ADEPT-STAR therapy for high risk neuroblastoma

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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.