On 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.

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.

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.

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.