CIRM awards $8 million to advance vision loss gene therapy to clinical trials

FOR IMMEDIATE RELEASE

Contact:
Esteban Cortez
Director, Marketing & Communications
press@cirm.ca.gov

 

South San Francisco, CA, April 24, 2025 The California Institute for Regenerative Medicine (CIRM), one of the world’s largest institutions dedicated to regenerative medicine, has approved awarding Ray Therapeutics nearly $8 million to fund a phase 1 clinical trial testing a therapy for a form of vision loss known as retinitis pigmentosa (RP).

The award will support a project in the Agency’s clinical program, which provide funding for eligible stem cell and gene therapy-based projects through all stages of clinical development. This award brings the total number of CIRM-funded clinical trials to 116.

Details of Ray Therapeutics’ application and award are as follows:

Application # Program Title Principal Investigator/Institution Amount
CLIN2-17086 Optogenetic Gene Therapy for Treatment of Retinitis Pigmentosa Paul Bresge – Ray Therapeutics INC $7,975,224

 

Developing a gene therapy for retinitis pigmentosa (RP)

People with RP typically begin to lose their vision in childhood or during young adulthood, and symptoms usually begin with a difficulty of seeing at night. The disease—which impacts roughly 100,000 people in the U.S—then progresses over time, leaving those affected with severely restricted tunnel vision or blindness. With the exception of a very rare genetic form of the disease, there is no cure.

With prior funding from CIRM, Ray Therapeutics advanced development of a potential gene therapy for RP  which introduces a gene that produces a light-sensitive protein into retinal cells found in the back of the eye that normally sense light and send signals to the brain. Animal studies showed the approach was safe and improved the animals’ ability to detect light.

“Retinitis pigmentosa remains a devastating condition with no approved treatments for the vast majority of patients,” said Paul Bresge, CEO & Co-Founder, Ray Therapeutics. “We are deeply grateful to CIRM for their belief in our science and their continued support of our programs. We are honored to partner with CIRM as we advance therapies that have the potential to transform the lives of patients.”

CIRM previously awarded Ray Therapeutics two grants totaling $8 million to support the development of their gene therapy approach in both RP and geographic atrophy, another form of age-related macular degeneration. The newly approved award will support the first clinical trial to test Ray Therapeutics’ approach in patients.

“It’s incredibly rewarding to see a project that CIRM supported in its early stages progress to clinical trials, moving us closer to delivering effective treatments for patients who need them,” said Joe Gold, PhD, Senior Director of Clinical Development at CIRM. “We are eager to collaborate with the team at Ray Therapeutics on this clinical trial and are committed to improving the lives of patients in California and around the globe who are living with retinitis pigmentosa.”


 

About the California Institute for Regenerative Medicine (CIRM)

The California Institute for Regenerative Medicine (CIRM) is a funding agency established by Californians to accelerate regenerative medicine research to deliver treatments for patients with unmet medical needs.

Established in 2004 through the passage of Proposition 71, CIRM was initially funded with $3 billion from the state of California to support ongoing research, and in 2020, was funded again with another $5.5 billion through Proposition 14 to continue the Agency’s important work.

CIRM has provided billions in funding to support stem cell, genetic research, and development programs in its portfolio. Through the Agency’s research, infrastructure, and education programs, CIRM aims to transform the field of regenerative medicine, stimulate economic growth, and improve the lives of diverse communities throughout the state.

For more information, go to www.cirm.ca.gov.