First in class to first in human: Harnessing physiological regulation mechanisms to enhance safety and efficacy of next generation bispecific antibodies

同类首创到人类首创：利用生理调节机制增强下一代双特异性抗体的安全性和有效性

• 批准号: 10028428
• 金额: $ 75.26万
• 依托单位: NOVALGEN LIMITED
• 依托单位国家: 英国
• 项目类别: Collaborative R&D
• 财政年份: 2022
• 资助国家: 英国
• 起止时间: 2022 至 无数据
• 项目状态: 未结题
• 来源: https://gtr.ukri.org/projects?ref=10028428
• 关键词: First; class; first; human; Harnessing

Therapeutic antibodies are biological therapies that have revolutionised the way we treat cancer and inflammatory diseases. Novalgen, an emerging immunotherapy company, has developed a novel and smart type of antibody that stops exerting its therapeutic activity once they have completed their activity. In so doing, the NovalGen technology offers a new generation of antibody therapies with potential for maximal safety alongside maximal efficacy.Our first smart antibody therapy will treat Haemophilia A (HA), an inherited condition in which blood doesn't clot normally because it lacks sufficient blood-clotting proteins (clotting factors). Consequently, patients with haemophilia, experience spontaneous bleeds or bleed for a longer time after an injury than normal. Our new antibody drug will replace the missing clotting factor (FVIII) in patients with HA, but with the peculiarity that it will get inactivated once it has helped the formation of a blood clot, to avoid potentially life-threatening side effects associated with uncontrolled clotting.Before we test this new drug in people, we need to test it in the laboratory to establish that its safety and efficacy. This requires a comprehensive data package demonstrating that it is worth progressing this candidate drug to the clinic.The Biomedical Catalyst grant will enable NovalGen to develop very detailed data packages and prepare a clinical trial application which will be submitted to the UK regulatory agency (the Medicines and Healthcare Products Regulatory Agency \[MHRA\]) at the end of the project, to gain permission to administer this new drug to haemophilia patients, the first step in bringing a new drug to the market.Haemophilia products have a very good track record of obtaining approval if they clear the first hurdle of a Phase I clinical study, so funding from UKRI in support of this program could result in the UK being at the forefront of HA treatment in the future. Success with this drug will encourage NovalGen and other companies to make other "smart drugs" representing a step change in the way we treat diseases.

治疗性抗体是一种生物疗法，它彻底改变了我们治疗癌症和炎症性疾病的方式。Novalgen是一家新兴的免疫治疗公司，它开发了一种新型的智能抗体，一旦它们完成了活性，就会停止发挥其治疗活性。通过这种方式，NovalGen技术提供了新一代的抗体疗法，具有最大的安全性和最大的疗效。我们的第一个智能抗体疗法将用于治疗血友病A（HA），这是一种遗传性疾病，由于缺乏足够的凝血蛋白（凝血因子），血液无法正常凝固。因此，血友病患者在受伤后会经历自发性出血或出血时间比正常人长。我们的新抗体药物将取代HA患者中缺失的凝血因子（FVIII），但其特点是一旦它帮助形成血凝块，它将被灭活，以避免与不受控制的凝血相关的潜在危及生命的副作用。在我们测试这种新药之前，我们需要在实验室中进行测试，以确定其安全性和有效性。这需要一个全面的数据包，证明这是值得发展的候选药物的临床。生物医学催化剂赠款将使NovalGen开发非常详细的数据包，并准备临床试验申请，将提交给英国监管机构（药品和保健品管理局[MHRA]）在项目结束时，获得许可，将这种新药用于血友病患者，这是将新药推向市场的第一步。如果血友病产品清除了I期临床研究的第一个障碍，则其获得批准的记录非常好，因此UKRI支持该计划的资金可能会使英国在未来处于HA治疗的最前沿。这种药物的成功将鼓励NovalGen和其他公司生产其他“智能药物”，代表着我们治疗疾病方式的一步改变。