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Gene Therapy Manufacturing

The promise of new gene therapies for patients with few or no treatments available is driving demand for rapid innovation, with hundreds of gene therapies now in development worldwide. But bringing a new gene therapy to life demands a high degree of expertise in viral vector manufacturing and testing. Yet there are many challenges to producing a gene therapy including accelerated time-to-patient, lack of an established process template, and evolving regulatory guidelines.

Gene therapy manufacturers must find their own path through complex obstacles in process development, scale-up, manufacturing, and regulatory compliance ­– all within compressed timelines of just 3-5 years, less than half the time for conventional drugs.

Successful manufacturers must also show agility in clearing regulatory, safety, and process development hurdles. Consequently, alliances with experienced global technology partners or contract development and manufacturing organizations (CDMOs) are often essential for bringing a gene therapy to life.

Related Videos

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Selecting The Right CMO
Selecting The Right CMO
Partnering with a CMO may be the quickest and most efficient way to produce your therapy
Reducing The Risk of Gene Therapy
Reducing The Risk of Gene Therapy
Managing uncertainties can help you avoid regulatory risks
3 Cost Modeling Insights to Reduce Gene Therapy Manufacturing Costs
3 Cost Modeling Insights to Reduce Gene Therapy Manufacturing Costs
Manufacturing costs present a key challenge to gene therapy commercialization
Process Development Mistakes
Process Development Mistakes
There is a sense of urgency to produce curative therapies for genetic diseases, but costly mistakes can happen when rushing through process development
Assuring Viral Safety
Assuring Viral Safety
Adventitious viruses may contaminate production processes
Intensified Virus Purification
Intensified Virus Purification
Often the virus size is too large to enter the bead pores resulting in low dynamic binding capacity
Efficient DNA Clearance
Efficient DNA Clearance
Benzonase® nuclease efficiently digests DNA and RNA into fragments smaller than 5 base pairs
Start Right to Scale Right
Start Right to Scale Right
Successfully scale your gene therapy production by implementing a platform process from the start.

Related Product Resources

Article: Anticipating the Next Decade of Gene Therapy
Article: Beyond 2020: Looking into the Crystal Ball for Gene Therapy
Infographic: Making Rare Disease Therapies Less Rare (Labiotech)
Article: Regionalizing and Democratizing Cell and Gene Therapy
Article: Perspectives of Value-driven, Integrated Solutions in Gene Therapy

Poster: AAV Process Development Guidance Based on Cost Modeling
Article: One Size Does Not Fit All: Utilizing Multiple Technology Providers in Gene Therapy Manufacturing
Brochure: Gene Therapy Capabilities for AAV and Lentivirus Production

Gene therapy development: Grow, purify, formulate

Gene therapy process infographic, from cell culture media to final filtration

Workflow

Viral Vector Upstream Processing

Viral Vector Upstream Processing

Making the right upstream process decisions not only impacts viral vector titer, but downstream processes, timelines, and regulatory acceptance 

Viral Vector Downstream Processing

Efficient virus purification processes can improve yield, decrease time to patient, and lower manufacturing costs

Viral Vector Formulation and Final Fill

Formulating a commercially viable gene therapy demands a high level of application and regulatory expertise

Viral Vector Characterization and Biosafety Testing

Critical biosafety testing and characterization of viral vector products can help to fully analyze key quality attributes: identity, potency, safety, and stability

Viral Vector Contract Development and Manufacturing

CDMO partnerships play a critical role in advancing clinical pipelines and achieving successful commercialization

Related Applications
Cell Therapy Manufacturing
Introducing a new cell therapy demands optimal performance in upstream and downstream processing, process development, testing, and manufacturing. World-class products and partners are critical to success, speeding advancement of therapeutics that are effective, safe, and affordable.
Related Services
Viral Vector CTDMO Services
Our viral and gene therapy manufacturing site in Carlsbad, California has produced over 1,000 batches of virus, meeting rigorous timelines for more than 20 years with a complete range of services, a state-of-the-art cGMP facility, and deep regulatory understanding.
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