jCyte Begins Famzeretcel RP Trial

jCyte has announced the successful dosing of the first patients in the JC02-88 clinical study, a key step in evaluating famzeretcel (jCell), an investigational cell therapy for retinitis pigmentosa (RP). This milestone brings new momentum to the ongoing development of a treatment for a condition with limited therapeutic options.

Clinical Evaluation of Famzeretcel Progresses in Retinitis Pigmentosa Trial

“The initiation of the patient dosing in this trial marks an important milestone in our mission to bring a breakthrough treatment to the majority of RP patients who currently have limited treatment options,” said John Sholar, Chief Executive Officer of jCyte, in a press release.

The JC02-88 study is a Phase 2 trial designed to evaluate the safety, tolerability, and efficacy of a single intravitreal injection of 8.8 million cells of famzeretcel. Notably, the dose administered in this study is approximately 50% higher than the highest dose tested in previous jCyte trials, representing a significant escalation in the company’s clinical research efforts.

Participants in the trial will receive either a single dose of famzeretcel or a sham treatment (control), with assessments focused on safety and changes in vision over a 6-month period.

Collaborative Support and Future Outlook

The trial is being supported by a network of key collaborators, including the Gavin Herbert Eye Institute, the UCI GMP Facility, Lexitas, and the California Institute for Regenerative Medicine (CIRM). A follow-up extension study is planned to continue evaluating long-term outcomes after the conclusion of the JC02-88 study.

Addressing a Rare and Debilitating Disease

Retinitis pigmentosa is a rare genetic disorder affecting an estimated 2 million people globally, including approximately 100,000 individuals in the United States. It is characterized by the progressive loss of rod and cone photoreceptors in the retina, often diagnosed in adolescence. Many patients with RP experience a steady decline in vision and become legally blind by middle age.

“This is an exciting development for the RP community, and I am eager to see how this promising therapy advances toward providing a novel cell-therapy treatment for a patient population with vast unmet need,” said Paul Sieving, MD, PhD, Neil and MJ Kelly Professor of Ophthalmology at UC Davis School of Medicine and former Director of the National Eye Institute at the NIH.