Ocugen to launch phase 3 clinical trial of modifier gene therapy for retinitis pigmentosa

Key Takeaways

• The US Food & Drug Administration (FDA) has provided clearance for the launch of a phase 3 clinical trial for OCU400, a modifier gene therapy under development for people with retinitis pigmentosa (RP). 
• The therapy, which delivers copies of the NR2E3 gene to improve regulation of multiple functions in the retina, is designed to work regardless of the underlying mutation that caused the RP. 

The biotech company Ocugen has announced that the US FDA has provided clearance for launch of its Phase 3 liMeliGhT clinical trial for OCU400, the company’s modifier gene therapy under development for people with RP. The trial will enroll approximately 150 participants at 15 sites in the US. One arm of the trial will enroll 75 participants with mutations in the RHO gene. The other arm will enroll 75 participants with mutations in other genes causing RP. In each arm, participants will be randomized 2:1 to treatment and untreated control groups, respectively. Ocugen will use its newly developed mobility course known as the Luminance Dependent Navigation Assessment system to measure changes in participants’ vision under different levels of light.

OCU400 is a one-time therapy injected underneath the retina to deliver copies of the NR2E3 gene to improve regulation of multiple functions in the retina, including light-sensing photoreceptor maintenance and development, metabolism, phototransduction (conversion of light to electrical signals), inflammation, and cell survival. As such, it is gene agnostic, meaning that it is designed to work regardless of the underlying mutation that caused RP. OCU400 uses a human-engineered, adeno-associated virus (AAV), which works like a container system, to deliver NR2E3 copies into the recipients’ photoreceptors. 

For more information about this trial, visit https://clinicaltrials.gov/study/NCT06388200 or contact Umar Qazi, MD, MPH at  umair.qazi@ocugen.com or 484-237-3390. 

Edited by Miriam Kaplan, PhD

Source: Foundation Fighting Blindness, Eye on the Cure Research News, April 8, 2024; see source article