New Study Demonstrates Breakthrough with Retinal Progenitor Cell Therapy in a Preclinical Model of Photoreceptor Degeneration

University of California, Irvine and University of California, Santa Barbara Researchers Report Groundbreaking Findings

IRVINE, Calif.--(BUSINESS WIRE)--Researchers from the Gavin Herbert Eye Institute and Sue & Bill Gross Stem Cell Research Center at the University of California, Irvine, in collaboration with the Neuroscience Research Institute at the University of California, Santa Barbara, have published a pioneering preclinical study demonstrating the effectiveness of intravitreal injection of retinal progenitor cells (RPCs) (jCell) in treating photoreceptor degeneration. This research has been published on July 24 in the International Journal of Molecular Sciences.

Photoreceptor dystrophies such as Retinitis Pigmentosa (RP) are a major cause of blindness, affecting millions worldwide and existing treatments are extremely limited. In RP, for example, there is no FDA approved treatment for 99% of patients. The study titled "Amelioration of photoreceptor degeneration by intravitreal transplantation of retinal progenitor cells in rats" presents a novel approach using RPCs to provide neuroprotection and functional rescue of degenerating photoreceptors.

Key findings include:

• Innovative Treatment Approach: The research utilized intravitreal injection of RPCs in a well-established animal model of photoreceptor degeneration. The RPCs release neurotrophic factors which are required to maintain the health and function of retinal cells. This is a novel use of these cells and delivering them to the vitreous cavity greatly simplifies the treatment procedure versus more invasive alternatives.
• Therapeutic Benefits: The RPC treatment demonstrated substantial preservation of both retinal structure and visual function.
• Clinical Progress: Supported by this preclinical work in rats, jCell has progressed through multiple clinical trials in patients with retinitis pigmentosa (RP). jCyte held a successful post-phase 2 Type B meeting with the FDA in January 2024 marking a significant milestone towards a pivotal trial in RP.

Henry J. Klassen, MD, PhD, senior author and professor at the Gavin Herbert Eye Institute, stated, "This study offers promising proof-of-concept for using intravitreal injection of RPCs to treat photoreceptor degeneration. Our findings underpin the clinical development of jCell which has the potential to change the course of the disease in retinal degenerations like RP."

The activities described were made possible by grant funding from the California Institute of Regenerative Medicine (CIRM).

The publication of this study marks an important step forward in the field of regenerative medicine and ophthalmology. Related patents have been awarded in the US and internationally, underscoring the innovative nature of this approach.

For more information, please contact: Dr. Henry Klassen Email: [e-mail]

About the Gavin Herbert Eye Institute

The Gavin Herbert Eye Institute is dedicated to advancing the understanding and treatment of eye diseases through innovative research and clinical care.

About the Neuroscience Research Institute

The Neuroscience Research Institute at the University of California, Santa Barbara, focuses on understanding the fundamental principles of brain and neural function through interdisciplinary research.

About the California Institute for Regenerative Medicine (CIRM)

At CIRM, we never forget that we were created by the people of California to accelerate stem cell treatments to patients with unmet medical needs, and act with a sense of urgency to succeed in that mission. To meet this challenge, our team of highly trained and experienced professionals actively partners with both academia and industry in a hands-on, entrepreneurial environment to fast track the development of today’s most promising stem cell technologies.

With $5.5 billion in funding and more than 150 active stem cell programs in our portfolio, CIRM is one of the world’s largest institutions dedicated to helping people by bringing the future of cellular medicine closer to reality.

About jCyte

jCyte, Inc. is a clinical-stage biotech company focused on developing its first-in-class allogeneic cell therapy, jCell, for retinitis pigmentosa (RP) and other retinal degenerative disorders. The treatment has received FDA Regenerative Medicine Advanced Therapy (RMAT) designation and is administered as a minimally invasive intravitreal injection. Currently there are no FDA approved therapies for over 99% of RP patients. The company is pioneering a new era of regenerative therapies to address the significant unmet medical needs of patients suffering from a broad set of retinal degenerative diseases. For more information, visit www.jcyte.com.