Ocugen (NASDAQ: OCGN) has announced the publication of positive Phase 1 GARDian1 trial results for OCU410ST, its novel modifier gene therapy for Stargardt disease, in the peer-reviewed journal Nature Eye, published by the Royal College of Ophthalmologists.
According to Ocugen, the publication reports comprehensive 12-month safety, tolerability, and exploratory efficacy data from the first-in-human Phase 1 trial evaluating OCU410ST in patients with early to advanced Stargardt disease.
In a statement, Dr. Huma Qamar, CMO at Ocugen, commented, “This publication in Eye validates the scientific approach and clinical promise of OCU410ST as a modifier gene therapy for Stargardt disease. The Phase 1 GARDian1 trial demonstrated convergent functional and structural benefits. This represents a paradigm shift from any other approaches, including oral or mutation-constrained replacement approaches, to an agnostic modification strategy that can potentially benefit patients regardless of their underlying ABCA4 mutation with a potential single gene therapy for life. These results provide important support for our ongoing Phase 2/3 GARDian3 trial.”
