Insights On Cell and Gene Therapy Manufacturing
BlogSeptember 15, 2020

Gene therapy has revolutionized medicine since the early 1990s and is one of the most promising fields in medicine. In the coming years it will continue to become more relevant in the treatment and cure of some of the most severe diseases and disorders that affect humans.

What is gene therapy and how does it work?

Gene therapy is the means through which new healthy genetic material is inserted into human cells to modify faulty, corrupted, or missing genes. To deliver genetic material to target cells, scientists use a neutralized virus that can be engineered to modify genetic conditions such as cystic fibrosis, hemophilia, heart disease, and more. A vital difference between this vehicle virus and other viruses is, this neutralized agent doesn’t threaten human health because it cannot replicate in the body without external help. This makes it an ideal vehicle for healthcare professionals to deliver genes by infecting cells with precisely engineered genetic material. Once the genetic material is inside the target cell, the new gene is released and the target cell begins to synthesize the protein from the code contained in the new gene.

What is the goal of gene therapy?

Gene therapy was created to correct mutations or missing genetic material that causes severe diseases and debilitating disorders. A key difference that sets genetic therapy apart from traditional medicine is that it’s designed to stop or reverse a disease’s progress instead of simply managing its symptoms. By altering the genes that cause diseases and disorders, a patient’s lifestyle could improve significantly. Everyday hassles that patients often have to deal with such as continual management of the disease/disorder and reducing dependence on conventional medications could be completely eliminated. Furthermore, the physical, psychological, and emotional impact these diseases or disorders have on patients would be significantly reduced.

How is gene therapy progressing?

Over the past 50 years, gene therapy has made momentous progress toward curing and effectively treating previously incurable genetic diseases, conditions, and disorders. Gene therapy has opened new possibilities to the medical field, and with growing investment from the market, countless companies are seeking ways to manufacture and commercialize their therapies. Since gene therapy products are not a one-size-fits all, manufacturing these products is a complex process that must adhere to various standards and proceedings to ensure the final product is safe and high-quality.

Andelyn’s Work In Gene Therapy Manufacturing

Andelyn Biosciences provides clients with a broad range of viral vector manufacturing platforms and solutions, including AAV adherent and suspension systems along with plasmid manufacturing. When your project is advancing to either clinical testing or commercial manufacturing, there are several things to take into consideration:

  • Scalability: Scalability is a crucial aspect that must be considered when your gene therapy project is advancing toward clinical testing. In order to determine scale, you must identify how much material you require for clinical trials and eventually for commercial manufacturing. From that, you must understand how much a facility can make in a single batch. As product demand and the number of treatment patients increase, it’s important to design a scalable process that will be able to meet the market’s demands while adhering to quality standards.
  • Process development: Due to their infectious nature, viral vector systems are commonly used to transmit their genetic material to target cells. Manufacturing a safe and efficient product is the priority. Manufacturing facilities are continuously using analytics to enable process optimization and improve therapeutic performance.
  • Quality Control: Manufacturing a safe product should be one of the top priorities for gene therapy projects. Partnering with a manufacturing organization that uses the latest technology, has trained personnel, and meets the desired quality metrics will ensure you are developing a safe product.

As a full-spectrum gene therapy partner, Andelyn Biosciences supports clients from preclinical through Phase 3 trials. By being compliant with global regulatory bodies, Andelyn Biosciences maximizes efficiency by utilizing advanced technology to support your needs to develop new gene therapies.

Contact Andelyn Biosciences to easily source quality gene therapy products and clinical manufacturing.