Pre-clinical Development of novel ‘self’-antibodies for disease modification in Huntington’s Disease

炼金术治疗公司（Alchemab Therapeutics）有一种接近药物发现的新方法，即专注于让人们保持健康的因素，有时甚至是克服困难。我们所有人都会产生大量不同的抗体，作为我们通常免疫反应的一部分，以抵御病原体并帮助身体保持健康状态。我们相信，有些人能够利用这种机制来保护自己免受他们易患的疾病的侵害。亨廷顿舞蹈病（HD）是一种毁灭性的神经退行性疾病，目前没有任何药物可以减缓或阻止其进展。它有一个遗传原因，在HD高危家庭的成员中进行筛查。Alchemab分析了对各种神经退行性疾病有抵抗力的患者的B细胞（产生抗体），并确定了抵抗疾病的个体共有的抗体，而在出现症状的患者中不存在。我们已经确定了抗体，我们相信这种抗体可能对减缓亨廷顿氏病的进展有特别的作用。这笔拨款将加速我们的先导抗体的临床前开发，以证明监管机构要求的首次人体临床试验的安全性、有效性和可制造性，为后期开发阶段做准备，并吸引所需的资金，使我们通过一个创新的临床项目进入市场。我们的目标是开发和测试这些抗体作为治疗方法，以帮助患有HD诊断的个体，这些个体可能没有那么幸运，无法自己产生保护性抗体反应，这可能在减缓疾病进程方面具有变革性。我们希望这将帮助我们比传统药物发现更快地为亨廷顿舞蹈症患者带来急需的治疗方法。

Alchemab Therapeutics has a new way of approaching drug discovery by focussing on what keeps people well, sometimes against the odds. All of us produce a vast repertoire of different antibodies as part of our usual immune responses to fend off pathogens and to help the body maintain its health status. We believe that some individuals are able to use this mechanism to protect themselves against diseases to which they are pre-disposed. Huntington's disease (HD) is a devastating neurodegenerative disease, without any current medicines to slow or stop its progress. It has a genetic cause that is screened for in members of HD high-risk families. Alchemab analysed B cells (which produce antibodies) of patients resilient to various neurodegenerative disorders and identified antibodies common to those individuals resisting disease and not present in those patients who are showing symptoms. We have identified antibodies which we believe may be of particular utility in slowing progression in Huntington's Disease. This grant will accelerate the pre-clinical development of our lead antibody to demonstrate the safety, efficacy and manufacturability profile required by regulators for first in human clinical trials, to prepare for later stages of development and to attract the funding required to take us through an innovative clinical program to the market. Our aim is to develop and test these antibodies as therapies to help individuals living with a HD diagnosis that may not have been so lucky as to develop a protective antibody response themselves, which could be transformative in slowing the course of the disease. We hope this will help us to bring much needed therapies to Huntington's Disease patients faster than traditional drug discovery.