Ray Therapeutics (RayTx), a clinical-stage biopharmaceutical company pioneering optogenetic therapies for those living with retinal degenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to its lead candidate, RTx-015, for the treatment of retinitis pigmentosa (RP).

"Receiving RMAT designation is a critical step as we move along the continuum of progress toward bringing hope to patients with advanced vision loss," said Paul Bresge, CEO and Co-Founder of Ray Therapeutics. "Our mission has always been to restore functional sight. This designation will help us work more closely with the FDA to accelerate the delivery of this potentially transformative therapy to the patient community."

The RMAT designation is an important regulatory milestone, granted to innovative regenerative therapies intended to treat serious conditions in which preliminary clinical evidence indicates the potential to address unmet medical needs. This designation provides Ray Therapeutics with access to FDA guidance on efficient drug development and the potential for priority review.

Ray Therapeutics is pioneering an innovative approach to vision restoration that is designed to work regardless of the underlying cause of disease and can address rare inherited retinal disorders, such as RP, and larger indications, such as geographic atrophy. While other approaches typically focus on slowing disease progression, RayTx’s differentiated technology is bioengineered to restore visual function by reprogramming targeted retinal cells.

As RTx-015 progresses into late-stage clinical development, RayTx is prioritizing efficacy endpoints that both reflect the real-world challenges patients with RP face and demonstrate meaningful improvement in patients’ visual function and quality of life.

About Ray Therapeutics

Ray Therapeutics (Berkeley, California) is a clinical-stage biopharmaceutical company advancing optogenetic therapies to restore vision in patients with retinal degenerative diseases. By delivering a bioengineered, highly light-sensitive protein to targeted retinal cells, the approach is designed to improve visual function regardless of the underlying genetic mutation.

RTx-015 is Ray Therapeutics’ lead optogenetic gene therapy currently being evaluated in patients with retinitis pigmentosa. Delivered as a single intravitreal injection, the treatment aims to restore functional vision to those with advanced visual impairment.

Ray Therapeutics has a second clinical-stage program, RTx-021, which has been designed to restore vision to those with macula diseases, such as Stargardt disease and geographic atrophy age-related macular degeneration (GA AMD) by targeting retinal bipolar cells.

For more information, visit www.raytherapeutics.com.

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 treatments. 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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