I Corps: Development of Genome Edited Cancer Vaccines

这个i-Corps项目的更广泛的影响/商业潜力是将一种新的癌症免疫疗法疫苗推向市场，用于治疗实体和转移性肿瘤。在过去的几十年里，实体肿瘤的发病率和死亡率在美国是最高的。免疫治疗的许多最新进展，包括双特异性抗体和CAR-T细胞治疗，对实体瘤的影响微乎其微。该小组发明的疫苗填补了这一空白，提供了一种个性化的免疫疗法疫苗，可以阻止或减缓癌症的进展，并防止其复发。通过用这种新开发的疫苗治疗那些无法治愈的癌症，它将拯救数百万人的生命。该计划是调查其客户群，发现其市场，并了解其监管审批流程。最初的客户发现将是NSF I-Corps短期课程的重点，随后是市场发现和筹集资金的战略，以将疫苗推向市场。I-Corps项目是将基于该小组的一项发现开发的免疫疗法疫苗商业化，发现基因组编辑的肿瘤细胞对实体瘤具有很强的免疫原性，从而发挥疫苗的作用。最初的动物研究支持这种方法的可行性。在癌症免疫治疗和基因组编辑的巨大进步基础上，这种疫苗协同结合了CRISPR基因组编辑、疫苗接种和个性化药物，以充分激活患者的免疫系统，防止癌症复发。这是第一种可防止癌细胞逃避免疫系统的基因工程肿瘤疫苗。与传统的癌症治疗不同，它没有严重的非靶向副作用。虽然癌症疫苗已经得到了广泛的探索，但由于监管批准和采用现有治疗制度方面的障碍，其临床开发经历了很长一段时间的滞后阶段。该小组开发的疫苗将克服这些障碍。这一奖项反映了NSF的法定使命，并通过使用基金会的智力优势和更广泛的影响审查标准进行评估，被认为值得支持。

The broader impact/commercial potential of this I-Corps project is to bring a novel cancer immunotherapy vaccine to the market for treating solid and metastatic tumors. Solid tumors exhibit the highest rates of incidence and mortality in the United States for the past few decades. Many of the newest advancements in immunotherapy, including bi-specific antibodies and CAR-T cell therapy have shown minimal effect on solid tumors. The vaccine invented by this group fills the gap by providing a personalized immunotherapy vaccine that can stop or reduce the progression of the cancer and prevent its recurrence. It will save millions of lives by treating those incurable cancers with this newly developed vaccine. The plan is to investigate its customer segments, to discover its market, and to understand its regulatory approval processes. The initial customer discovery will be the focus of the NSF I-Corps Short Course, followed by a market discovery and a strategy to raise funds for bringing the vaccine to the market.This I-Corps project is to commercialize an immunotherapy vaccine that was developed based on a discovery made by this group, where genome edited tumor cells were found to elicit a strong immunogenicity against solid tumors, thereby functioning like a vaccine. The initial animal studies supported the feasibility of this approach. Building upon tremendous advances in cancer immunotherapy and genome editing, this vaccine synergistically combines CRISPR genome editing, vaccinations, and personalized medicine to fully activate a patient's immune system against cancer recurrence. This is the first engineered tumor vaccine that prevents cancer cells from evading the immune system. Unlike conventional cancer therapy, it does not have severe off-target side effects. While cancer vaccines have been explored extensively, their clinical development has seen a long lag phase due to hurdles in regulatory approvals and their adoption into existing therapy regimes. The vaccine developed by this group will overcome these hurdles.This award reflects NSF's statutory mission and has been deemed worthy of support through evaluation using the Foundation's intellectual merit and broader impacts review criteria.