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NCATS-DPI-22-0101 - CREATION OF AN OPEN-SOURCE PROCESS TO MANUFACTURE AAV VECTOR-BASED GENE THERAPY CANDIDATES FOR NON-COMMERCIAL CLINICAL STUDIES

NCATS-DPI-22-0101 - CREATION OF AN OPEN-SOURCE PROCESS TO MANUFACTURE AAV VECTOR-BASED GENE THERAPY CANDIDATES FOR NON-COMMERCIAL CLINICAL STUDIES
NCATS-DPI-22-0101 - 创建开源流程来制造用于非商业临床研究的基于 AAV 载体的基因治疗候选药物
批准号:
10716633
负责人:
VENKATA MANGALAMPALLI
金额:
$930.83万
依托单位国家:
美国
项目类别:
财政年份:
2022
资助国家:
美国
项目状态:
未结题
起止时间:
2022-09-12 至 2027-09-11

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中文摘要
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项目目标:
英文摘要
Project Objectives: • Develop an open- access process for the generation of at least two of the AAV serotypes broadly in current use. All AAV serotypes will use a GFP gene, and a gene of interest (GOI) identified by NCATS to validate the lab scale process. • Demonstrate the scalability of that process by manufacturing the NCATS GOI for all AAV serotypes (non-GMP) at 50 L pilot scale and subsequently at a 200 L bioreactor scale. • Develop all the required analytical methods for in-process testing and quality attributes of the material generated throughout the development stages to support regulatory filings. • Provide comprehensive project management support • Provide quality audit support during the process development and final process definition stage • Provide regulatory support for preparation of the Chemistry, Manufacturing and Controls (CMC) section of an Investigational New Drug (IND) application • Manufacture AAV-GOI material at cGMP for clinical use and validate relevant analytical methods for release and stability analysis. • Perform stability studies on the drug substance and drug product for up to 48 months • Provide inventory support for storage and shipping of cell banks, drug substance and drug product
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SCIENCE CHINA Chemistry
Science China Chemistry
运用Linkage Chemistry合成新型聚合物缀合物和刷形共聚物
  • 批准号:
    20974058
  • 项目类别:
    面上项目
  • 资助金额:
    12.0万元
  • 批准年份:
    2009
  • 负责人:
    袁金颖
  • 依托单位: