Gene Therapy for Rare Diseases: Considerations for Both Clinical and Post-Marketing Studies

Multicellular organisms and dynamic biological processes consist of a complex and coordinated interplay of multiple individual cells. Until recently, most molecular studies have relied on bulk analysis, combining all cells into a single average readout. In recent years, single-cell RNA sequencing technologies have enabled researchers to study development and disease at the level of individual cells, revealing cell-to-cell gene expression differences that were obscured with bulk or low throughput methods.

In this webinar, we will discuss how to set up and utilize a flow cytometer for nanoparticle analyses. We will first describe how to properly set up and clean a flow cytometer for small-particle experiments. Next, we will characterize the scatter and fluorescence sensitivity of the CytoFLEX. Finally, we will demonstrate how to immunophenotype extracellular vesicles (EVs) in human blood and plasma, including a discussion of common sample preparation techniques.

Since the year of 2000, there’re 13 ADCs on the market and 9 of them were approved in the last three years. The enthusiasm of pharmaceutical and biotechnology companies for the research and development of ADC has been increasing.

Attribute Sciences, Process Development, Amgen Biopharmaceuticals are drugs manufactured by growing genetically engineered cells in bioreactors to produce a therapeutic protein. Plastic single-use bioreactors are of interest to biopharmaceutical drug manufacturers due to their significant environmental and cost benefits and flexibility over stainless steel bioreactors. Effect of plastics on the biomanufacturing process is not yet completely understood. A case study on extractables from single-use bioreactors/bags and the impact on cell culture performance will be presented.