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

Spliceor Ltd是剑桥大学一家早期的衍生公司，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。Spliceor针对的是专门在癌细胞中发现的分子，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，它们为这种方法提供了理想的目标。利用这些，Spliceor的目标是为许多类型的癌症产生一种平台方法，从肝癌和胰腺癌开始，为癌症患者产生治愈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，以指导后续关键的临床前疗效研究的治疗剂量范围，帮助将该疗法推向制造、安全测试和临床试验。它还将有助于完善我们的商业计划，并成为确保公司未来关键投资的垫脚石。这笔资金将使公司能够扩大规模并创造新的就业机会，为改善医疗保健结果和亟需的经济增长做出贡献。

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.