Process and non-clinical development of a stem cell therapy for hearing loss

世界上有5亿人患有致残性听力损失，导致巨大的个人、社会和经济困难。绝大多数听力损失是由于感觉毛细胞和/或其相关神经元的丧失造成的。一般来说，哺乳动物，尤其是人类，不具备恢复这些细胞的能力。我们天生就有一批细胞，这些细胞应该可以持续一生。当这些器官受损时，永久性耳聋就会随之而来。目前的护理标准是基于助听器和人工耳蜗等听力设备。然而，这些设备并没有解决细胞丢失的根本问题。目前，还没有可以再生或恢复听力损失细胞的治疗方法。Rinri Treeutics正在开发一种新的治疗方法--Rincell-1，这是一种有可能取代内耳中死亡或受损的神经细胞并恢复听力的细胞疗法。为了达到临床试验，Rinri需要调整目前在研究实验室中使用的产生相关细胞的方法，以满足严格的制造和安全法规的工业标准。Rinri一直在谢菲尔德大学与其创始人和原始技术的创造者合作，将学术实验室的研究转化为与电池制造组织兼容的研究。我们请求提供资金，将谢菲尔德大学在听力损失和干细胞技术方面具有专业知识的研究员Leila Abbas博士借调到Rinri，以管理和加快这些工业流程的发展。这一借调将证明对Leila、Rinri、谢菲尔德大学和听力损失科学界直接有利。具体来说，莱拉将了解制造细胞药物产品所涉及的战略、挑战和法规。加上她现有的研究技能，这将为她提供在听力领域生物技术行业几乎独一无二的技能，可以在未来应用于其他新的听力疗法的开发，这些疗法是解决全球听力损失负担迫切需要的。同样，Rinri将从Leila对支撑听力和耳聋的基础生物学、干细胞行为的广泛知识，以及她对听力科学家和Rincell-1的S目标人群：听力损失人群的独特理解中获益。最终，这次借调将加强Rinri和谢菲尔德大学之间的想法交流，以开发一种革命性的听力损失新疗法。

Half a billion people in the world have disabling hearing loss resulting in enormous personal, social and economic hardship. The vast majority of hearing loss cases are produced by the loss of sensory hair cells and/or their associated neurons. Mammals in general, and humans in particular, do not have the ability to restore these cells. We are born with a complement of cells that should last a lifetime. When these are damaged, permanent deafness ensues. The current standard of care is based on hearing devices, such as hearing aids and cochlear implants. However, these devices do not address the underlying problem of cell loss. Currently, no treatments that can regenerate or restore cells exist for hearing loss.Rinri Therapeutics is developing a new treatment, 'Rincell-1', a cell therapy with the potential to replace dead or damaged nerve cells in the inner ear and restore hearing. In order to reach clinical trials, Rinri needs to adapt the methods to generate the relevant cells currently used in a research lab to an industrial standard that fulfils the stringent manufacture and safety regulations. Rinri have been working with its founder and creator of the original technology at the University of Sheffield, translating the research from the academic lab and making it compatible with a cell manufacture organization. We request funds for the secondment of Dr. Leila Abbas, a researcher with expertise in hearing loss and stem cell technology working at the University of Sheffield, to Rinri, in order to manage and accelerate the development of these industrial processes. This secondment will prove directly beneficial to Leila, Rinri, the University of Sheffield and the hearing loss scientific community. Specifically, Leila will learn about the strategies, challenges and regulations involved in the manufacture of a cell-drug product. Along with her existing research skills, this will provide her with an almost unique skillset in the biotech industry in the hearing field that could be applied to the development of other novel hearing therapies in the future, which are desperately needed to address the global burden of hearing loss. Likewise, Rinri will gain from Leila's extensive knowledge of the fundamental biology underpinning hearing and deafness, the behaviour of stem cells along with her unique understanding of hearing scientists and Rincell-1's target population: people with hearing loss. Ultimately, this secondment will enhance the exchange of ideas between Rinri and the University of Sheffield towards the development of a revolutionary new treatment for hearing loss.