Understanding The Gene Therapy Manufacturing Process

BlogApril 15, 2020

Gene therapy is the process of replacing damaged or unhealthy genetic material with new material for treating various diseases and conditions. Curious to learn more about the process of gene therapy manufacturing? At Andelyn Biosciences, we’re a leading provider of this service, so let’s break down the details now.

What Is Gene Therapy And How Does It Work?

Gene therapy is a type of medicine that treats or prevents diseases by inserting genes into a patient’s body. This is still an experimental technique, but if it’s perfected, it may allow some diseases and conditions to be treated without drugs, surgery, or other traditional medical techniques.

It works by using a “viral vector,” which is a special type of inert virus that carries some genetic material like DNA or mRNA. This virus is able to quickly “infect” the body and its cells, delivering new genetic material to the cells, where it can replace the damaged or improper genetic material.

How Do You Manufacture A Gene Therapy Treatment?

The gene therapy manufacturing process is very complex and can vary somewhat depending on the type of viral vector being used. However, it uses similar processes to other biopharma products like vaccines.

At a high level, this involves culturing and creating a small amount of the gene therapy virus along with a specific type of “host cells,” and special processes are used to duplicate the cells until the desired concentration of viral vectors has been reached.

This is necessary because viruses cannot duplicate on their own – they must do so using other cells. Then, the host cells are processed and the viral vectors are isolated and purified for later use.

What Are The Steps That Go Into Manufacturing A Gene Therapy Treatment?

Four basic steps are involved in the gene therapy manufacturing process, as follows:

  1. Preparing raw materials – First, three components are assembled into the gene therapy vector – the vector genome, AAV protein, and helper proteins. These are combined into a “shell” called a capsid, which will function as the “vehicle” that carries a “cargo” of functioning genes into the cells.
  2. Upstream process – The capsids will be assembled and integrated with an inert virus, creating the viral vector. Special cells are used as “hosts” for the viral vector, allowing it to reproduce and reach the required concentration for harvesting.
  3. Downstream process – In this stage, the viral vector is processed to purify it and remove non-essential matter. Then, it will be reviewed and tested to make sure it’s functioning properly.
  4. Completion & packaging – Finally, the viral vector will be processed and packaged to be sent away for clinical use.

How Long Does It Take To Develop and Manufacture A Gene Therapy Treatment?

This depends mostly on the type of viral vector, the targeted disease, and some other factors. But, in general, it takes about 9-10 months to develop and manufacture a gene therapy treatment.

Contact Andelyn Biosciences For Specialized Gene Therapy Manufacturing

If you need a partner for the gene therapy manufacturing process, Andelyn Biosciences is always standing by to help. With our advanced manufacturing technology, skilled personnel, and spotless track record in the process of gene therapy manufacturing, you can trust us to help you bring new viral vectors to market.

To learn more and get started, contact us online to speak to one of our team members and get more information about what we do – and how we can help your organization with our expertise in the gene therapy manufacturing process.