Ocugen Completes Dosing in Stargardt Disease Patients in Phase 1/2 Trial of OCU410ST

Ocugen, Inc., a biotechnology firm based in Malvern, Pennsylvania, has successfully completed dosing in the third cohort of its Phase 1/2 GARDian clinical trial for OCU410ST (AAV-hRORA). This gene therapy candidate is being developed to treat Stargardt disease, a genetic eye disorder that affects roughly 100,000 individuals in the U.S. and Europe. According to Dr. Huma Qamar, Ocugen's Chief Medical Officer, the completion of dosing in the third cohort marks the end of Phase 1 of this dose-escalation trial.

Three patients received a single subretinal injection of the highest dose, which is 2.25×10^11 vg/mL. The GARDian clinical trial, carried out at six prominent retinal surgery centers across the U.S., is assessing both the safety and effectiveness of OCU410ST when administered unilaterally to subjects with Stargardt disease.

Dr. Charles Wykoff, Director of Research at Retina Consultants of Texas and the study's lead investigator, emphasized the unique potential of OCU410ST as a one-time treatment administered in the operating room. He noted that the safety and tolerability of the therapy remain promising, offering hope to patients with Stargardt disease, a condition with no current FDA-approved treatment options.

The clinical trial is divided into two phases. Phase 1 is a multicenter, open-label, dose-ranging study with three dose levels: low dose (3.75×10^10 vg/mL), medium dose (7.5×10^10 vg/mL), and high dose (2.25×10^11 vg/mL). Ocugen is committed to continuously updating the public on the progress of its clinical trials, reflecting its dedication to advancing treatments for blindness through innovative gene therapy approaches.

Stargardt disease is a genetic disorder that leads to retinal degeneration and progressive vision loss, primarily affecting the macula, the central portion of the retina. The degeneration of photoreceptor cells in this area results in decreased central vision, although some peripheral vision is typically preserved. The disease often manifests during childhood or adolescence, though the age of onset and rate of progression can vary. The retinal pigment epithelium, a layer of cells supporting photoreceptors, is also affected in those with Stargardt disease.

OCU410ST employs an AAV (adeno-associated virus) delivery system to transport the RORA (RAR Related Orphan Receptor A) gene to the retina. This approach leverages Ocugen's modifier gene therapy platform, which focuses on Nuclear Hormone Receptor RORA. The therapy aims to address various pathways linked to Stargardt disease, including lipofuscin formation, oxidative stress, complement formation, inflammation, and cell survival networks.

Ocugen, Inc. specializes in discovering, developing, and commercializing novel gene and cell therapies as well as vaccines. The company strives to improve health outcomes and provide hope for patients worldwide through innovative scientific research. Ocugen's breakthrough modifier gene therapy platform holds the promise of treating multiple retinal diseases with a single product. Additionally, the company is advancing research in infectious and orthopedic diseases to meet unmet medical needs.