MALVERN, Pa., April 1, 2024 (Globe Newswire) — Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN) is focused on the discovery, development and commercialization of novel gene and cell therapies and vaccines. A biotechnology company. Today, the Data Safety Monitoring Board (DSMB) for the OCU410ST clinical trial recently convened and announced that it has been approved to continue administering intermediate doses of OCU410ST during the dose escalation phase of the study.
To date, the drug has been administered to three patients with Stargardt disease in Phase 1/2 clinical trials. During the dose escalation phase, three additional patients will receive a medium dose (cohort 2) of his OCU410ST and three patients will receive a high dose (cohort 3) of her OCU410ST.
“The DSMB recommended transitioning to an intermediate dose for Stargardt disease recipients,” said Peter Chan, MD, FACS' DSMB Chair of the OCU410ST clinical trial. “To date, no serious adverse events (SAEs) have been reported related to OCU410ST. We believe this is an important next step in determining the optimal dosing regimen for OCU410ST. ”
“We are pleased that OCU410ST continues to demonstrate a favorable safety and tolerability profile, allowing us to evaluate higher doses in Stargardt retinal dystrophy patients,” said Huma, Chief Medical Officer, Ocugen. – said Kamal, MD, MPH. “We recognize the high unmet medical need for Stargardt patients, as there is currently no FDA-approved treatment for this indication, and we plan to launch an interim safety study in a Phase 1 study in the second quarter of 2024.” We look forward to sharing your sexual data.”
The GARDian clinical trial will evaluate the safety and efficacy of unilateral subretinal administration of OCU410ST in patients with Stargardt disease and will be conducted in two phases. Phase 1 is a multicenter, open-label, dose-ranging study consisting of three dose levels. [low dose (3.75×1010 vg/mL), medium dose (7.5×1010 vg/mL), and high dose (2.25×1011 vg/mL)]. Phase 2 was a randomized, outcome accessor-blinded, dose expansion study in which adult and pediatric subjects were randomly assigned in a 1:1:1 ratio to one of two OCU410ST dose groups or to untreated subjects. Masu. We continue to provide clinical updates.
Ocugen is committed to finding solutions for people with inherited retinal diseases for which no effective treatment options exist. Stargardt is an orphan disease, affecting approximately 40,000 people in the United States alone.
About Stargardt disease
Stargardt disease is an inherited eye disease that causes retinal degeneration and vision loss. Stargardt disease is the most common form of inherited macular degeneration. The progressive vision loss associated with Stargardt disease is caused by degeneration of photoreceptor cells in the central part of the retina called the macula.
Decreased central vision due to loss of photoreceptors in the macula is a hallmark of Stargardt disease. Some peripheral vision is usually preserved. Stargardt disease usually begins in childhood or adolescence, but the age of onset and rate of progression varies. In Stargardt disease patients, the retinal pigment epithelium (RPE), the cell layer that supports photoreceptors, is also affected.
About OCU410ST
OCU410ST utilizes an AAV delivery platform for retinal delivery. roller (RAR-related orphan receptor A) gene. This represents his Ocugen modified gene therapy approach based on nuclear hormone receptors (NHR). roller Regulates Stargardt disease-related pathways including lipofuscin formation, oxidative stress, complement formation, inflammation, and cell survival networks.
About Ocugen Corporation
Ocugen, Inc. is a biotechnology company focused on discovering, developing and commercializing new gene and cell therapies and vaccines that improve health and give hope to patients around the world. We impact patients' lives through courageous innovation and pioneer new scientific avenues, leveraging our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, supporting public health and orthopedic diseases to address unmet medical needs. In order to do so, we are conducting research on infectious diseases. Learn more at www.ocugen.com and follow us on Twitter and LinkedIn.
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