Bioprocessing solutions for gene therapy
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.
Involving the direct expression of a therapeutic protein or restoring the expression of an under-expressed protein, gene therapy uses vectors for delivery of a payload that has a therapeutic benefit to the patient. Payloads include plasma DNA, short interference and short hairpin RNA (siRNA, shRNA), and micro and messenger RNA, among others. Vectors include viruses (e.g., AAV, AV, Lenti, and Retro) and non-viral approaches (e.g., cationic lipids and electroporation). The therapeutic payload can be delivered directly into the patient (viral), or into a cell provided by the patient or a non-related donor (e.g., CAR-T).
Approaches can be labeled as in-vivo or ex-vivo. Thus, the use of gene therapy overlaps with the development of therapeutic cell therapy applications. Previously developed for vaccine products, VLPs are gaining popularity in gene therapy applications and drug delivery vectors to supplement the absence or defective form of an essential protein. Depending on their complexity, VLPs can be expressed in either microbial or mammalian expression systems.
Maintaining the integrity and activity of the gene therapy product during upstream and downstream purification through final fill is achieved with products from VWR, part of Avantor. Choose from a broad range of cell culture components, fermentation media and supplements, growth factors, reagents and excipients, and single-use use products to enable aseptic fluid transfer supporting “closed-system” cell culture.