With the rapid evolution of new reagents and technologies on the horizon, many researchers will optimise their existing processes as they progress towards the clinic. Ultimately, developers want reagents that are fully chemically defined, controllable and traceable, but those reagents do not necessarily exist now, however, suppliers are working hard to address these requirements.įinding the right GMP manufacturing partner with the right facilities and experience is critical and building this essential relationship can take time For cell therapy companies approaching first‑in‑human trials, good manufacturing practice (GMP)‑compliant options with complete documentation often do not exist, so researchers must risk assess the most appropriate reagents available, which might mean those containing animal-derived components, such as a bovine serum for example. Often prohibited by funding, academic labs will utilise standard research grade reagents, with often reduced purity and more importantly, minimal traceability of components/ingredients.
Given the relatively young history of cell-based therapies, many academic and pre-clinical labs develop and use manufacturing processes that they recognise will not be what they eventually take to market.
Despite the pace of transition for academic discoveries to applied research and eventual commercialisation increasing significantly over the last decade, challenges relating to chemistry, manufacturing and controls, as well as alignment with regulatory and manufacturing frameworks, do remain and must be overcome if we are to see the desired result for patients. This is even harder for advanced therapy medicinal products (ATMPs) given the ambiguity that remains in such an innovative but young field. Taking a discovery from academia to the commercial market is historically a difficult process. Nearly 30 years ago, scientists discovered methods to derive embryonic stem cells, which have the potential to make any cell in the body, from early mouse embryos A lack of treatment options means these patients have the poorest health outcomes and quality of life, impacted by their poor speech-perception abilities. Cochlear implants are simply not effective for individuals diagnosed with auditory neuropathy, caused by a disruption of the nerve impulses travelling from the inner ear to the brain. With no approved pharmaceutical treatments available, patient options are limited to hearing aids or cochlear implant devices that work with an individual’s remaining sense of hearing to amplify sounds – but crucially, this does not restore natural hearing. Mammals cannot repair or regenerate auditory sensory cells, leading to an irreversible loss of hearing should they be damaged. Hearing loss is the most common global sensory disability, affecting more than 430 million individuals worldwide, 1 with these numbers expected to rise with increasing and ageing populations.
In this article, Dr Terri Gaskell, Chief Technology Officer at Rinri Therapeutics, explores some of the pre-clinical challenges and questions associated with using a novel stem cell technology as a potential therapeutic strategy for hearing loss. Expectations are growing worldwide for discoveries that will harness the potential of cell therapy, which has already brought breakthroughs in therapeutic areas where there is an unmet need, from oncology to ophthalmology and rare disease.