24 November 2023

bioProcessUK insight: The role of candidate screening and feasibility studies

Ahead of the 20th annual bioProcessUK conference next week in Brighton, Ian Goodwin, Director of CMC Program Design in Advanced Therapies (covering Cell and Gene Therapy services) at FUJIFILM Diosynth Biotechnologies explores the benefits of candidate and feasibility studies to AAV production.

Drug developers in the adeno-associated virus (AAV) therapy space are up against several challenges and limitations as they work to deliver innovative therapeutics to patients. Perhaps most notably, limited funding that has become increasingly gated, that is, requiring programs to reach certain milestones before additional resources are unlocked. CDMOs should understand the challenges that investigators face when it comes to raising funding, and in turn, offer solutions.

Candidate screening and feasibility studies are efficient and effective mechanisms that can provide material enabling generation of clinical data to support the next round of funding. Specifically, candidate screening should narrow down the most promising AAV constructs from a manufacturing and clinical efficacy standpoint and evaluate critical quality attributes.

Candidate screening requires a minimal amount of bioprocessing, generally a shake flask or small stirred tank bioreactor’s worth. It allows screening of multiple different candidates simultaneously which are run through a common downstream process. Previously, less readily scalable methods like ultracentrifugation or precipitation followed by ultracentrifugation were commonly used. At FDB, we moved away from these to identify a more manufacturing-ready process to rapidly evaluate as many different constructs as possible.

Gain more insight with feasibility studies

Once construct design is complete and the most promising AAV constructs identified, subsequent feasibility studies include scale-up, additional material for your preclinical studies, biodistribution analysis, and dose range finding prior to toxicology design. They provide a more expansive product quality and residual panel of analytics, including genome titer, full-to-empty ratio analysis, capsid titers, residual host cell DNA, and host cell protein. These are critical to ensure process performance and scalability. As such, feasibility studies enable success criteria to be established prior to moving into process development and demonstration runs and secure additional material for preclinical studies.

Choose a modular approach to suit your needs

While there are a variety of entry points to a CDMO partnership, it is critical to design a path that accommodates the needs of your project. At FDB, we offer our clients several modular paths, that allow the project’s scope to shift with a client’s needs. Our full program encompasses candidate screening, feasibility studies, small-scale process/analytical development, toxicology material generation, cGMP, and release.

If you are curious about how candidate screening and feasibility studies could help you, view the recent webinar hosted by FDB on the impact of candidate and feasibility studies for AAV therapeutics.

About FUJIFILM Diosynth Biotechnologies

FUJIFILM Diosynth Biotechnologies is an industry-leading Biologics Contract Manufacturing Organisation with locations in Billingham, UK and Research Triangle Park, North Carolina, USA.

FUJIFILM Diosynth Biotechnologies has over 25 years’ combined experience in the development and manufacturing of recombinant proteins, vaccines, monoclonal antibodies, among other large molecules expressed in a wide array of microbial, mammalian, and insect systems. The company offers a comprehensive list of services from cell line development, including its proprietary pAVEway™ system, to process development, analytical development, clinical and commercial manufacturing. Both sites are FDA-approved for the production of commercial products.

Meet and learn more about FUJIFILM Diosynth Biotechnologies at the 20th Annual bioProcessUK conference