Intravitreal Injection of Retinal Progenitor Cells Shows Promise in Preclinical Study for Diabetic Retinopathy

New study from University of California, Irvine and University of California, Santa Barbara researchers demonstrates improved retinal function and vascular integrity, supporting further investigation of cell-based therapy for diabetic eye disease

IRVINE, Calif.--(BUSINESS WIRE)--A new preclinical study titled “Intravitreal Transplantation of Retinal Progenitor Cells Improves Outcome Measures in a Rat Model of Diabetic Retinopathy” reports that retinal progenitor cells (RPCs) may provide both functional and anatomical benefits in diabetic retinopathy (DR), one of the leading causes of vision loss worldwide and the most common eye disease afflicting over 600 million people worldwide with diabetes.

The research addresses a critical unmet need: current therapies for DR focus primarily on vascular complications and often carry significant side effects, while failing to address the loss of retinal neurons. This study evaluated whether intravitreal transplantation of RPCs could improve outcomes in a rat model of DR.

In the study, diabetic Long Evans rats received intravitreal injections of either allogeneic rat RPCs (without immunosuppression) or human RPCs (with cyclosporin A) one week after induction of diabetes. At six weeks post-induction, animals underwent functional testing via electroretinogram (ERG), optomotor response (OR), and contrast sensitivity (CS). Retinal tissues were subsequently analyzed using immunohistochemistry at eight weeks.

Key findings include:

• Improved Retinal Function – Rat RPC-treated eyes demonstrated stronger ERG responses (b-wave and oscillatory potentials), along with enhanced optomotor response and contrast sensitivity compared to controls.
• Neuroprotection and Vascular Integrity – Immunohistochemistry revealed reduced vascular leakage (diminished albumin extravasation), decreased VEGF expression, and improved morphology across cellular and synaptic retinal layers.
• Cross-Species Effect – Human RPCs reproduced several of these functional and anatomical improvements, highlighting translational relevance.

Henry J. Klassen, MD, PhD, senior author and professor at the Gavin Herbert Eye Institute, stated, "These findings provide important proof-of-concept that retinal progenitor cell transplantation can deliver both neuroprotective and vasculoprotective benefits in an animal model of diabetic retinopathy. Our results support further investigation into RPCs as a potential cell-based therapeutic approach for clinical treatment of this sight-threatening condition.”

The full paper, “Intravitreal Transplantation of Retinal Progenitor Cells Improves Outcome Measures in a Rat Model of Diabetic Retinopathy,” is now published in The International Journal of Molecular Sciences.

The activities described were made possible by grant funding from the Discovery Eye Foundation and a gift from the Polly and Michael Smith Foundation. The authors acknowledge support via the Gavin Herbert Eye Institute at the University of California, Irvine, from an unrestricted grant from Research to Prevent Blindness, and from NIH grant P30 EY034070.

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 Discovery Eye Foundation
The Discovery Eye Foundation supports research, education and advocacy related to sight-threatening eye diseases and their treatments, improving the quality of life for patients and their families.

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.