Apr 8, 2024

Ocugen to Launch Phase 3 Clinical Trial of Modifier Gene Therapy for RP

Eye On the Cure Research News

The emerging treatment is designed to work independent of the patient’s mutated gene.

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The biotech company Ocugen has announced that the US Food & Drug Administration has provided clearance for launch of its Phase 3 liMeliGhT clinical trial for OCU400, the company’s gene-agnostic, modifier gene therapy under development for people with retinitis pigmentosa (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.

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: photoreceptor maintenance and development, metabolism, phototransduction, inflammation, and cell survival. OCU400 uses a human-engineered, adeno-associated virus (AAV), which works like a container system, to deliver NR2E3 copies into the recipients’ photoreceptors.

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.

Ocugen also has two Phase 1/2 clinical trials underway for its modifier gene therapies for people with geographic atrophy (GA) associated with advanced dry age-related macular degeneration and Stargardt disease. These emerging therapies — OCU410 for GA and OCU410ST for Stargardt disease — are designed to deliver copies of the RORA gene to retinal cells to improve lipid metabolism and reduce inflammation. Ocugen believes boosting RORA expression will slow retinal degeneration and vision loss in people with GA and Stargardt disease.

Inquiries for Ocugen’s trials can be sent to clinical.request@ocugen.com.