Gene therapy can be described as the delivery of genetic material into cells in order to generate a therapeutic effect or repair a genetic defect. Gene therapy products belong to the large group of advanced therapy medicinal products (ATMPs) and have gained a lot of attention in recent years. Currently, there are more than 10 gene therapy products already on the market and many more are in (pre-)clinical phases. A gene therapy product usually consists of a genetic material (DNA or RNA) and a vector delivery system. The latter can be virus-based or of non-viral nature.

Viral vectors

The majority of gene therapies utilize viral vectors for gene delivery, such as adeno-associated viruses (AAVs), lentiviruses and retroviruses. While each vector system comes with its own advantages and disadvantages, the common goal is to obtain a stable drug product with a high infectivity, precise target specificity and low toxicity. Coriolis is experienced in developing stable drug products for many types of viral vectors and offers a large variety of services that support your viral vector development including in-depth analytical characterization.

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Non-viral vectors

Gene therapy products may deliver their genetic material by utilizing non-viral vectors. They generally exhibit a lower immunogenicity than viral vectors and are easier to produce at large scale. In the simplest form, naked DNA is used, but its administration to patients often generates only a low gene expression. This can be improved by using carriers such as polymeric or lipid nanoparticles; the latter is being used successfully in mRNA vaccines against COVID-19. Non-viral vectors are usually tailored to each genetic payload and application. Thus, achieving a stable drug product requires a dedicated development accompanied with an in-depth analytical characterization from a scientific expert. Coriolis has a strong scientific background in analyzing non-viral vectors and a wide analytical portfolio suitable to this task.

In vivo versus ex vivo gene therapy products.

In vivo gene therapy describes the delivery of genetic material via administration of the vector drug product to the patient most commonly via injection. In contrast, ex vivo gene therapy describes the genetic manipulation of cells outside the patient’s body using allogeneic and autologous material. Coriolis supports the development of these cell therapy products and procedures with a variety of services.

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Coriolis is your expert partner for the development and characterization of gene therapy products

A gene therapy product should have a good safety profile combined with a high infectivity, to achieve a sufficient gene expression and thus, a therapeutic effect. Moreover, the infectivity should be maintained over the entire shelf-life of the product. Titer and infectivity are important attributes of a virus preparation, and their analytical characterization is a central part of development, manufacturing, and quality control of gene therapy products.

Developing a stable gene therapy drug product is a challenge and requires experienced scientists, a suitable set of analytical tools combined with an adaptable product development plan. Indeed, a dedicated drug product development that takes liquid and lyophilized formulations into considerations, is the most promising approach to obtain a stable product.

Coriolis can draw from 14+ years of experience in formulation development including many successful projects utilizing viral and non-viral vectors. Our scientists have a strong scientific background in virus formulation, particle analysis and the development of infectivity assays. In our new and certified ATMP development facility, we handle cell cultures and viral vectors up to biosafety level S2.