药品
制造工艺
产品(数学)
过程(计算)
药理学
计算机科学
医学
材料科学
数学
几何学
复合材料
操作系统
作者
Mohamad Kasim Mohd Som,Benson Gikanga,Varna Kanapuram,Sandeep Yadav
标识
DOI:10.1016/j.xphs.2024.03.024
摘要
Abstract
Adeno-associated viruses (AAVs) have become the delivery medium of choice for a variety of genomic medicine applications i.e., gene therapy, gene editing/regulation, and ex-vivo cell therapy. AAVs are protein-DNA complexes which have unique stability characteristics that are susceptible to various stress exposure conditions commonly seen in the drug product (DP) life cycle. This review takes a comprehensive look at AAV DP formulation and process development considerations that could impact critical quality attributes (CQAs) during manufacturing, packaging, shipping, and clinical use. Additional aspects related to AAV development reviewed herein are: (1) Different AAV serotypes with unique protein sequences and charge characteristics potentially leading to discrete stability profiles; (2) Manufacturing process challenges and optimization efforts to improve yield, recovery and purity especially during early development activities; and (3) Defining and identifying CQAs with analytical methods which are constantly evolving and present unique characterization challenges for AAV-based products.
科研通智能强力驱动
Strongly Powered by AbleSci AI