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 …

Biolayer interferometry for adeno-associated virus capsid titer measurement and applications to upstream and downstream process development

TM Leibiger, LA Remmler, EA Green, KH Lee - Molecular Therapy Methods …, 2024 - cell.com
Faster and more accurate analytical methods are needed to support the advancement of
recombinant adeno-associated virus (rAAV) production systems. Recently, biolayer …

[HTML][HTML] The Unveiled Novel regulator of Adeno-associated virus production in HEK293 cells

J Yan, Z Li, Y Shu, H Chen, T Wang, X Li, Y Zhang… - Gene, 2025 - Elsevier
The field of gene therapy using Adeno-associated viral (AAV) vector delivery is rapidly
advancing in the biotherapeutics industry. Despite its successes, AAV manufacturing …

Transcriptomics-informed pharmacology identifies epigenetic and cell cycle regulators that enhance AAV production

J Tworig, F Grafton, K Fisher, M Hörer, CA Reid… - … Therapy Methods & …, 2024 - cell.com
Recombinant adeno-associated virus (rAAV) is a widely used viral vector for gene therapy.
However, these vectors have limited availability due to manufacturing challenges with …

Probe capsid structure stability and dynamics of adeno‐associated virus as an important viral vector for gene therapy by hydrogen‐deuterium exchange‐mass …

X Ye, Y Hu, H Qiu, N Li - Protein Science, 2024 - Wiley Online Library
Abstract Adeno‐associated virus (AAV), a widely used gene therapy vector, is a small,
nonenveloped virus that contains a single‐stranded DNA genome with a maximum length of …

Transcriptomics-informed pharmacology identifies epigenetic and cell cycle regulators as enhancers of AAV production

J Tworig, F Grafton, M Horer, CA Reid, MA Mandegar - bioRxiv, 2024 - biorxiv.org
Recombinant adeno-associated virus (rAAV) is a widely used viral vector for gene therapy.
Despite its clinical efficacy, the manufacturing of rAAV faces challenges in productivity and …

Unlocking DOE potential selecting the most appropriate design for rAAV optimization

K Tzimou, D Catalan-Tatjer, LK Nielsen… - bioRxiv, 2024 - biorxiv.org
The production of recombinant adeno-associated virus (rAAV) for gene therapy via triple
transfection is a highly intricate process involving many cellular interactions. Each of the …

Cell Line and Process Development Strategies to Establish HEK293-Based Recombinant Adeno-Associated Virus Production Platforms

EA Green - 2024 - search.proquest.com
Recombinant adeno associated virus (rAAV) vectors have become popular delivery vehicles
for in vivo gene therapies, but demand for rAAVs continues to outpace supply due to low …

Developing strategies to enhance transfection efficiency and mitigate auto-transduction in transient lentiviral vector bioprocessing

TE Williams-Fegredo - 2024 - discovery.ucl.ac.uk
Lentiviral vectors are highly efficient gene delivery vehicles used extensively in the rapidly
growing field of cell and gene therapy to treat a wide range of diseases. Demand for …

[引用][C] Advancing AAV production with high-throughput screening and transcriptomics

CA Reid, M Hörer, MA Mandegar - Cell and Gene Therapy Insights, 2024