FOR IMMEDIATE RELEASE
Contact:
Esteban Cortez
Director, Marketing & Communications
press@cirm.ca.gov
South San Francisco, CA, May 29, 2025 – The California Institute for Regenerative Medicine (CIRM), one of the world’s largest institutions dedicated to regenerative medicine, awarded $4.7 million to fund preclinical research that aims to develop a gene therapy for the rare disease blue cone monochromacy (BCM).
The award will support a project in the Agency’s late-stage preclinical projects program, which provides funding for late-stage preclinical studies necessary to attain an active IND with the FDA for a stem cell-based or genetic therapy candidate.
The successful applicant this month is:
| Application # | Program Title | Principal Investigator/Institution | Amount |
| CLIN1-17090 | Development of a novel intravitreal AAV gene therapy for the rare disease blue cone monochromacy | Aaron Nagiel – Blue Gen Therapeutics Foundation | $4,691,936 |
Through the $4.7 million award, Aaron Nagiel, MD, PhD, and the team at Blue Gen Therapeutics Foundation will advance the development of a novel gene therapy for BCM, a rare, inherited eye disease that impairs color vision and severely affects visual function.
Dr. Nagiel said, “This funding decision by CIRM represents a huge milestone for BCM patients, their families, and the entire retinal dystrophy community as we move this therapy further towards clinical trials.”
The project will advance a potential one-time, intravitreal AAV-based gene therapy designed to deliver a functional copy of the L-opsin gene directly to cone photoreceptor cells in the retina. The goal is to restore the function of red and green photoreceptors, which are disrupted by genetic mutations in individuals with BCM.
Typically diagnosed in infancy, BCM significantly impacts quality of life, causing low vision, light sensitivity, impaired color discrimination, and involuntary eye movements. Current treatments are limited to symptom management, underscoring the urgent need for more effective therapeutic options.
“This award reflects CIRM’s commitment to supporting cutting-edge science that has the potential to change lives,” said Shyam Patel, PhD, CIRM’s Associate Vice President of Preclinical Development. “Blue cone monochromacy is a rare but deeply impactful disease with no meaningful treatment options. We’re proud to support Dr. Nagiel and the team at Blue Gen Therapeutics Foundation as they work to advance a potential one-time gene therapy treatment that aims to restore vision and improve quality of life for individuals living with this condition.”
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.
