Challenges for Viral Vector Production for Translational Gene Therapy
In academic and/or translational laboratories, usually small amounts of virus is required. Most upstream vector production at this stage is adherent and done in small flasks and possibly scaled up slightly to cell stacks. Vector production also requires transient transfection of the adherent cell lines with plasmids which means that optimization of clones that is generally carried out during process development of mAbs is not applicable for these technologies. There can be considerable challenges with scaling up adherent cultures.
Once the cell culture is complete, the virus must then go through recovery, purification, and formulation. In many cases, the product is contained within the cell, so cell lysis needs to take place. Usually, precipitation and ultracentrifugation techniques are used to recover and purify the virus, but these are not scalable processes.
Formulation then requires getting the vector into high concentrations and this can cause both clumping and stability issues. All in all, there are many issues with lab-scale generation of viral vectors and completely new processes must be used when scaling these up.
The Benefits of Using Scalable Technologies for Viral Vector Manufacturing During the Early Stages of Development Work
Usually, the manufacturing processes during early-stage development of the gene therapy are based on non-commercially viable production platforms. Transferring and optimizing the process to a scalable manufacturing platform is therefore a necessity.
To achieve manufacturing platform advancement, the product needs to be very well-characterized during development so that investigators can generate data sets which demonstrate comparability between products used in clinical studies and those generated with the final manufacturing process.
Our downstream process technologies are available in bench-scale format for early-stage bioprocess development.
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The process-development scale consumables are designed to scale directly to larger configurations to meet the demands for cGMP clinical and manufacturing production.
Our Scientific and Laboratory Services group is the Right Partner for Your Gene Therapy Development
Scientific and Laboratory Services (SLS) is our customer-focused technical support organization. It comprises scientific and technical staff working closely on-the-bench with customers and offering support and consulting for (early phase) drug development projects.
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Sterile Filtration of AAV
The first in a short series of practical guidance notes for the selection and testing of sterilizing grade filtration used in the production of viral vectors. This video focuses on the importance of filtration and the filter formats available to scale from bench-scale testing to large scale GMP production.