World first regenerative cell therapy to replace damaged sensory cells in the inner ear, aiming to restore hearing, is expected to be tested in clinical trials in the UK in 2025.
Rinri Therapeutics said its lead product, Rincell-1, aimed to replace damaged auditory neurons with regenerated auditory neurons, re-establishing nerve connections in the cochlea and reversing neural hearing loss.
It said the therapy aimed to restore nerve connections between the inner ear and brain, offering “a groundbreaking treatment for severe age-related hearing loss and auditory neuropathy” and “revolutionise treatment for hearing loss”.
Rinri Therapeutics, a spinout company of the University of Sheffield, announced on 13 November 2024 that it had developed an innovative surgical method to deliver the therapy directly to the auditory nerve which connects the inner ear to the brain.
The new technique, published in Scientific Reports (a Nature journal), was developed as a safe and minimally invasive way to deliver treatments directly to the auditory nerve.
It will be used in Rinri’s upcoming first human trial for Rincell-1.
The company said 90% of people with hearing loss have sensorineural hearing loss caused by damage to sensory cells in the cochlea. Damage to one type of these sensory cells, the auditory neurons, cannot currently be treated, it said.
Its first target is regeneration of auditory neurons which transmit signals from the cochlea to the brain. The treatment targets the potential of stem cells.
“At Rinri we make auditory progenitors from stem cells; these are the early forms of the mature sensory cells in the inner ear,” Rinri Therapeutics said on its website.
“Our data shows that when our therapeutic progenitor cells are delivered to the inner ear, they have the capability to become mature auditory neurons and re-establish nerve connections in the cochlea and reverse neural hearing loss.
“Our therapy is potentially permanent and invisible.”
Renri said the cells were the first functionally restorative human auditory sensory cells, using progenitor cells that can mature into functional auditory neurons.
Reversed hearing loss
In preclinical studies, these cells reversed hearing loss and improved the hearing threshold by ~40%, which Rinri said were “strong preclinical results showing safety and effectiveness”.
Working with ENT surgeons internationally, the company said it modelled a safe and secure surgical access route to the central core of the cochlea, the area to which the cell therapies needed to be delivered.
Hearing problems such as age-related hearing loss and auditory neuropathy spectrum disorder happen when the nerve connections in the inner ear deteriorate.
“A cell therapy that can rebuild these connections could significantly improve hearing and quality of life for patients,” Rinri said.
Professor Marcelo Rivolta from the University of Sheffield led development of the technology which he said sought to “reverse sensorineural hearing loss through the repair of the damaged cytoarchitecture in the inner ear”.
Previously, accessing these nerves required highly invasive surgery, as they were located deep within the skull.
Funded by Rinri Therapeutics, researchers from the universities of Sheffield and Nottingham, King’s College London, Canada and Sweden, discovered the new way to access these nerves through the inner ear’s round window, a much less invasive and more accessible approach.
“This paper describes a novel surgical pathway to access the human cochlear nerve, opening up an important route to deliver new therapeutic agents, such as cells,” Prof Rivolta said.
“For us, it is a fantastic achievement and it consolidates our plans of commencing critical trials to treat neural hearing loss in the very near future.”
The treatment was developed using Rinri’s OSPREY platform, which creates ready-to-use regenerative cell therapies.
Dr Simon Chandler, CEO of Rinri Therapeutics, said: “The novel access route developed in elegant work by this team of leading hearing loss researchers and surgeons makes the delivery of transformative cell therapies like Rincell-1 possible.
“We’re delighted that the first-in-human trial for our lead product is on track to start in 2025, bringing the potential to transform the lives of people with neural hearing loss.”
Professor Doug Hartley from the University of Nottingham and chief medical officer at Rinri Therapeutics said: “Neural hearing loss has a significantly detrimental effect on the ability to understand speech, particularly with background noise, with a significant detrimental impact on quality of life.
“The novel surgical access devised by this research collaboration provides a therapeutic pathway to the cochlear nerve which is expected to be highly valuable in clinical practice as part of regenerative cell therapy for this global unmet medical need.’
