ADEPT-STAR therapy for high risk neuroblastoma

ADEPT-STAR 治疗高危神经母细胞瘤

基本信息

批准号：
10760738
负责人：
Christopher Bradley Doering
金额：
$ 40万
依托单位：
EXPRESSION THERAPEUTICS
依托单位国家：
美国
项目类别：
财政年份：
2023
资助国家：
美国
起止时间：
2023-09-19 至 2024-08-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/10760738
关键词：
Address Affect Allogenic American Biodistribution Categories Cell Line Cell Therapy Cell model Cells Chemistry Childhood Classification Clinical Collection Commercial Sectors Custom DNA cassette Data Development Development Plans Diagnosis Disease remission Dose Embryo Gene Delivery Gene Transfer Generations Genes Genotype Goals Health Hemophilia A Human Immunooncology In Vitro In complete remission Individual Infant Kidney Lead Length Live Birth Malignant Neoplasms Marketing Medical Monoclonal Antibody Therapy Mus Neuroblastoma Neuroendocrine Tumors Oncology Outcome Patients Performance Pharmaceutical Preparations Pharmacology Phenotype Prevalence Process Production Program Development Property Recombinants Red Cross Relapse Survival Rate System T-Cell Development T-Lymphocyte Technology Testing Therapeutic Tissues Traditional Medicine Tumor Antigens Validation Xenograft Model chemotherapy clinical development cost cytotoxicity cytotoxicity test design drug development expectation experience expression vector gene therapy high risk high risk population immuno-gene therapy improved in vivo innovation lead optimization manufacture manufacturing run meetings neuroblastoma cell novel therapeutics pharmacokinetics and pharmacodynamics pharmacologic pre-clinical preclinical development preclinical efficacy product development response standard of care success technology platform therapeutic transgene transgene expression tumor tumor xenograft vector γδ T cells

项目摘要

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.

项目摘要尽管在治疗方面取得了重大进展，癌症仍然是主要健康问题。在过去的十年中，牢房和基因（C＆G）疗法以及免疫肿瘤（I-O）疗法已经改变了治疗期望通过扩展无癌症的生存和完全缓解。尽管取得了巨大的成功，但它们还是有限的癌症的一部分和许多重大挑战仍在更广泛的应用中，并解锁所感知的医疗和商业潜力。 Expression Therapeutics，Inc。（ET）是一家完全集成的C＆G治疗公司通过使用专有和互补的平台技术来满足这些未满足的临床需求优化治疗转基因表达和基因递送，以开发现成的同种异体形式基因疗法。这些技术解决了C＆G I-O中遇到的主要挑战：身份证明安全有效的肿瘤靶标，最大化治疗耐用性和功效并降低商品成本。初步体外和体内研究的结果支持拟议应用的可行性和成功开发这种可靠的平台，用于治疗小儿神经母细胞瘤和其他形式的该应用中提出的神经内分泌肿瘤（NETS）。