Breakthrough Gene Therapy Trial for Retinitis Pigmentosa at UF College of Medicine – Jacksonville
The new trial seeks to improve treatment for individuals suffering from Retinis Pigmentosa.
The UF College of Medicine – Jacksonville is pioneering a groundbreaking gene therapy treatment trial for retinitis pigmentosa, an inherited retinal disease, or IRD, impacting vision. Spearheaded by the UF Department of Ophthalmology – Jacksonville, this initiative aims to possibly enhance treatments for individuals suffering from this condition, characterized by the degeneration of retinal cells.
According to Sandeep Grover, MD, a professor and associate chair of the department of ophthalmology, a defect in a specific gene leads to the formation of unhealthy proteins in the retina, causing progressive degeneration of retinal cells. This leads to blinding and untreatable IRDs.
The trial, titled “A Phase 1 Open-Label, Single Arm Dose Escalation Study to Evaluate the Safety and Tolerability of Intravitreally Administered VP-001 in Participants with Confirmed PRPF31 Mutation-Associated Retinal Dystrophy,” is sponsored by PYC Therapeutics LLC and specifically targets people with PRPF31 gene defect (also known as RP11). The University of Florida is one of just six centers in the country conducting this trial, with Grover as the principal investigator.
“We are injecting a study drug (healthy PRPF31 protein) in one eye to evaluate for its safety and tolerability over a one-year period,” Grover said.
This marks a historic moment for the organization. For the first time at the University of Florida and the city of Jacksonville, Grover administered the study drug as an injection into the eye on Feb. 13, 2024, a day that he says, “will forever be etched in his mind.”
“This is a significant moment not only for me, personally, but for our department of ophthalmology and University of Florida, Jacksonville,” Grover said. “A lot of team effort has gone into developing the IRD center here.”
Grover credits the members of his excellent research team, who played a significant role in making this possible. He also credits his patients, who are hopeful for a treatment in the future. They believe trials like this are a step in the right direction.

“This person with RP11 who was injected with the gene molecule is very enthusiastic and is not only wanting to do this for himself, but to help other people as well,” Grover said.
Grover has dedicated his career to the discovery, research and treatment of inherited (genetic) retinal diseases. The establishment of this specialized IRD Clinic at UF Health Jacksonville gives increased access to trials like this to patients. The UF Department of Ophthalmology – Jacksonville is currently conducting five active trials for inherited retinal diseases with a projected 10 trials by mid-2024.
He envisions this trial and others as a pivotal step toward expanding research and treatment opportunities for IRDs at the UF College of Medicine – Jacksonville and UF Health Jacksonville. He emphasizes that individuals affected by an IRD can benefit from advanced testing and genotyping at UF Health Jacksonville and the UF College of Medicine – Jacksonville, positioning them as recognized leaders capable of executing and facilitating clinical trials.