Ray Therapeutics receives EMA PRIME status for gene therapy

The designation recognises the potential of RTx-015 to address significant unmet medical needs in patients with severe retinal degeneration.

It advances the therapy’s regulatory support following a regenerative medicine advanced therapy (RMAT) designation from the US Food and Drug Administration (FDA) earlier this month.

Ray Therapeutics’ lead programme RTx-015 is an optogenetic gene therapy targeting RP, an inherited retinal disease that leads to progressive vision loss and blindness.

Administered as a single intravitreal injection, the genotype-independent therapy aims to restore visual function regardless of genetic mutation.

Ray Therapeutics co-founder and CEO Paul Bresge said: “Receiving PRIME designation for RTx-015 in RP from the EMA, alongside our recent RMAT designation from the FDA, is a powerful validation of our science and clinical outcomes, and the urgent need we are addressing.

“These designations accelerate our ability to advance RTx-015 globally and deliver on our mission to restore vision for patients living with blinding diseases.”

The PRIME status for RTx-015 was granted on the basis of preliminary safety and efficacy data from an ongoing Phase I/II clinical trial in patients with advanced RP.

The study reported improvements in visual function in the treated eye across all dose levels, supporting the therapy’s potential to restore vision.

Ray Therapeutics’ optogenetic approach uses a bioengineered, light-sensitive protein to target retinal cells, aiming to restore vision in retinal degenerative diseases.

The company’s second programme, RTx-021, is supported by the California Institute for Regenerative Medicine. The therapy targets bipolar cells to treat macular diseases such as Stargardt disease and geographic atrophy age-related macular degeneration.

The post Ray Therapeutics receives EMA PRIME status for gene therapy appeared first on Pharmaceutical Business review.