Ocugen, Inc. (NASDAQ: OCGN) shares drop slightly Tuesday. The Malvern, Pa.-based company, a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that the first patient was dosed in the global Phase 3 registrational trial of OCU410 (AAV5-hRORA), its first-in-class modifier gene therapy candidate for GA secondary to dry age-related macular degeneration (dAMD).
The Company also highlighted the recent FDA RMAT designation for OCU410, which supports a potential accelerated development and review pathway for the program.
“Dosing the first patient in our global Phase 3 trial, just weeks after receiving RMAT designation, marks a defining moment for the OCU410 program – and for the millions of people living with geographic atrophy.
Outside the U.S., there are currently no approved treatments for GA, while in the U.S., available treatment options address only one of the four disease pathways and require ongoing, repeated eye injections,” said CEO Shankar Musunuri.
“This is our third modifier gene therapy program to advance into late-stage development, demonstrating the strength of our platform and our vision for potentially delivering a one-time treatment for life.”
The initiation of dosing follows the successful completion of a Type B End-of-Phase 2 (EOP2) meeting with FDA’s Center for Biologics Evaluation and Research (CBER) in July 2026, resulting in alignment on all critical Phase 3 design elements, including primary and secondary endpoints, dose, adaptive design, and a single pivotal trial pathway to support a BLA.
OCGN shares began Tuesday eased three cents, or 2.3%, to $1.30.
