Shankar Musunuri, PhD, on Continuing Research With Gene Therapy in Inherited Retinal Diseases


The chairman, chief executive officer, and cofounder of Ocugen discussed programs including OCU400 and OCU410.

“We have data to suggest [OCU410] can actually go after all those different pathways or causes of the disease, and could be a potential, one-time, curative therapy. Current therapies you have to continuously take, every month or so, intravitreal injections, and the degeneration is continuing. So, if we can show stabilization or improvement over those products, that's gold. So, we believe this gene therapy has that potential.”

Patients with retinitis pigmentosa (RP) treated with OCU400 (Ocugen) in a phase 1/2 clinical trial (NCT05203939) experienced improvement or stabilization of vision. Preliminary data in patients with Leber congenital amaurosis (LCA) treated in the latest cohort are also promising. There were serious adverse events (AEs) in 1 patient who received the high dose and 1 patient with LCA included in the open enrollment cohort, but the therapy has been generally well-tolerated. Most AEs were due to the surgical procedure and resolved in days to weeks. OCU400 (Ocugen) is an investigational modifier gene therapy intended to treat inherited retinal diseases,

CGTLive spoke with Shankar Musunuri, PhD, chairman, chief executive officer, and cofounder, Ocugen, to learn more about the OCU400 as well as other research the company is pursuing for inherited retinal diseases. He discussed the OCU410 program, which is treating patients with dry age-related macular degeneration (AMD) in a phase 1/2 trial (NCT06018558), and the unmet needs that remain in the population with dry AMD that gene therapy may address. He also shared plans to evaluate OCU410 in patients with Stargardt disease.

Ocugen announces positive clinical study update from the phase 1/2 trial of OCU400, a modifier gene therapy product candidate, for the treatment of retinitis pigmentosa (R) and Leber congenital amaurosis (LCA). News release. Ocugen. September 13, 2023.
Related Videos
George Tachas, PhD, on Tackling DMD Treatment From Multiple Angles
David Suhy, PhD, the cofounder and chief scientific officer of Earli
Deepak L. Bhatt, MD, MPH, MBA, on Incorporating AI into Genetic Research for Cardiovascular Disease
Jeffrey Chamberlain, PhD, on Helping Progress Cell and Gene Therapy Development
Jonathan W. Weinsaft, MD, on Integrating Genetic Research into Cardiovascular Medicine
Jacques Galipeau, MD, on Highlights from ISCT 2024’s Presidential Plenary
Vanee Pho, PhD, the senior director of product management, cell and gene therapy, at Mission Bio
Michael Wang, MD, a professor in the Department of Lymphoma/Myeloma at MD Anderson Cancer Center
Robert J. Hopkin, MD, on Looking Deeper into Fabry Disease Biology
Steven W. Pipe, MD, on Confirming Efficacy, Safety of Hemgenix Gene Therapy in Hemophilia B Populations
© 2024 MJH Life Sciences

All rights reserved.