The advanced therapies field is expected to grow at an impressive pace even with the recent global macroeconomic and COVID-19 pandemic setbacks. Despite the large pipeline of cell and gene therapy discovery research programs entering preclinical and clinical development, drug developers continue to encounter significant manufacturing and regulatory hurdles in moving from proof-of-concept research to conducting first-in-human clinical trials. The crux of these challenges is often tied to manufacturing unpreparedness associated with higher-quality raw and starting materials, controlled manufacturing processes, and qualifiable analytical assays for generating and characterizing research materials and subsequent clinical products.
Drug developers can accelerate clinical development and shorten timelines by putting more emphasis upfront at the discovery stage by incorporating a scalable manufacturing process, utilizing robust analytics, and accounting for regulatory requirements and cleanroom compatibility rather than delaying decisions to the Investigational New Drug (IND) application stage. By implementing these principles early on, drug developers can ensure consistent and high-quality data generation at the research stage and gain better predictability to cGMP (current good manufacturing practices) manufacturing, while also reducing the time and resources involved in conducting comparability studies in the transition from research to IND enablement.
In 2022, Thermo Fisher Scientific launched Patheon™ Translational Services to serve advanced therapy drug developers with lead identification and process establishment. Housed in a new, 32,000-square-foot facility in San Diego, Translational Services has dedicated space for molecular biology, viral vector manufacturing and cell therapy services, as well as cell therapy research and development (R&D) area dedicated to developing cGMP cell processing innovations. Offering industry-leading, state-of-the-art instruments for production, analytics, and R&D, Translational Services supports clinically bound drug developers by providing access to an interdisciplinary team of scientists, bioprocessing engineers, and regulatory and quality assurance experts with over 30 years of cell and gene therapy experience.
Current capabilities include:
· Cell therapy services
o Process evaluation (viral and non-viral modification, allogeneic, autologous)
o Raw material compatibility and performance assessment
o Analytics assay establishment
· Viral vector services
o Up to 10 L of lentivirus (LV) or adeno-associated virus (AAV) production using the scaled-down cGMP Patheon Quick to Clinic™ viral vector manufacturing process
o Modern analytical assays
· Molecular biology services
o Plasmid design that incorporates regulatory requirements
o Cloning (e.g., de novo synthesis, antibiotic resistance gene swapping)
o Access to chemistry, manufacturing, and control (CMC)–compatible plasmids
o Small-scale (up to 4 mg) plasmid expansion
o Plasmid linearization
The Translational Services materials and processes are designed to seamlessly transfer into the cGMP environment.
The journey from designing advanced therapy manufacturing materials for clinical studies to bringing these therapies to patients is costly, time-sensitive, and filled with numerous risks. Partnering with an experienced service provider, such as Patheon Translational Services, at the discovery phase can derisk and expedite the drug development process.