Gene Therapy Products & Technology for Effective Research

Gene therapy offers the prospect of long term and potentially curative benefits to patients with genetic or acquired diseases by harnessing the body’s natural bioprocesses, instead of relying on foreign substances.

Gene therapy uses vectors to deliver a payload that has a therapeutic benefit to the patient. It can involve the direct expression of a therapeutic protein or restore the expression of an under-expressed protein. Payloads include plasma DNA, short interference, and short hairpin RNA (siRNA, shRNA), and micro and messenger RNA, among others. Vectors include viruses, such as AAV, AV, Lenti, and Retro, and non-viral approaches that include cationic lipids and electroporation. Therapeutic payloads can be delivered directly into the patient (viral), or into a cell provided by the patient or a non-related donor.

These approaches can be labeled as in-vivo or ex-vivo and may overlap with the development of therapeutic cell therapy applications. Virus-like particles (VLP) were previously developed for vaccine products and are gaining popularity in gene therapy applications and drug delivery vectors because they can supplement the absence or defective form of an essential protein. VLPs can be expressed in either microbial or mammalian expression systems depending on their complexity.

Avantor® products can help maintain the integrity and activity of gene therapy products during upstream and downstream purification through final fill. Choose from a broad range of cell culture components, fermentation media and supplements, growth factors, reagents and excipients, and single-use use products to help enable aseptic fluid transfer that supports “closed-system” cell culture.

J.T.Baker® Viral Inactivation Solution

Improve viral inactivation efficiency and control of protein reactive impurities with Avantor’s biodegradable J.T. Baker® Viral Inactivation Solution.

Learn More

mRNA vaccines & therapies – Bioprocessing solutions for the mRNA revolution

Avantor offers solutions for every stage of the mRNA vaccine and therapy manufacturing workflow. Partner with Avantor to support your speed to market.

Discover More

Gene Therapy Workflow

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.

Ready to Get Started?

  • Contact us
  • Literature
  • Read case studies
  • Watch videos

Ready to Maximize Production Efficiency?

Our Bioprocessing Solutions team can help you streamline your biomanufacturing processes!

Get Started