Three weeks ago, Ray Therapeutics got FDA RMAT designation for RTx-015, its op togenetic gene therapy for retinitis pigmentosa. We covered that here.
Today they pulled in $125 million led by Janus Henderson Investors, with Adage Capital, Franklin Templeton, Invus, Marshall Wace, 4BIO, Deerfield, Merck’s MRL Ventures, Norwest, Novo Holdings, and Platanus piling in.
That’s one heck of a crossover book and it likely means Ray is setting up for a potential IPO window, or at minimum positioning for the kind of late-stage private round that precedes one.
Ray Therapeutics pipeline
RTx-015 — Retinitis Pigmentosa
Optogenetic AAV gene therapy · FDA RMAT designation
RTx-021 — Stargardt Disease
Gene therapy · IND-enabling
RTx-021 — Geographic Atrophy
Gene therapy · IND-enabling
For the unfamiliar: optogenetics puts a light-sensitive protein into the remaining retinal cells of a blind patient. The cells that used to be photoreceptors are gone, but the downstream circuitry is still intact. Put the light-sensing protein into those downstream cells and suddenly the retina can respond to light again. Different wavelength, different resolution than a normal eye, but real vision where there was none.
RTx-015 is in the Phase 1 ENVISION trial right now with 18 patients. RTx-021 targets two much bigger indications: Stargardt disease and geographic atrophy.
Here’s the interesting collision.
Earlier today, Belite Bio started a rolling NDA submission for tinlarebant in Stargardt – its an oral small molecule that modulates the visual cycle. That’s a slower mechanism with decent evidence behind it.
Ray’s going to arrive at the same indication with a one-shot gene therapy with a different bet entirely. Tinlarebant wants to slow progression…RTx-021 wants to undo damage.
Proceeds go toward late-stage development for RTx-015 plus clinical studies for both RTx-021 indications. Commercial readiness is on the list too, which tells you how Ray is thinking about timelines.
