Chemistry, manufacturing and control considerations of rAAV-based gene therapy products
10.16438/j.0513-4870.2019-0789
- VernacularTitle:关于重组腺相关病毒基因治疗制品药学评价的思考
- Author:
Bo-ning LIU
1
;
Long-chang XU
1
;
Dong-mei HAN
1
;
Jian-hui LUO
1
Author Information
1. Center for Drug Evaluation, National Medical Products Administration, Beijing 100022, China
- Publication Type:Research Article
- Keywords:
recombinant adeno-associated virus;
gene therapy;
chemistry manufacturing controls review;
manufacturing process;
quality control
- From:
Acta Pharmaceutica Sinica
2020;55(8):1965-1970
- CountryChina
- Language:Chinese
-
Abstract:
Recombinant adeno-associated virus (rAAV)-based vector has shown great promise for human gene therapy, due to its advantage in eliciting long-term transgene expression, absence of adverse effect, infection ability to both dividing and non-dividing cells, non-genomic integration, and low immunotoxity. To date, three AAV-based products have been authorized to enter European and American markets, and more than 200 rAAV-based candidates are in the process of clinic trails. Nevertheless, domestic industry is facing the challenge of manufacturing clinical grade rAAV vector, and regulatory agencies are lack of practical experience in assessing such products. Herein, this paper summarizes the latest research progress of rAAV-based gene therapy products, and discusses some quality assessment concerns in raw materials, manufacturing process and quality control, expecting to promote its clinical transformation and application.
