UofL researchers receive $4.23 million NIH grant to advance precision therapy for inherited blindness

Retinitis pigmentosa (RP) affects approximately one in every 4,000 people in the United States and is among the most common inherited causes of blindness. The condition gradually damages the retina, often beginning with difficulty seeing at night and progressing to tunnel vision and, ultimately, blindness. While it can be caused by many different genetic changes, one of the most common is related to a mutation in a gene called rhodopsin, which is essential for vision in low light. 

There is currently no approved treatment or cure for this form of the disease, but UofL researchers Maureen McCall, professor in the Department of Ophthalmology and Visual Sciences, and Ron Gregg, professor in the Department of Biochemistry and Molecular Genetics, are working to change that. 

With support from a five-year, $4.23 million grant from the National Eye Institute, receiving $807,000 for the first year, McCall and Gregg will help advance a promising therapy designed to slow or stop vision loss before permanent damage occurs. 

Reaching this point has also been made possible through earlier support from the Jewish Heritage Foundation, a grant from the Knights Templar awarded to Archana Jalligampala, Ph.D., a senior postdoctoral researcher in the McCall lab and funding from Mr. R. W. Rounsavall, Jr., which supported the confocal microscope used in all of these studies. 

The treatment, called Wave1A, was developed through a partnership with Wave Life Sciences. It is injected into the eye and travels to search for and destroy the faulty genetic instructions that lead to retinal damage while leaving healthy cells untouched. 

“Our goal is to protect vision by addressing the root cause of the disease,” said McCall. “If we can stop the harmful process early enough, we may be able to preserve sight for people who otherwise face progressive vision loss.” 

Researchers are testing the therapy in pigs, which have eyes with a composition most similar to that of humans. Early studies have shown encouraging results. The team is now working to determine how long the treatment remains effective and whether periodic injections could provide lasting protection against vision loss. 

The newly funded project represents an important step toward human clinical trials and, ultimately, a future where a diagnosis of retinitis pigmentosa does not inevitably lead to blindness. By generating the safety and effectiveness data needed to move this treatment toward FDA approval, McCall and Gregg are turning years of scientific discovery into a potential treatment with real-world impact. While significant work remains, the research offers new hope to those affected by inherited vision loss and reflects the power of discovery that extends beyond the laboratory to improve lives.