Ray Therapeutics wins $8M state grant for vision-restoring gene therapy

What's the deal? Ray Therapeutics has been awarded an $8 million grant from the California Institute for Regenerative Medicine (CIRM)Dealroom has a profile for this one. Try Dealroom → to advance RTx-021, its gene therapy for Stargardt disease. The clinical-stage biopharmaceutical company will use the money to fund its ongoing Phase 1/2 trial and manufacturing work.

What's the endgame? RTx-021 uses optogenetic gene therapy to make surviving retinal cells respond to light, aiming to restore vision regardless of the underlying genetic mutation. Delivered through a single intravitreal injection, the approach targets patients who have lost the retina's light-sensing photoreceptors.

Why now? Stargardt disease is the most common form of juvenile macular degeneration, affecting about 40,000 people in the US, and has no approved treatments. Patients with advanced disease lose the central vision needed to read and recognise faces.

CIRM's independent Grants Working Group placed the application in its highest funding tier, with all 13 scientific reviewers backing it. "CIRM understands what this work could mean for patients," said Paul Bresge, chief executive officer and co-founder of Ray Therapeutics.

The company's pipeline also includes lead program RTx-015, which targets retinal ganglion cells for retinitis pigmentosa and other inherited retinal diseases.

The signal: At $8 million, the award sits in the top 6% of grant rounds for US health companies. It reflects continued state-backed appetite for gene and stem cell therapies targeting conditions with no approved treatment options — a mandate CIRM has held since California voters created it in 2004.

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