The University of Sheffield's spin-out company Rinri Therapeutics has received approval from the UK's Medicines and Healthcare products Regulatory Agency (MHRA) to begin the first-ever human trial exploring regenerative cell therapy as a treatment for sensorineural hearing loss. This pioneering study represents a significant advancement in addressing a condition that affects millions worldwide and for which no regenerative treatment currently exists.
Revolutionary Approach to Auditory Nerve Regeneration
Sensorineural hearing loss occurs when the sensitive, hair-like cells within the cochlea or the hearing nerve become damaged. While current standard treatments such as cochlear implants can help manage the condition, their effectiveness depends on the integrity of the cochlear nerve. Rinri Therapeutics has developed Rincell-1, a first-in-class optic neural progenitor cell therapy designed to regenerate damaged auditory neurons.
"This MHRA approval is a culmination of years of rigorous scientific exploration and an unwavering belief in the potential of regenerative medicine," said Professor Marcelo Rivolta from the University of Sheffield's School of Biosciences and Founder and Chief Scientific Officer at Rinri Therapeutics. "Our research into Rincell-1 has consistently shown its ability to target and restore the delicate neural structures of the inner ear."
Trial Design and Patient Population
The clinical trial will take place at three leading hearing research centers in the UK and will operate as a randomized, open-label trial. The study will involve 20 patients undergoing cochlear implantation for hearing loss, with ten patients having postsynaptic auditory neuropathy spectrum disorder (ANSD) and ten having 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.
Rincell-1 is an auditory neuron progenitor cell line that can differentiate into mature auditory neuron cells. The therapy will be delivered to the cochlea during cochlear implant surgery via a novel extension of the current procedure, which involves a minimally invasive technique to ensure the precise and safe delivery of the regenerative cells.
Measuring Success and Safety
The study aims to measure safety and detect significant changes in neural health, as measured by telemetry from Advanced Bionics' cochlear implant monitoring AIM™ system, alongside a range of speech perception measures and patient-reported outcomes. Clinical proof-of-concept data from the phase 1/2a trial is expected within 12 months of trial initiation.
Professor Doug Hartley, CMO of Rinri Therapeutics and Chief Investigator on the trial, emphasized the clinical significance: "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."
Industry Support and Future Implications
Simon Chandler, CEO of Rinri Therapeutics, noted that the approval represents "a major milestone and recognition of the potential of our regenerative cell therapy in this area of significant unmet medical need." The company has received support from investors including Boehringer Ingelberg Venture Fund, UCB Ventures, and Pioneer Group.
The therapy brings the potential to transform the lives of people with neural hearing loss and to play a significant role in healthy aging. Unlike current treatments that manage symptoms, Rincell-1 aims to address the underlying cause by replacing damaged sensory cells in the inner ear to restore hearing function, representing a paradigm shift in the treatment of sensorineural hearing loss.
