Novel gene therapy safe for retinitis pigmentosa
Key Takeaways
- A novel gene therapy for retinitis pigmentosa showed positive safety and efficacy results in a clinical trial.
- Unlike most gene therapies, the novel therapy is designed to work regardless of the underlying mutation that caused the retinitis pigmentosa.
Trial results are demonstrating the safety and effectiveness of a novel gene therapy for treating retinitis pigmentosa associated with NR2E3 and RHO mutations. OCU400, developed by Ocugen, is designed to deliver functional copies of the NR2E3 gene to retinal cells using a harmless virus. NR2E3 is a gene that regulates several physiologic functions within the retina, including light-sensing photoreceptor development and maintenance, metabolism, phototransduction (conversion of light to electrical signals), inflammation, and cell survival networks. OCU400 can potentially improve retinal health and function in patients with retinitis pigmentosa and is designed to work regardless of the underlying mutation that caused the retinitis pigmentosa.
Raj Mukherjee, an ophthalmologist based in Leeds, England, told Medscape Medical News that most current gene therapy strategies focus on individual genes associated with retinitis pigmentosa. While these targeted therapies can benefit specific patient populations, they do not address the broader spectrum of retinitis pigmentosa cases caused by the various mutations. He explained that the incidence of each genetic mutation is small, making it especially challenging to find a therapy targeted to each of the hundreds of subgroups. “Developing one treatment that will be independent of their genetic mutation and treat everybody is helpful,” he said. “A gene-agnostic therapy would be accessible for all retinitis pigmentosa patients without needing genetic testing.”
Researchers speaking at the American Society of Retina Specialists (ASRS) 2024 Annual Meeting reported that OCU400 was generally well tolerated in a study of 18 participants, with 89% of participants demonstrating stabilization or improvement in visual function measures. Ocugen is currently conducting a phase 3 clinical trial for OCU400, which will enroll 150 participants, including those with RHO gene mutations and patients with gene-agnostic (unknown genetic cause) retinitis pigmentosa. For more information on this trial, visit https://clinicaltrials.gov/study/NCT06388200 or contact Umair Qazi, MD, MPH, by calling 484-237-3390 or by emailing umair.qazi@ocugen.com.
Edited by Miriam Kaplan, PhD
Source:
Manuela Callari, Medscape Medical News, July 23, 2024; see source article