Gene therapy upstream processing

Achieve optimal generation of gene therapy vectors with our non-viral transfection and viral transduction products. Avantor^® can provide culture systems that support processes development, scale-up and full manufacturing. Our systems include a wide variety of gene therapy bioprocessing products, including transfection and electroporation products, chemicals, buffers, salts, and reagents, viral vector generation kits, Seradigm animal sera (e.g., FBS), single-use products, and sterile cell factories.

Gene therapy upstream processing typically uses transient or stable transfection to produce viral vectors. Both methods have advantages and disadvantages, so selecting the optimal vector manufacturing process for the specific dose and patient population is important.

Regardless of method used, multiple steps and processes are involved, including:

Plasmid production
Cell expansion
Infection or transfection of the vector producing cells
Production of vectors
Different bioprocessing systems, along with the chosen virus and cell line, depending on the therapeutic application

At Avantor, we are here to help optimize the upstream process workflow to ensure efficient upstream processing for your final gene therapy product. From single-use solution products to viral vector generation kits, our proprietary and supplied products together help ensure your gene therapy bioprocessing solutions meet your manufacturing needs, whether for clinical, research, or manufacturing scale.

Our products are designed to maintain the integrity and efficacy of gene therapy products during upstream processing and support an easy transition to downstream processing. Our systems include a wide variety of gene therapy bioprocessing products, including:

Transfection and electroporation products
Chemicals, buffers, salts, and reagents
Viral vector generation kits
Seradigm animal sera (e.g., FBS)
Single-use products
Sterile cell factories

Avantor® Seradigm

Cell culture experts everywhere are discovering how well their cells thrive on nutritionally superior sera from Avantor Seradigm. Seradigm’s unique collection and manufacturing techniques preserve more of the naturally-occurring growth factors in sera that cells require to thrive. Request information and order samples today!

Biopharma production chemicals and excipients

Avantor manufacturers and distributes biopharmaceutical products and excipients providing structured choice and risk mitigation – for biopharmaceutical development, pilot scale and commercial manufacturing.

Single-use solutions

Avantor is the only open-architecture, single-use provider to offer complete design, manufacturing and logistics to support every stage of your biomanufacturing process—wherever your operations are located across the globe. Our global capabilities and supply chain and quality programs are designed to give the peace of mind so you can focus your time on producing critical therapies, quickly and safely.

Literature

Case studies

Biopharma videos

Gene therapy bioprocessing solutions for discovery, product development & pre-clinical (mammalian)

Gene therapy bioprocessing products for scale-up & production (mammalian)

Gene therapy upstream processing products for discovery, product development & pre-clinical (microbial)

Microbial Selection Antibiotics

Gene therapy upstream bioprocessing products for scale-up & production (microbial)

Viral Vector Characterization

What is Gene Therapy Upstream Bioprocessing?

Bioprocessing of gene therapies is the manufacture of vectors used to treat diseases. Upstream vector manufacturing involves multiple steps and processes, including:

Plasmid production
Cell expansion
Infection or transfection of the vector producing cells
Production of vectors

Different bioprocessing systems are used along with the chosen virus and cell line, depending on the therapeutic application. Other factors considered during gene therapy development include:

Cost of goods
Development timeline
Expected product yield
Time to market

What is a Gene Therapy Viral Vector?

A gene therapy viral vector is a virus that has been modified to deliver therapeutic encoded genes, regulatory RNAs, or other substrates to cells. Specific DNA sequences are designed and synthesized in a laboratory to target specific cells. Modified viruses are an excellent vector to deliver these sequences because viruses can enter cells, including hard-to-reach specific cells. Using viral vectors for gene therapy makes it possible to modify gene expression by programming the cells with targeted genetic material.

Upstream bioprocessing for gene therapy typically uses transient or stable transfection to produce viral vectors. Both methods have advantages and disadvantages, so selecting the optimal vector manufacturing process for the specific dose and patient population is important.

What Is the Difference Between Gene and Cell Therapy?

The difference between gene and cell therapy is that gene therapy involves using a carrier, or vector, to transfer genetic material. The genetic material is inserted into the genome of targeted cells in the body.

In comparison, cell therapy involves introducing or injecting cells that function as desired into the patient's body. Cells may come from the patient (autologous cells) or a donor (allogeneic cells) and are expanded and/or modified before injecting them. Genetic modification, for example to produce CAR T-cell therapies or a modified stem cell therapy, can be achieved using viral vectors, mRNA or gene editing technologies like CRIPSR.Both gene and cell therapies improve body functions, but only gene therapy alters the patient's cellular genetics.

Another difference between gene and cell therapy is in bioprocessing. Gene therapy can be easier to manufacture at larger scales when compared to cell therapy. One reason is that gene therapy upstream bioprocessing is comparable to recombinant manufacturing. Cell therapy bioprocessing remains manual and requires customization that limits its manufacturing capacities.

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We offer upstream support for all areas, including cell culture, cell banking, and onsite media/buffer preparation. Let us reduce the burden of routine tasks and improve the productivity of your gene therapy endeavors.

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