Preclinical development of a first in class stem cell therapy for hearing loss

Over 400m people have disabling hearing loss in the world, with this figure expected to nearly double by 2050\. Deafness has a devastating effect on people, significantly affecting quality of life, education and employment prospects. The direct and indirect costs of hearing loss are estimated at £30B a year for the UK alone due to direct costs of life long treatment, personal costs to mental health, and lost economic productivity. There are no drugs for patients with hearing loss, the only treatments options available are medical devices such as hearing aids and cochlear implants. These devices only work for some patients and don't fully restore hearing for them. For patients with damage to the nerve cells of the ear (40m people globally), there are no treatments. Hearing devices do not work and these patients have no options. Patients and doctors have been demanding drugs that cure the underlying causes of all hearing loss conditions but particularly hearing caused by nerve damage, and this is the issue that Rinri Therapeutics was created to address.

Rinri's approach is to repair the damage in the ear that causes hearing loss by replacing the damaged cells with ones that work. To do this, we are developing a unique cell-based therapy for patients with hearing loss caused by nerve damage. These cells will be injected near to the site of damage in the ear where they will grow. These new cells are then able to restore the broken connections in the inner ear. Once hearing is restored, the patient will be able to communicate properly and fully participate in society, engaging socially and economically without restriction.

Currently, Rinri has shown that the therapy works and is safe in models of the disease and has shown that it can be made to high quality for patients. In this project, Rinri wants to complete its manufacturing development so it can produce clinical-grade cells, complete the background datasets, and develop the approach to deliver the product to patients. These project objectives will accelerate the pre-clinical development of Rincell-1 to demonstrate the safety, efficacy and manufacturability profile required by regulators for first in human clinical trials to demonstrate proof that the therapy works in humans and attract the funding required to take us through the remaining clinical program to the market.