CIRM Funds $4.7 Million AAV Gene Therapy for Rare Eye Disease

South San Francisco, CA – May 29, 2025 – The California Institute for Regenerative Medicine (CIRM), a leading institution in regenerative medicine, has awarded $4.7 million to the Blue Gen Therapeutics Foundation. This funding will support preclinical research for a novel AAV gene therapy targeting blue cone monochromacy (BCM), a rare inherited eye disease.

The award falls under CIRM’s late-stage preclinical projects program, which aims to advance stem cell-based or genetic therapy candidates toward an active Investigational New Drug (IND) application with the FDA. Dr. Aaron Nagiel, Principal Investigator at Blue Gen Therapeutics Foundation, will lead the project to develop an intravitreal AAV-based gene therapy designed as a potential one-time treatment. This AAV gene therapy seeks to deliver a functional copy of the L-opsin gene directly to cone photoreceptor cells in the retina, with the goal of restoring red and green photoreceptor function disrupted by genetic mutations in BCM patients.

Dr. Nagiel emphasized that this funding is a “huge milestone for BCM patients, their families, and the entire retinal dystrophy community,” pushing this AAV therapy closer to clinical trials.

BCM, typically diagnosed in infancy, severely impacts quality of life, causing low vision, light sensitivity, impaired color discrimination, and involuntary eye movements. Currently, treatments are limited to symptom management, highlighting the urgent need for more effective therapies.

Dr. Shyam Patel, CIRM’s Associate Vice President of Preclinical Development, stated, “This award reflects CIRM’s commitment to supporting cutting-edge science that has the potential to change lives.” He added that CIRM is proud to support Dr. Nagiel’s team in their efforts to advance a potential one-time AAV gene therapy treatment that aims to restore vision and improve the quality of life for individuals with BCM.