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

Commentary
Video

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.

REFERENCE
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. https://finance.yahoo.com/news/ocugen-announces-positive-clinical-study-121500823.html
Recent Videos
Daniela van Eickels, MD, PhD, MPH, the vice president and head of medical affairs for Bristol Myers Squibb’s Cell Therapy Organization
Paul Melmeyer, MPP, the executive vice president of public policy & advocacy at MDA
Daniela van Eickels, MD, PhD, MPH, the vice president and head of medical affairs for Bristol Myers Squibb’s Cell Therapy Organization
Arun Upadhyay, PhD, the chief scientific officer and head of research, development, and Medical at Ocugen
Arun Upadhyay, PhD, the chief scientific officer and head of research, development, and Medical at Ocugen
John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia
John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia
Barry J. Byrne, MD, PhD, the chief medical advisor of Muscular Dystrophy Association (MDA) and a physician-scientist at the University of Florida
John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia
Related Content
© 2024 MJH Life Sciences

All rights reserved.