Recent advances in upstream process development for production of recombinant adeno-associated virus.
Biotechnol Bioeng
; 121(1): 53-70, 2024 01.
Article
em En
| MEDLINE
| ID: mdl-37691172
ABSTRACT
Recombinant adeno-associated virus (rAAV) is rapidly emerging as the preferred delivery vehicle for gene therapies, with promising advantages in safety and efficacy. Key challenges in systemic in-vivo rAAV gene therapy applications are the gap in production capabilities versus potential market demand and complex production process. This review summarizes current available information on rAAV upstream manufacturing processes and proposed optimizations for production. The advancements in rAAV production media were reviewed with proposals to speed up the cell culture process development. Furthermore, major methods for genetic element delivery to host cells were summarized with their advantages, limitations, and future directions for optimization. In addition, culture vessel selection criteria were listed based on production cell system, scale, and development stage. Process control at the production step was also outlined with an in-depth understanding of production kinetics and quality control.
Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Dependovirus
/
Vetores Genéticos
Tipo de estudo:
Risk_factors_studies
Idioma:
En
Revista:
Biotechnol Bioeng
Ano de publicação:
2024
Tipo de documento:
Article
País de afiliação:
Estados Unidos