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Scientific Core One

科学核心一

基本信息

批准号：
10589643
负责人：
Corey Casper
金额：
$ 37.52万
依托单位：
FRED HUTCHINSON CANCER CENTER
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：
2023-01-30 至 2027-12-31
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/10589643
关键词：
Antigen Targeting Antigens Binding Biological Cells Clinical Cloning Complement 4b Complex DNA Data Evaluation Flow Cytometry Formulation Fred Hutchinson Cancer Research Center Future Genetic Transcription HIV Harvest In Vitro Liquid substance Membrane Molecular Sieve Chromatography Nanostructures Phase I Clinical Trials Process Production Protocols documentation RNA RNA amplification RNA delivery RNA replication RNA vaccine Research Ribonucleases Techniques Technology Testing Transfection Vaccine Production Vaccines Venezuelan Equine Encephalitis Virus Western Blotting biophysical properties delivery vehicle design immunogenicity in vivo evaluation manufacture nanoparticle nanoparticle delivery particle plasmid DNA product development research and development stability testing structured lipid vaccine evaluation vaccine platform

项目摘要

Abstract Scientific Core One has the responsibility of creating, validating, and providing R&D-grade HIV-Env immunogen-expressing self-amplifying RNAs (saRNAs) complexed with the nanostructured lipid carrier (NLC) delivery vehicle to project leads at Fred Hutchinson Cancer Research Center for in vivo testing as described in Scientific Project One. AAHI’s saRNA/NLC vaccine platform has previously demonstrated robust immunogenicity against many antigen targets, including HIV, and unparalleled stability. This technology will now be adapted to deliver the HIV immunogens of our partners at the Fred Hutchinson Cancer Research Center. Scientific Core One activities include design and cloning of the saRNA template DNA plasmids, saRNA production using well-characterized and validated in vitro transcription and purification processes, and manufacturing of the NLC delivery formulation. Upon successful saRNA and NLC manufacture, Scientific Core One will complex of saRNA/NLC vaccine and test these vaccines in vitro to verify successful saRNA replication and antigen production. Scientific Core One will also provide transfected cell materials to Scientific Core Two for immunogen antigenicity testing. Finally, the stability of these R&D-grade saRNA, NLC, and complexed saRNA/NLC materials will be verified using standard stability testing techniques to gather necessary data to enable GMP manufacturing by Scientific Project Two, successful filing of an IND and completion of a Phase I clinical trial.

抽象的 Scientific Core One 负责创建、验证和提供研发级 HIV-Env 表达免疫原的自扩增 RNA (saRNA) 与纳米结构脂质载体 (NLC) 复合向 Fred Hutchinson 癌症研究中心的项目负责人运送车辆，进行体内测试，如下所述科学项目一. AAHI 的 saRNA/NLC 疫苗平台此前已表现出强大的功能针对许多抗原靶标（包括 HIV）的免疫原性和无与伦比的稳定性。这项技术将现在可以为 Fred Hutchinson 癌症研究中心的合作伙伴提供 HIV 免疫原中心。科学核心一活动包括 saRNA 模板 DNA 质粒、saRNA 的设计和克隆使用经过充分表征和验证的体外转录和纯化工艺进行生产，以及 NLC 递送制剂的制造。在成功生产 saRNA 和 NLC 后，Scientific Core 人们将复合 saRNA/NLC 疫苗并在体外测试这些疫苗以验证 saRNA 复制是否成功和抗原的产生。科学核心一号还将向科学核心二号提供转染细胞材料用于免疫原抗原性测试。最后，这些研发级 saRNA、NLC 和复合物的稳定性 saRNA/NLC 材料将使用标准稳定性测试技术进行验证，以收集必要的数据通过科学项目二实现 GMP 生产，成功提交 IND 并完成第一阶段临床试验。