CIRM Awards $4.7 Million for Vision Loss Gene Therapy

The California Institute for Regenerative Medicine (CIRM) awarded $4.7 million to Blue Gen Therapeutics Foundation (Principal Investigator Aaron Nagiel, MD, PhD) 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, Dr. Nagiel 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. 

"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," Dr. Nagiel said.

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.

“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.”