Showing papers by "Vertex Pharmaceuticals published in 2022"

Anti-drug Antibody Validation Testing and Reporting Harmonization.

Abstract: Evolving immunogenicity assay performance expectations and a lack of harmonized anti-drug antibody validation testing and reporting tools have resulted in significant time spent by health authorities and sponsors on resolving filing queries. Following debate at the American Association of Pharmaceutical Sciences National Biotechnology Conference, a group was formed to address these gaps. Over the last 3 years, 44 members from 29 organizations (including 5 members from Europe and 10 members from FDA) discussed gaps in understanding immunogenicity assay requirements and have developed harmonization tools for use by industry scientists to facilitate filings to health authorities. Herein, this team provides testing and reporting strategies and tools for the following assessments: (1) pre-study validation cut point; (2) in-study cut points, including procedures for applying cut points to mixed populations; (3) system suitability control criteria for in-study plate acceptance; (4) assay sensitivity, including the selection of an appropriate low positive control; (5) specificity, including drug and target tolerance; (6) sample stability that reflects sample storage and handling conditions; (7) assay selectivity to matrix components, including hemolytic, lipemic, and disease state matrices; (8) domain specificity for multi-domain therapeutics; (9) and minimum required dilution and extraction-based sample processing for titer reporting.

Implementation of High Throughput Experimentation across Medicinal Chemistry, Process Chemistry and Materials Science

Abstract: High Throughput Experimentation (HTE) has revolutionized the pharmaceutical industry over the last two decades, developing from a technology for screening compounds against biological targets, to now being used for everything from parallel synthesis of diverse libraries of analogues and optimization of catalytic systems and reaction conditions, to screening for optimal crystallization solvents and API polymorphs. Many pharmaceutical companies (and several academic institutions) have invested in HTE capabilities (often including automation) to support their drug discovery pipelines and fundamental research. With HTE workflows now being firmly established and many successful HTE groups to draw inspiration from, new facilities being set up today have options on where and how to build and integrate new high throughput and automation capabilities into their organizations. This chapter discusses and classifies different options for the build and integration of high throughput experimentation resources into existing chemistry departments.