Ray Therapeutics Receives EMA PRIME Designation for RTx-015 Gene Therapy in Retinitis Pigmentosa

Ray Therapeutics has received priority medicines (PRIME) designation from the European Medicines Agency (EMA) for its RTx-015 gene therapy to treat retinitis pigmentosa (RP). The designation recognises the potential of RTx-015 to address significant unmet medical needs in patients with severe retinal degeneration and 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.

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 and targets bipolar cells to treat macular diseases such as Stargardt disease and geographic atrophy age-related macular degeneration.

The company also announced the appointment of Michael Murtagh as Chief Regulatory Officer. With more than 20 years of experience in regulatory affairs, he has led multiple Investigational New Drug Applications (INDs), Clinical Trial Applications (CTAs) and marketing applications across therapeutic areas including oncology, cardiovascular disease, neurology and inborn errors of metabolism. Prior to joining Ray Therapeutics, he served as Senior Vice President of Regulatory Affairs at AAVantgarde Bio and held senior regulatory leadership roles at Vedere Bio II, Astellas Gene Therapies, and BioMarin Pharmaceutical.

The CEO stated that Murtagh's appointment reflects the company's continued focus on building a leadership team with expertise to support the development of optogenetic therapies for patients with vision loss. Murtagh said he is particularly excited to define the registrational pathway for RTx-015 in retinitis pigmentosa, with the goal of delivering therapies to patients with high unmet need.

RP is a genetic disease in which the photoreceptors gradually degenerate, resulting in complete or nearly complete blindness for most patients. Symptoms include night blindness, reduced visual fields, and eventual loss of visual acuity. It is estimated that more than half a million people are affected by RP worldwide, and no effective treatment is currently available.