardt disease. The AONs target mutations in RNA, the genetic messages used to build proteins that are necessary for a cell’s health and proper functioning.

Restoring Dormant Retinal Cell Function

Hendrik Scholl, MD, Institute of Molecular and Clinical Ophthalmology Basel, is developing an optogenetic therapy to restore function to dormant cone photoreceptor cells for potentially a broad range of inherited retinal diseases. Cones are responsible for high-acuity, daytime vision, and in a certain percentage of patients, remain in a dormant state. This effort will perform late-stage preclinical studies that are required to start the first-in-human cone-based optogenetic vision restoration clinical trial. This optogenetic therapy produces a protein that makes dormant cone cells sensitive to light.