Engineering and preliminary safety assessment of an RNA trans-splicing therapeutic vector for liver cancer.

肝癌 RNA 反式剪接治疗载体的工程设计和初步安全性评估。

• 批准号: 10075525
• 金额: $ 6.37万
• 依托单位: SPLICEOR LTD
• 依托单位国家: 英国
• 项目类别: Grant for R&D
• 财政年份: 2023
• 资助国家: 英国
• 起止时间: 2023 至 无数据
• 项目状态: 已结题
• 来源: https://gtr.ukri.org/projects?ref=10075525
• 关键词: Engineering; preliminary; safety; assessment; RNA

Spliceor Ltd is an early-stage University of Cambridge spin-out company developing novel gene therapies for certain types of aggressive cancers for which current treatments are ineffective. Spliceor targets molecules found exclusively in cancer cells using them to generate a lethal gene product within the cancer cell. The Spliceor team has identified a specific class of molecules that provide ideal targets for this approach. Using these, Spliceor aims to generate a platform approach to many types of cancers, beginning with liver and pancreatic cancer, to generate curative and well tolerated gene therapies for cancer patients. The project will consolidate the evidence we have already obtained and give us important pre-clinical study data to guide therapeutic dosing range for subsequence pivotal pre-clinical efficacy studies to help to progress the therapy towards manufacturing, safety testing and clinical trials. It will also help to refine our business plan and serve as a stepping stone to secure future pivotal investment for the company. This funding will allow the company to grow in scale and create new job opportunities, contributing to improving healthcare outcomes and much needed economic growth.

Spliceor Ltd是一家早期的剑桥大学分拆公司，为某些类型的侵袭性癌症开发新的基因疗法，目前的治疗方法对这些癌症无效。剪接体靶向癌细胞中特有的分子，利用它们在癌细胞内产生致命的基因产物。Spliceor团队已经确定了一类特定的分子，为这种方法提供了理想的靶点。利用这些，Spliceor旨在为许多类型的癌症（从肝癌和胰腺癌开始）提供一种平台方法，为癌症患者提供治愈性和耐受性良好的基因疗法。该项目将巩固我们已经获得的证据，并为我们提供重要的临床前研究数据，以指导后续关键临床前有效性研究的治疗剂量范围，从而帮助将治疗推向生产，安全性测试和临床试验。它还将有助于完善我们的商业计划，并作为确保公司未来关键投资的垫脚石。这笔资金将使该公司能够扩大规模，创造新的就业机会，为改善医疗保健成果和急需的经济增长做出贡献。