Skip to content

Ocugen Inc completes dosing of a Phase 1/2 study for a modifier gene therapy to treat Stargardt disease.

Ocugen Inc., (NASDAQ:OCGN), an ophthalmic biopharmaceutical company based in Malvern, Pennsylvania, has announced the completion of dosing in their second cohort of its Phase 1/2 “GARDian” clinical trial for OCU410ST (AAV-hRORA) – a modifier gene therapy candidate being developed for Stargardt disease “as a one-time treatment for life”, subject to independent evaluation and regulatory approval.  The study, entitled: “Study to Assess the Safety and Efficacy of OCU410ST for Stargardt Disease (GARDian)”, listed on (NCT05956626) and Ocugen expects to provide a clinical trial update for OCU410ST in the third quarter of 2024.  This is a multicenter study, which will be conducted in two phases and will enroll up to a total of 42 subjects. Commenting on the announcement, Huma Qamar, MD, MPH, Chief Medical Officer of Ocugen, said “the completion of dosing for Cohort 2 participants signifies an important clinical milestone for our pioneering modifier gene therapy. We are encouraged by the ongoing positive safety and tolerability profile demonstrated by OCU410ST, enabling us to consider higher doses in patients as we progress with the dose-escalation study. We look forward to sharing preliminary safety and efficacy data from phase 1 of the clinical trial”.

Stargardt disease (STGD1) is an inherited disorder that can cause vision loss in children and young adults with an estimated 30,000 people affected in the U.S. and more than 150,000 worldwide. The disorder is caused by variants in the photoreceptor-specific ABCA4 gene, which encodes the adenosine triphosphate-binding cassette, subfamily A, member 4. The gene encodes a transporter protein within retinal photoreceptor cells facilitating the active transport of potentially toxic retinoid compounds removing toxic by-products from the visual cycle. A number of pathogenic variants within certain introns of the ABCA4 gene may result aberrant splicing of the gene – including pseudogenes – essentially non-functional segments of DNA.  Loss of the protein results in the accelerated dimerization of vitamin A, forming toxic by-products that irreversibly damage the retina, resulting in progressive vision loss.

The GARDian clinical trial will assess the safety and efficacy of unilateral subretinal administration of OCU410ST in subjects with Stargardt disease and will be conducted in two phases. OCU410ST utilizes an AAV delivery platform for the retinal delivery of the RORA gene (RAR related orphan receptor A), which the company represents a modifier gene therapy approach to regulate certain pathways that may impact lipofuscin formation, oxidative stress, compliment formation, inflammation, and cell survival. 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 is a randomized, outcome accessor-blinded, dose-expansion study in which adult and pediatric subjects will be randomized in a 1:1:1 ratio to either one of two OCU410ST dose groups or to an untreated group.

Ocugen Inc., Therapeutic areas,

Following the announcement, Benjamin Bakall, MD, PhD, Director of Clinical Research at Associated Retina Consultants, and Clinical Assistant Professor at University of Arizona, College of Medicine, Phoenix, commented that, “there remains a great unmet medical need for patients with Stargardt disease, which is the most common inherited retinal disease affecting the center of the vision and does not have any FDA-approved treatment options. I am excited that we completed dosing of the last patient in Cohort 2, who received medium dose of this novel therapeutic leveraging a gene-agnostic approach”.