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Gene Therapy

Gene and gene-modified cell therapies treat diseases by delivering therapeutic DNA into a patient’s cells. To ensure effective clinical translation and commercialization, the bioprocessing community must devote intense effort to scalability of vector manufacturing. Applications in this therapeutic class inclue the bioprocessing of
       • Viral vectors
       • Plasmid DNA

Recent advances in genomic sequencing is positioning gene and gene-modified cell therapy as a new therapeutic class that could rival monoclonal antibodies (mAbs) in importance.

Gene and gene-modified cell therapies treat diseases by delivering therapeutic DNA into a patient’s cells. The first step is packaging a therapeutic transgene into a delivery vehicle (e.g., a viral vector), followed by expansion of its host cell lines to produce high-enough vector concentrations.

In direct gene delivery, therapeutic transgenes are injected directly into target tissues in vivo. Cell-based gene therapy requires isolation and ex vivo cultivation of the target cells before genetic modification, followed by expansion and enrichment for readministration into a patient. 

gene therapy process FLOWs and applications

Viral vector manufacturing

To manufacture viral vectors, two approaches generally are adopted for culturing purposes: scale-out of adherent-cell systems and scale-up of suspension cell cultures in stirred-tank bioreactors. For large-scale manufacturing, developers are increasingly choosing to operate larger-scale apparatus such as stainless steel or single-use bioreactor culture systems. Industrialization of viral vector manufacturing is maturing as companies tackle problems in process control, scale-up, facility design, characterization and quality, and regulatory considerations.

 

Cell culture ►

 

Cell line optimization
Cell banking
Media development
Seed train optimization
Cell culture expansion
Analytics

Applications
Perfusion cell culture

Repligen solutions

 

Infection ►

 

(if needed)

 

 Harvest

 

Centrifugation
Depth filtration
Analytics

 

Purification ►

 

Resin screening
Capture / Polish chromatography
Tangential flow filtration
Scale-down validation
Analytics

Application
Pre-packed columns
Concentration and Dialfiltration

Repligen solutions

 

Formulation

 

Filter screening
Tangential flow filtration
Analytics

Application
Concentration and Diafiltration

Repligen solutions

Plasmid manufacturing

Plasmid DNAs (pDNAs) serve as nonviral gene delivery vehicles for some gene therapies. With a favorable safety profile, low toxicity, and an ability to deliver larger genetic loads, pDNAs provide an alternative option to viral-mediated approaches.

 

Fermentation ►

 

 

Plasmid recovery ►

 

Clarification
Media exchange
Analytics

Application
Diafiltration

Repligen solutions

 

Purification ►

 

Capture / Polish chromatography
Tangential flow filtration
​​​​​​​Analytics

Application
Pre-packed columns
Concentration and Diafiltration

Repligen solutions

 

Formulation

 

Tangential flow filtration
​​​​​​​Analytics

Application
Concentration and Diafiltration

Repligen solutions