Developing non-viral nanopore drug delivery technology for first-in-human trials

rna疗法的新浪潮有可能改变对现有小分子或抗体治疗无效的癌症患者的预后。在Covid疫苗取得成功的背景下，rna疗法已经走到了最前沿，许多生物技术公司正在为目前没有反应的患者群体开发这些疗法。这个价值60亿英镑的新型rna疗法的潜在市场需要一种安全的药物输送技术：保护疗法在体内不被降解，将它们靶向肿瘤，并将它们释放到细胞内。Vitarka是一家英国生命科学技术中小企业，正在开发一种新的细胞内递送技术——EndoPore。EndoPore是一种药物输送技术，它将携带rna治疗药物穿过人体，然后将它们释放到肿瘤细胞内，从而为新的抗癌治疗开辟了许多目前经过验证但无法药物治疗的靶点。通过这笔拨款，我们将通过建立配方和gmp级的大规模生产来进行首次人体临床试验，从而大大降低开发路径的风险。我们将开始验证临床相关的配方，并确定监管相互作用的路线图。这将使该项目获得更大规模的私人投资，并与生物技术公司合作进行临床试验，从而使Vitarka和英国能够迅速产生技术和商业影响。

A new wave of RNA-therapeutics has the potential to transform outcomes for patients with cancers which do not respond to existing small molecule or antibody treatments. RNA-therapeutics have come to the forefront on the back of Covid vaccine success, and many Biotechs are now developing these for currently non-responding patient groups. This £6Bn potential market of new RNA-therapeutics needs a safe drug-delivery technology: to protect the therapeutics from degradation within the body, to target them to the tumour, and to release them inside the cells.Vitarka is a UK life sciences technology SME which is developing a novel intracellular delivery technology, EndoPore. EndoPore is a drug delivery technology which will carry RNA-therapeutics through the body and then release them within tumour cells - opening up many currently validated but undruggable targets for new anti-cancer treatments.Through this grant, we will substantially de-risk the development path by establishing formulation and GMP-grade large-scale manufacturing of our technology for first-in-human clinical trials. We will start validating clinically relevant formulations and define the roadmap for regulatory interaction. This will position the programme for larger scale private investment, and for partnership with biotechs for clinical trials, thereby enabling rapid technological and commercial impact for Vitarka and the UK.