IRVINE, Calif. – jCyte, a clinical-stage biotechnology company developing transformative cell therapies for retinal diseases, today announced the publication “Retinal Progenitor Cells (jCell) for Retinitis Pigmentosa” containing results from its Phase I/IIa clinical trial evaluating the safety and tolerability of intravitreal injection of retinal progenitor cells (RPCs) in adults with non-syndromic retinitis pigmentosa (RP).

The prospective, multicenter, open-label, single-arm study enrolled 28 adults with RP across two vision cohorts. Participants received a single intravitreal injection of allogeneic RPCs at escalating dose levels (0.5, 1.0, 2.0, or 3.0 million cells). The primary objective was safety, with exploratory efficacy endpoints including best-corrected visual acuity (BCVA) and patient-reported outcomes.

Key findings include:

• Intravitreal RPC injection was well tolerated at all dose levels. The majority of adverse events were mild to moderate and transient in nature. Importantly, there were no signs of graft rejection.
• While designed as a safety study, exploratory efficacy data suggested potential vision benefits. At Month 12, mean BCVA changes (treated vs. untreated eyes) were:
  • 0.5M dose: +1.4 letters
  • 1.0M dose: +1.0 letters
  • 2.0M dose: +4.8 letters
  • 3.0M dose: +9.0 letters
• Patients also reported improvements in light sensitivity, object recognition, color discrimination, and reading ability.

“These encouraging results demonstrate that a single intravitreal injection of RPCs was safe and well tolerated, with exploratory signals suggesting a potential dose-related treatment effect,” said Henry Klassen MD, PhD and Co-Founder of jCyte. “While these findings must be interpreted with caution, they provide important rationale for advancing into later-stage controlled studies.”

The RPC technology has received FDA Regenerative Medicine Advanced Therapy (RMAT) designation, underscoring its potential to address a serious unmet medical need. Ongoing and planned future trials will further evaluate the therapy’s efficacy and durability in a larger RP population.

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

About Retinitis Pigmentosa

Retinitis pigmentosa (RP) is a group of rare inherited retinal diseases that cause progressive vision loss due to degeneration of photoreceptor cells. There are currently no broadly available disease-modifying therapies for RP, and patients face inevitable decline in visual function.

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 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 and is currently recruiting for its JC02-88 trial. For more information, visit www.jcyte.com.

 

Contacts

jCyte, Inc.
Victor Chao
Vice President Commercial Operations and Strategy
Media@jcyte.com