Ray Therapeutics (RayTx), an industry-leading clinical-stage biopharmaceutical company pioneering differentiated vision restoration therapies for those living with severe retinal degenerations, today announced it has been awarded an $8 million grant from the California Institute for Regenerative Medicine (CIRM) to advance RTx-021 for the treatment of Stargardt disease. The funding will support the ongoing Phase 1/2 clinical trial and manufacturing activities to prepare the program for late-stage development.

“CIRM shares our commitment to bringing therapies to people around the world who need them, including those living with significant vision loss,” said Paul Bresge, Chief Executive Officer and Co-Founder of Ray Therapeutics. “Their partnership has been essential to advancing RTx-021 for Stargardt disease, supporting the science and manufacturing needed to move from preclinical research into clinical development. We are deeply grateful for their continued investment in this program. CIRM understands what this work could mean for patients. That shared sense of purpose is at the heart of our relationship.”

Stargardt disease causes progressive loss of central vision, affecting the ability to read, recognize faces and perform everyday tasks. There are currently no approved treatments. Patients with advanced disease experience extensive loss of the retina's light-sensing photoreceptors, creating a need for approaches that restore visual function without those cells. RTx-021 is designed to address this need by enabling surviving retinal ON-bipolar cells to respond to light, with the goal of restoring vision regardless of the underlying genetic mutation.

“CIRM staff have interacted with people living with Stargardt disease and other blinding eye diseases at outreach events, most recently the Bay Area VisionWalk in June. It is clear that Stargardt’s patients need life-changing treatments that can help them maintain their independence and navigate daily life with confidence,” said Paul Webb, PhD, Senior Science Officer for Clinical Development of CIRM. “CIRM’s mission is to help bring promising science to the patients who need it. We’re hopeful that this approach will advance a potential vision-restoring therapy for people in California and around the world who currently have no approved treatment options.”

CIRM’s independent Grants Working Group placed RayTx’s application in its highest funding tier, with all 13 scientific reviewers recognizing its exceptional merit and potential for impact.

About Stargardt Disease

Stargardt disease is the most common form of juvenile macular degeneration, and about 40,000 people in the United States are living with the condition. It is caused by mutations that lead to photoreceptor loss over time, resulting in progressive loss of central vision.

About Ray Therapeutics

RayTx is a clinical-stage biopharmaceutical company advancing optogenetic gene therapies to restore vision in people with retinal degenerative diseases. By delivering bioengineered, highly light-sensitive proteins to surviving retinal cells through a single intravitreal injection, RayTx’s approach is designed to improve visual function in people with advanced visual impairment, regardless of the underlying genetic mutation. The Company’s pipeline includes lead program RTx-015, which targets retinal ganglion cells and is being developed for retinitis pigmentosa and other inherited retinal diseases, and RTx-021, which targets retinal bipolar cells and is being developed for Stargardt disease and geographic atrophy. For more information, visit www.raytherapeutics.com and follow RayTx on LinkedIn.

About the California Institute for Regenerative Medicine (CIRM)

The California Institute for Regenerative Medicine (CIRM) is a state agency created by California voters to accelerate stem cell and gene therapies for people with unmet medical needs. Since 2004, Californians have entrusted CIRM with $8.5 billion to accelerate promising discoveries through clinical trials, train a regenerative medicine workforce, strengthen the state’s biotechnology economy, and expand access to transformative therapies. Today, CIRM is pioneering new models of therapy development and accelerating medical breakthroughs that change lives — in California and around the world. For more information, visit www.cirm.ca.gov.

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