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Author Spotlight: Advancing Cell Therapy Manufacturing with Dissolvable Microcarriers
Published on: July 7, 2023
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Gene therapy: challenges in cell culture scale-up
Jenny Shupe1, An Zhang1, Daniel C Odenwelder1
1Pharmaceutical Operations and Technology, Biogen, Inc., 225 Binney Street, Cambridge, MA 02142, USA.
Current Opinion in Biotechnology
|April 10, 2022
Summary
Manufacturing recombinant adeno-associated viral vectors (rAAV) for gene therapy is challenging. This review examines popular rAAV production platforms and cell culture scale-up to improve therapeutic delivery.
Area of Science:
- Biotechnology
- Gene Therapy
- Viral Vector Manufacturing
Background:
- Gene therapy offers potential cures for genetic diseases.
- Recombinant adeno-associated viral vectors (rAAV) are primary in vivo gene delivery vehicles.
- Current rAAV production capacity is a bottleneck for clinical demand.
Purpose of the Study:
- To review current rAAV production platforms.
- To analyze advantages and limitations of these platforms.
- To focus on cell culture process scale-up for rAAV manufacturing.
Main Methods:
- Literature review of industrial rAAV production platforms.
- Analysis of scalability and productivity metrics.
- Focus on cell culture scale-up strategies.
Main Results:
- Multiple rAAV production platforms have emerged with improved productivity.
- Cell culture scale-up presents specific challenges and opportunities.
- Platform selection impacts overall manufacturing efficiency.
Conclusions:
- Optimizing rAAV production platforms is crucial for advancing gene therapy.
- Addressing manufacturing bottlenecks requires strategic scale-up approaches.
- Further innovation in production technology is needed to meet clinical demand.

