Ocugen, Inc., a biotechnology company specializing in gene and cell therapies as well as vaccines, has reported the completion of dosing in the second cohort of its Phase 1/2 ArMaDa clinical trial for OCU410 (AAV-hRORA). OCU410 is a potential gene therapy candidate aimed at treating geographic atrophy (GA), an advanced form of dry age-related macular degeneration (dAMD) that affects approximately 1 million individuals in the United States.

Dr. Huma Qamar, Ocugen's Chief Medical Officer, expressed optimism about OCU410, noting its potential as a gene-agnostic treatment option for GA. OCU410 targets various pathways associated with the disease, such as lipid metabolism, inflammation, oxidative stress, and the complement system, with just a single sub-retinal injection.

In the second cohort of the trial, three subjects received a 200 mL single subretinal administration of the medium dose (5x1010 vg/mL) of OCU410. The trial involves the participation of up to 13 leading retinal surgery centers across the United States.

Dr. Syed M. Shah, the lead investigator for the OCU410 Phase 1/2 trial, highlighted the complexity of GA and the limitations of current therapies, which mainly focus on a single pathway of the disease. OCU410, as a novel modifier gene therapy, has the potential to revolutionize GA treatment by addressing multiple facets of the condition.

A Data and Safety Monitoring Board meeting is scheduled for the next month to evaluate the safety data of the medium dose cohort before proceeding with the high dose, which marks the final dose in the Phase 1 dose-escalation study.

The ArMaDa Phase 1/2 clinical trial aims to assess the safety of unilateral subretinal administration of OCU410 in GA subjects, divided into two phases. Phase 1 is a dose-ranging study with three dose levels, while Phase 2 is a dose-expansion study, incorporating a randomized, outcome accessor-blinded approach.

Ocugen will continue to provide updates on the clinical progress of OCU410. As for dAMD and GA, these conditions affect millions globally, with dAMD being the leading cause of vision impairment.

OCU410 employs an AAV delivery platform for the retinal delivery of the RORA gene, which has demonstrated efficacy in targeting various pathways linked to dAMD pathophysiology. Ocugen envisions OCU410 as a promising one-time gene therapy for GA treatment.

 

Source: globenewswire.com

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