Ocugen Advances Stargardt Disease Therapy with Positive Clinical Trial Progress

Ocugen

MALVERN, PAOcugen, Inc. (NASDAQ: OCGN) this week announced the progression of its OCU410ST GARDian clinical trial after the Data and Safety Monitoring Board (DSMB) approved moving forward with high-dose administration. The OCU410ST (AAV5-hRORA) gene therapy candidate targets Stargardt disease, affecting approximately 100,000 people in the U.S. and Europe.

To date, six patients have received doses in the Phase 1/2 clinical trial’s low and medium dose cohorts. The next phase involves dosing an additional three patients with the high dose in cohort 3.

“The DSMB has recommended moving forward to dose subsequent subjects with Stargardt disease at the targeted high dose,” said Dr. Peter Y. Chang, MD, FACS, DSMB Chair for the OCU410ST clinical trial. “No serious adverse events related to OCU410ST have been reported to date. This is an important next step in the clinical progress for OCU410ST and encouraging for patients living with this most common form of inherited retinal disease.”

Huma Qamar, M.D., MPH, Chief Medical Officer of Ocugen, expressed optimism about the trial’s progress. “We are delighted to report a second positive DSMB recommendation for the treatment of Stargardt disease and build upon the favorable safety and tolerability profile exhibited by OCU410ST. We recognize the high unmet medical need for Stargardt patients as there is no approved product. We are enthusiastic about OCU410ST as a potential one-time treatment for life with a single sub-retinal injection.”

The Phase 1/2 GARDian trial will include up to 42 subjects—30 adults and 12 children with Stargardt disease showing mild to moderate symptoms. The trial will evaluate the safety of unilateral subretinal administration of OCU410ST. Phase 1 is an open-label, dose-ranging study with three dose levels: low (3.75× 10¹⁰ vg/mL), medium (7.5× 10¹⁰ vg/mL), and high (2.25× 10¹¹ vg/mL). Phase 2 will be a randomized, outcome assessor-blinded study, enrolling subjects into different treatment groups or a control group to assess the therapy’s efficacy across dosages.

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Stargardt disease, a genetic disorder leading to vision loss, currently lacks FDA-approved treatments. Ocugen aims to address this gap through its gene-agnostic approach with OCU410ST. The company remains committed to advancing its clinical trials to provide a much-needed therapeutic option for individuals affected by inherited retinal diseases.

Revolutionizing Stargardt Treatment: Ocugen’s Gene Therapy Breakthrough

The implications of successful clinical trials for OCU410ST are significant. If proven effective, this gene therapy could transform the treatment landscape for Stargardt disease by offering a one-time intervention that mitigates the progressive vision loss associated with the condition. This would not only improve patient quality of life but also reduce the long-term healthcare burden associated with managing chronic visual impairment.

Ocugen’s progress in the GARDian trial reflects broader trends in gene therapy and personalized medicine. By targeting the genetic basis of diseases, such therapies offer the potential for durable, if not permanent, solutions to conditions that previously had limited or no treatment options.

As the trial progresses, stakeholders in the medical and investment communities will closely monitor the outcomes. Success in these trials could pave the way for broader applications of gene therapy in other retinal diseases and potentially other genetic disorders.

In conclusion, Ocugen’s advancement with the OCU410ST GARDian clinical trial represents a promising development in the fight against Stargardt disease. The company’s commitment to innovative treatment approaches has the potential to bring new hope to patients suffering from this debilitating condition, marking a significant milestone in the field of gene therapy.

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