Ask the Experts

Improving Gene Therapy Viral Vector Manufacturing Economics: Modelling Bioprocess Costs

Gene therapy viral vector manufacturers strive to reduce time to market and cost per dose to ensure treatment affordability and patient access. Approved and commercialized AAV-based gene therapies Luxturna® and Zolgensma®, for patients suffering from vision loss and spinal muscular atrophy, are the first of many gene therapies that are expected to reach the market in the coming years. One major bottleneck is the cost per dose of the treatments, with Luxturna® costing almost half a million dollars per eye…

Stability Assessment of mRNA Formulations – Toward the Development of Degradation Assays for mRNA-based Vaccines

This webcast features: Caio Henrique Barros, PhD, Research Scientist, National Institute for Bioprocessing Research and Training & James Geiger, PhD, Field Application Scientist, PerkinElmer. The COVID-19 pandemic has demonstrated the potential of mRNA vaccines to protect against infectious diseases. These unprecedented circumstances have also highlighted the instability of the mRNA molecule, which required the first COVID vaccines to be distributed at ultracold temperatures. This limitation has intensified efforts to develop mRNA formulations that are sufficiently stable at room temperature. Dried…

Using Monolith-Based Chromatography to Monitor In Vitro Transcription Reactions

In vitro transcription (IVT) is a critical step in messenger RNA (mRNA) production. In a March 2022 webinar, Rok Sekirnik (head of process development for mRNA and plasmid DNA (pDNA) applications at BIA Separations, part of Sartorius) explained that optimizing concentrations of reagents used during IVT helps to maximize the amount of mRNA that is produced. Drug developers have great need for analytical methods that can measure IVT in real time. Sekirnik showed how CIMac PrimaS monolith chromatography columns enable…

Ask the Expert: Using High-Throughput Analytical Systems to Facilitate Binding-Kinetics Assays

Critical quality attributes (CQAs) must be characterized thoroughly to establish a biopharmaceutical’s potency, efficacy, and safety. Such characterization involves some of the most difficult analytical activities that are performed in a biologic’s product life cycle. There is great need for analytical tools that can facilitate such studies. In March 2022, Dr. Kalhari Silva (head of scientific research at Custom Biologics) and Dr. Bob Dass (senior scientist at Sartorius) joined BPI to describe how Octet biolayer interferometry (BLI) technology can be…

High Performance With GMP Ready CHT Prepacked Process-Scale Columns

This webcast features: Khaled S. Mriziq, PhD, Senior Marketing Manager, Process Chromatography, Bio-Rad Laboratories. In this webinar, we will present performance data for the newly launched, GMP ready CHT™ Ceramic Hydroxyapatite prepacked process-scale columns. This will include HETP and asymmetry data that shows excellent column performance and shipping stability over a range of column diameters. The columns are designed to eliminate any headspace formation during transportation. Cycling and pressure data will also be presented. Foresight Pro Columns can be used…

Bridging Anti-CHO HCP Antibodies From Two Third Generation Cygnus CHO HCP ELISA Kits by AAE-MS™

This webcast features: Eric Bishop, Vice President, Research & Development, Cygnus Technologies. A well-developed, broadly reactive, and qualified HCP ELISA remains a gold standard method effectively used during the purification process to ensure removal of HCP and to demonstrate process consistency and final drug substance purity. Regulatory guidelines require that sponsors use orthogonal methods for demonstrating antibody coverage to individual HCPs and provide a comprehensive assay qualification package to ensure the HCP ELISA is fit for purpose. In this presentation,…

Transform Your AAV Analytics With Mass Photometry

This webcast features: Gareth Rogers, Product Manager, Refeyn, and Kirsty McManus, Senior Scientist, Characterization Team, Pharmaron Biologics. Mass photometry is a novel, easy-to-use bioanalytical technology that measures the empty-full AAV capsid ratio in minutes using minimal sample amounts and without the need for sample preparation. Circumventing the requirement of large capital expense and skilled operators, it can be employed in different AAV workflows. In this webinar, we present a novel mass photometry instrument called SamuxMP, which is dedicated to the…

Leveraging NISTmAb Prior Knowledge to Demonstrate Analytical Competency

The NISTmAb IgG1κ monoclonal antibody reference material, from the National Institute of Standards and Technology, is intended for use in evaluating the performance of analytical methods that determine physicochemical attributes of biologics. As a case study, Almac Sciences have assessed NISTmAb and its prior knowledge to demonstrate competency in chromatography and capillary electrophoresis methods. Key Takeaways: Analytical challenges during establishment of NISTmAb methods. Comparability assessment of NISTmAb data to demonstrate analytical competency. Transition of capillary electrophoresis methods from 32Karat software…

Moving Beyond PEI: Improved Transfection Processes for AAV and LV Manufacture

GMP-compliant nucleic acid delivery to HEK 293 cells is often a critical first step in the manufacture of advanced therapies, utilizing recombinant adeno-associated virus (AAV) or lentivirus (LV) to facilitate delivery of a therapeutic transgene to patients. Accordingly, the need for safe and reproducible large-scale viral vector manufacture processes has never been greater. Mirus Bio has developed a fully synthetic, innovative transfection formulation to enable higher titer AAV and LV generation and reduce the cost of therapeutic development and manufacture…

Ask the Expert: Analytical Tools to Accelerate Cell-Line Development Workflows

Cell-line development (CLD) involves screening thousands of clones to identify the most stable and productive candidate for an upstream manufacturing process (Figure 1). Such assessment requires considerable time and resources for preparation and execution of multiple cultivations and analytical assays. In March 2022, Lukas Klein and Dirk Müller (scientist and manager of media and process development, respectively, at Sartorius) delivered a BPI Ask the Expert webinar about integrating their company’s Ambr 15 Cell Culture bioreactor and Octet label-free biolayer interferometry…