Gene therapy involves manipulating a patient’s genes to treat or prevent disease. It can involve replacing or repairing defective genes, adding new genes to help the body fight disease, or using genes to modify cell behavior. It can also involve modulating gene expression through the introduction of gene regulatory fragments (e.g. promoters and enhancers). Gene therapy can address genetic disorders such ascystic fibrosis, sickle cell anemia, as well as certain types of cancer and viral infections.
Gene Therapy
Gene therapies can provide significant and often curative benefits to patients who have genetic or acquired diseases. Through the direct expression of an engineered protein, diminished or absent in the disease state, gene therapy commonly uses viral vectors to deliver a functional replacement of a gene to patients. Avantor® offers trusted resources to help solve complex challenges in gene therapy workflows.
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Advancing Gene Therapy by Solving Challenges in Scale-Up Manufacturing.
Q&A: Our expert provides new insight into gene therapy.
Discover Avantor’s Gene Therapy solutions
Achieve the optimal generation of gene therapy vectors with our consultative approach, application knowledge, technical/compliance support and extensive product offering.
Extensive product offering to help you optimize the purification and fluid path – significantly improving your efficiency while mitigating your risks.
We’ll partner with you to match your therapy with appropriate gene therapy products and materials.
Our single-use solutions are designed for flexibility and versatility in aseptic fluid handling across the upstream, downstream and final-fill steps of producing gene therapies. Solutions for the gene therapy workflow include our proprietary AvantorAdjustable Volume Sampling System (AVSS) which is designed to aseptically draw a precise small sample volume from your most critical.
What is Gene Therapy?
Gene Therapy Manufacturing
A popular type of gene therapy is viral vectors. Viral vector production is challenging and involves complex upstream processes (production of DNA plasmid, developing a vector expression system, transfection of DNA, cell expansion & harvest) to downstream processes (depth filtration, purification, sterile filtration, fill and finish). We offer a broad range of cGMP chemicals/reagents, bioproduction buffers, fluid handling systems and single-use technologies that span across the entire workflow processes. Consulting with Avantor can help you develop gene therapies faster, from cell culture components, media and supplements to reagents and excipients.
Our experienced team is a trusted partner for scientists involved in breakthrough gene therapy research to advanced gene therapy technology. You can find high-quality gene therapy products and solutions to support every stage of your workflow.
Gene Therapy Products
Optimize your workflow with our innovative gene therapy products and solutions. Avantor provides compliant materials that include:
- Cell culture media and sera
- Bioproduction buffers and solutions
- End-to-end production chemicals from upstream to downstream processing
- Cell Lysis Solution for releasing viral vectors from producer cells
- Endonuclease for DNA/RNA clarification
- cGMP peristaltic pumps and pump tubing essential for fluid transfer and handling in biopharmaceutical production environment
- Scalable mixing systems that are essential for pooling, homogenous mixing, or temporary storage
- Single-use fluid sampling and sterile transfer system
- In-line pressure senor for monitoring the critical fluid paths susceptible blockage or breakage
- High quality pump tubing with consistent flow over lifetime and low particulates
We also offer tailored innovative solutions to help you solve process challenges. These include:
- Customized single-use solutions
- Custom-packaged buffers and excipients
- Customized formats for media, sterile solutions, excipients and preservation reagents
Explore our portfolio of cGMP chemicals, single-use solutions, sera and more.
Advance Your Gene Therapy Progress with Avantor
Avantor® can help you advance your Gene Therapy development at all stages, including early discovery, process development/scale up and commercial cGMP manufacturing.
We offer a broad range of cGMP chemical, reagents, bioproduction buffers, fluid handling systems and single-use technologies that span across the entire workflow processes. Consult with us on:
Scale up
- Fluid handling between unit operations
- Flexible packaging and volume for a wide range of media, buffer and solutions
- cGMP and bioproduction grade chemicals and reagents
- Platform-agnostic and end-to-end fluid path design
- CMC requirements and considerations during development
- Quality, regulatory and GMP-compliance support
Manufacturing Operations
- Optimized fluid path design for flexibility, efficiency and robustness
- Accurate and ascetic sampling for low-volume and high-value products
- Customized single-use solutions
- Custom-packaged buffers and excipients
- Media and buffer preparation strategy and technologies
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