Regenerative Cell Therapy Trial for Hearing Loss OK'd

Rinri Therapeutics, a world leading spinout company from the University of Sheffield dedicated to transforming sensorineural hearing loss (SNHL) with regenerative cell therapy, has received approval to begin its first-in-human clinical trial.

The UK's Medicine and Healthcare products Regulatory Agency (MHRA) approved Rincell-1, a first-in-class otic neural progenitor cell therapy, for the first-in-human Phase I/IIa trial¹. Clinical proof-of-concept data is expected within 12 months of trial initiation.

While current standard-of-care treatments like cochlear implants help manage the symptoms of sensorineural hearing loss, their effectiveness depends on the integrity of the cochlear nerve. Rincell-1 has been designed to regenerate damaged auditory neurons, for which no treatment currently exists, bringing the potential to transform the lives of people with neural hearing loss and to play a significant role in healthy aging.

Taking place at three leading hearing research centres in the UK, the randomised open-label trial will involve 20 patients undergoing cochlear implantation for hearing loss, 10 with postsynaptic auditory neuropathy spectrum disorder (ANSD) and 10 with severe-to-profound age-related hearing loss (presbycusis). Within each group, patients will be randomly assigned to receive a single dose of Rincell-1 in addition to cochlear implantation or cochlear implantation alone .

The study is powered to measure safety and detect significant changes in neural health, as measured by telemetry from Advanced Bionics' cochlear implant monitoring AIM^TM system, alongside a range of speech perception measures and patient-reported outcomes. Rincell-1 will be delivered to the cochlea during cochlear implant surgery via a novel extension of the current procedure, as previously published in 2024.²

Professor Marcelo Rivolta, from the University of Sheffield's School of Biosciences and Founder and Chief Scientific Officer at Rinri Therapeutics, said: "This MHRA approval is a culmination of years of rigorous scientific exploration and an unwavering belief in the potential of regenerative medicine. Our research into Rincell-1 has consistently shown its ability to target and restore the delicate neural structures of the inner ear. Now, to translate this scientific understanding into a human trial, bringing us closer to a future where we can truly restore auditory function, is profoundly rewarding for the entire team who have poured their expertise and passion into this work."

Simon Chandler, CEO of Rinri Therapeutics, said: "Approval to start our first clinical trial with Rincell-1 in hearing loss is a major milestone and recognition of the potential of our regenerative cell therapy in this area of significant unmet medical need. I'd like to thank our staff and partners for their meticulous work towards this achievement, and our investors, Boehringer Ingelheim Venture Fund, UCB Ventures and Pioneer Group, for their support."

Professor Doug Hartley, CMO of Rinri Therapeutics & Chief Investigator on the trial, said: "Working daily with individuals suffering from hearing loss, I know how devastating it can be, and the acute need for a therapy to change the disease course. It's very exciting to be starting a clinical trial with Rincell-1, working with otologists across the world to develop this promising new approach to auditory nerve regeneration."

¹ First in human, multi-centre open-label randomised trial to assess the safety of Rincell-1 otic neural progenitor cell-based therapy in addition to standard care, compared with standard care alone, in participants with presbycusis or postsynaptic auditory neuropathy who meet UK guidelines for Cochlear Implantation. IRAS Ref: 1006992. Clin trials gov NCT07032038

² Novel procedure for cell delivery in Rinri Therapeutics' first clinical trial described in peer-reviewed publication