Regulatory Frameworks and Clinical Trial Advances in Gene Therapy for Retinal Degenerations: A Poster Presentation at DIA

Please introduce yourself and your project.
My name is Patricia Jumelle. I am a physician with a background in anesthesiology and with fellowship training in pain management. I graduated with a Master of Science in Regulatory Science from USC. My research poster is titled, “Regulatory Frameworks and Clinical Trial Advances in Gene Therapy for Retinal Degenerations,” with Dr. Tony Succar as my faculty mentor.

What is your research about?
Retinal degenerations affect a countless number of patients worldwide. In 2017, the FDA approved the first ocular gene therapy called voretigene. Although numerous ocular gene therapy trials have been conducted, there have been no other FDA-approved ocular gene therapies since then. This research highlights the regulatory considerations for ocular gene therapy as addressed in FDA’s 2020 Guidance for Human Gene Therapy for Retinal Disorders. In addition, a review of clinical trials from January 2020 to May 2025 registered on clinicaltrials.gov was conducted.

What did you enjoy most about working on this project?
I enjoyed the process of learning about ocular gene therapy by performing a comprehensive literature review and reading FDA guidances. Initially, this was a topic in which I was not well-versed. However, by the time I completed my research poster, I was able to comfortably discuss and answer questions about regulatory considerations of ocular gene therapy with ophthalmic regulatory affairs specialists at the Drug Information Association (DIA) conference. That was a wonderful achievement!

Patricia Jumelle, MD, MS
Physician | Anesthesiology & Pain Management

Watch Dr. Jumelle's poster overview