First-in-class fully-biodegradable DNA delivery system for biomedical research and safe gene therapy

在细胞内转移外来DNA分子是生物医学研究中常规使用的一项基本实验室技术，也是治疗囊性纤维化、遗传性眼病和几种癌症等功能障碍基因引起的疾病的一种有希望的治疗方法。除了有争议的病毒载体外，DNA的传递还可以使用化学试剂，这种试剂能够使DNA分子复杂化，并将它们转移到细胞质中。然而，体内给药在很大程度上受到试剂毒性、DNA大小和靶细胞/组织的限制。在这项可行性研究中，DELIVERICS将研究一种新的可生物降解的递送系统，以实现DNA在体内细胞内的安全转移。这一独特的工具将解决研发和制药部门都存在的商业机会，在这两个部门，研发研究和基于DNA的生物制药的治疗性输送需要先进的输送系统。

The transfer of foreign DNA molecules inside cells is an essential laboratory technique routinely used in biomedical research and a promissing therapeutic approach to treat disorders caused by malfunctioning genes such as cystic fibrosis, inherited eye diseases and several kinds of cancer. Apart from the controversial virus-based carriers, DNA delivery can be achieved using chemical reagents with the ability to complex DNA molecules and translocate them into the cell cytoplasm. However, in vivo delivery is largely limited by the reagent toxicity, the DNA size and the target cell / tissue. In this feasibility study, DELIVERICS will investigate a novel biodegradable delivery system to achieve safe transfer of DNA inside cells in vivo. This unique tool will address a commercial opportunity that exists both in the R&D and Pharma sectors, where advanced delivery systems are required for R&D studies and for the therapeutic delivery of DNA-based biopharmaceuticals.