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，造成巨大的个人、社会和经济困难。绝大多数听力损失病例是由感觉毛细胞和/或其相关神经元的丧失引起的。一般来说，哺乳动物，尤其是人类，没有能力恢复这些细胞。我们生来就有一堆可以维持一生的细胞。当这些受损时，就会导致永久性耳聋。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，这些设备并不能解决潜在的细胞丢失问题。目前，还没有能够再生或恢复听力损失细胞的治疗方法。Rinri Therapeutics公司正在开发一种名为“Rincell-1”的新疗法，这种细胞疗法有可能取代内耳中死亡或受损的神经细胞，并恢复听力。为了达到临床试验，Rinri需要将目前在研究实验室中使用的相关细胞的生成方法调整为符合严格的制造和安全法规的工业标准。Rinri一直在与谢菲尔德大学的创始人和原始技术的创造者合作，翻译学术实验室的研究成果，使其与细胞制造组织兼容。我们请求为在谢菲尔德大学工作的具有听力损失和干细胞技术专长的研究人员Leila Abbas博士借调到Rinri提供资金，以便管理和加速这些工业过程的发展。这次借调将直接有利于Leila， Rinri，谢菲尔德大学和听力损失科学界。具体来说，Leila将学习到制造细胞药物产品所涉及的策略、挑战和法规。加上她现有的研究技能，这将为她在听力领域的生物技术行业提供几乎独一无二的技能，可以应用于未来开发其他新型听力疗法，这是解决全球听力损失负担所迫切需要的。同样，蕾拉在听力和耳聋基础生物学、干细胞行为方面的广泛知识，以及她对听力科学家和Rincell-1的目标人群（听力损失人群）的独特理解，将令Rinri受益。最终，这次借调将加强Rinri和谢菲尔德大学之间的思想交流，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.