ADEPT-STAR therapy for high risk neuroblastoma

项目总结 尽管在治疗方面取得了重大进展，但癌症仍然是一个主要的健康问题。在过去的十年里，细胞 基因(C&G)疗法和免疫肿瘤学(I-O)疗法已经改变了治疗预期 通过延长无癌生存期和完全缓解。尽管取得了这些令人瞩目的成就，但它们是有限的 对于癌症的一个子集，在更广泛的应用和解锁被感知的 医疗和商业潜力。Expression Treateutics，Inc.(ET)是一家完全整合的C&G治疗公司 通过使用专有和互补的平台技术来解决这些未得到满足的临床需求 优化治疗性转基因表达和基因传递，开发现成的同种异体基因 以基因疗法为基础。这些技术解决了C&G I-O中遇到的主要挑战：识别 安全有效的肿瘤靶点，最大限度地提高治疗的耐用性和有效性，并降低商品成本。 初步的体外和体内研究结果支持了拟议应用的可行性和成功的 这一治疗儿童神经母细胞瘤和其他形式神经母细胞瘤的强大平台的开发 神经内分泌肿瘤(Nets)就是在这种应用中提出的。

PROJECT SUMMARY Cancer remains a primary health concern despite significant advancements in treatment. In the past decade, cell and gene (C&G) therapies along with immuno-oncology (I-O) therapies have transformed treatment expectations by extending cancer free survival and complete remissions. Despite these remarkable successes, they are limited to a subset of cancers and many significant challenges remain for broader application and to unlock the perceived medical and commercial potential. Expression Therapeutics, Inc. (ET) is a fully integrated C&G therapy company addressing these unmet clinical needs by using proprietary and complementary platform technologies that optimize therapeutic transgene expression and gene delivery for the development of off-the-shelf allogeneic- based gene therapies. These technologies address major challenges being encountered in C&G I-O: identification of safe and effective tumor targets, maximizing treatment durability and efficacy and reducing the cost of goods. Results of preliminary in vitro and in vivo studies support feasibility of the proposed application and the successful development of this robust platform for the treatment of pediatric neuroblastomas and other forms of neuroendocrine tumors (NETs) proposed in this application.