Gene therapy product
Develop your gene therapy product with support from a scientific expert
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.
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.
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.
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.
Specialized services for your gene therapy product
Coriolis science-driven solutions are based on our deep understanding of the specific challenges of developing a gene therapy product. Our expert scientists will devise dedicated study designs tailored to the needs of your product and development phase.
Characterization of non-viral vectors
Non-viral vectors are nanoparticulate systems comprising lipids, polymers, or other materials. Their size and concentration are critical for their function to carry and release their genetic payload.
Coriolis has a strong scientific background in analyzing non-viral vectors with or without prior separation of the vector from its payload. Our analytical portfolio covers six orders of magnitude from the low nanometer to the visible size range. This allows us to accurately determine the absence of free nucleotides and the (sub)visible particle content.
Virus titration and infectivity assays
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.
Our scientists develop and apply tailored TCID50 and plaque forming assays to obtain infectious titers for your virus formulations. Further we provide qPCR to determine the viral genome copies. Above that, we offer a large portfolio of particle characterization techniques to quantify virus particles i.e., obtain physical titers.
Assessing and avoiding titer loss
Viral and non-viral vectors have the tendency to lose infectivity (i.e., functional titer) over time. Formulation compositions as well as storage and handling protocols should assure the stability of the vector and avoid a titer loss over time.
Our expert scientists are experienced in developing robust virus preparations and non-viral vector formulations, which maintain the infectivity of the product. We take both, liquid and lyophilized formulations into consideration and assess the stability and infectivity of your product under various storage and handling conditions. This assures that your gene therapy product maintains to be safe and effective until application.
Developing viral vector drug products
Viral vectors are frequently used for gene therapy purposes. Examples include adenovirus, lentivirus, adeno-associated virus (AAV), vesicular stomatitis virus (VSV), herpes simplex virus (HSV), poliovirus, reovirus, parvovirus and measles virus.
An optimal viral vector product should have a good safety profile, low toxicity, high infectivity, and in most cases cell type specificity. Each virus vector type has its own advantages and disadvantages and requires a dedicated development strategy. Our expert scientists are experienced in developing robust viral vector formulations, which maintain their infectivity over the intended shelf-life. Read more about our services related to viral vectors on this page.
Development services for your gene therapy product
Explore our large portfolio of development services. In a unique collaborative approach, we individually design each study to meet your expectations and the needs of your current development phase. You will benefit from the scientific experience and advise of our expert scientists to achieve your development goals faster and with a higher chance of success.
Analytical services for your gene therapy product
Choose from a large set of analytical techniques in a variety of categories or get our expert opinion on the most suitable methods for your analytical challenge. We offer standardized analytical methods for many common sample types and perform dedicated method development specific for your analytical challenge and drug product. Explore our analytical portfolio ranging from discovery phase analysis to lot release testing methods.
Our know-how related to gene therapy products
Coriolis is a science-driven service provider. We perform internal research projects with academic and industrial partners to develop new technologies and create in-depth knowledge, which directly benefits our client projects. Our internal Unit Science & Technology, hosting PhD candidates and Postdocs, also actively contributes to the scientific community with numerous peer-reviewed publications each year. Explore our latest publications, articles, and webinars.
Gust Nuytten, Susan Ríos Revatta, Pieter-Jan Van Bockstal, Ashish Kumar, Joris Lammens, Laurens Leys, Brecht Vanbillemont, Jos Corver, Chris Vervaet and Thomas De Beer
Other product types
We tailor our services to your specific product type and development phase. In a unique collaborative approach, our expert scientists focus on your scientific question or development milestone and support your drug development program with their many years of experience. Select your product type to learn more about our services.