[HTML][HTML] AAV vectors applied to the treatment of CNS disorders: Clinical status and challenges

L Kang, S Jin, J Wang, Z Lv, C Xin, C Tan… - Journal of Controlled …, 2023 - Elsevier
In recent years, adeno-associated virus (AAV) has become the most important vector for
central nervous system (CNS) gene therapy. AAV has already shown promising results in …

The state of technological advancement to address challenges in the manufacture of rAAV gene therapies

F Destro, W Wu, P Srinivasan, J Joseph, V Bal… - Biotechnology …, 2024 - Elsevier
Current processes for the production of recombinant adeno-associated virus (rAAV) are
inadequate to meet the surging demand for rAAV-based gene therapies. This article reviews …

AAV process intensification by perfusion bioreaction and integrated clarification

JP Mendes, B Fernandes, E Pineda… - … in Bioengineering and …, 2022 - frontiersin.org
Adeno-associated viruses (AAVs) demand for clinical trials and approved therapeutic
applications is increasing due to this vector's overall success and potential. The high doses …

Adeno-associated virus engineering and load strategy for tropism modification, immune evasion and enhanced transgene expression

X Zhou, J Liu, S Xiao, X Liang, Y Li, F Mo… - International Journal …, 2024 - Taylor & Francis
Gene therapy aims to add, replace or turn off genes to help treat disease. To date, the US
Food and Drug Administration (FDA) has approved 14 gene therapy products. With the …

Towards a scalable bioprocess for rAAV production using a HeLa stable cell line

J Escandell, F Moura, SB Carvalho… - Biotechnology and …, 2023 - Wiley Online Library
The majority of recombinant adeno‐associated viruses (rAAV) approved for clinical use or in
clinical trials areproduced by transient transfection using the HEK293 cell line. However, this …

Single-use chromatographic clarification to eliminate endonuclease treatment in production of recombinant adeno-associated viral vectors

G Thakur, S Mink, H Bak, AD Tustian - Separation and Purification …, 2025 - Elsevier
In current manufacturing processes for recombinant adeno-associated viral vectors (rAAV),
endonuclease treatment is used prior to depth filtration to break down host cell DNA that is …

Assessing multi-attribute characterization of enveloped and non-enveloped viral particles by capillary electrophoresis

RP Fernandes, JM Escandell, ACL Guerreiro, F Moura… - Viruses, 2022 - mdpi.com
Virus-based biopharmaceutical products are used in clinical applications such as vaccines,
gene therapy, and immunotherapy. However, their manufacturing remains a challenge …

AAV vector manufacturing process design and scalability-Bending the trajectory to address vector-associated immunotoxicities

JF Wright - Molecular Therapy, 2022 - cell.com
8. Newby, GA, Yen, JS, Woodard, KJ, Mayuranathan, T., Lazzarotto, CR, Li, Y., Sheppard-
Tillman, H., Porter, SN, Yao, Y., Mayberry, K., et al.(2021). Base editing of haematopoietic …

Process design and comparison for batch and continuous manufacturing of recombinant adeno-associated virus

O Yang, Y Tao, M Qadan, M Ierapetritou - Journal of Pharmaceutical …, 2023 - Springer
Recombinant adeno-associated virus (rAAV) is one of the most widely used viral vectors that
has been used for gene therapy to treat a variety of human diseases. As a newly developed …

Advances and opportunities in process analytical technologies for viral vector manufacturing

SA Sripada, M Hosseini, S Ramesh, J Wang… - Biotechnology …, 2024 - Elsevier
Viral vectors are an emerging, exciting class of biologics whose application in vaccines,
oncology, and gene therapy has grown exponentially in recent years. Following first …