Gene Therapy Products, Research & Technology

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.