Wednesday, May 18, 2022 Daily Archives

Regulatory Testing for Cell and Gene Therapies: Meeting the Challenges

Compared with small molecules and other biopharmaceuticals, advanced therapies typically have more complex mechanisms of action and therefore more complex testing requirements. These requirements also change over time as innovators, testing organizations, and regulators apply recent learnings to current projects. When developing a novel cell or gene therapy it is therefore crucial to have a robust, flexible plan for testing your drug. This plan must be sufficient for proving that your drug meets safety standards and other benchmarks, and ultimately,…

The Evolution of Predictive Toxicology: Improving Predictivity Using New-Approach Methodologies

A pharmaceutical’s approval for commercial distribution is contingent on submission of pharmacological and toxicological safety data as defined by regulatory agencies such as the US FDA and ICH. Guidances state that such data can come from in vivo or in vitro studies. The current paradigm works well for collecting critical data about, e.g., a drug’s pharmacological effects and mechanism of action (MoA). However, increasing evidence points to current methods’ inadequacy for predicting a drug’s risks to human patients. Such limitations…

Accelerating Cell Line Development with an Efficient, Combined Platform Approach

The determination of product critical quality attributes (CQAs), key process parameters and indicators (KPPs and KPIs respectively) is a critical step for process efficiency during cell line development. Thousands of clones are typically screened with the goal of finding those that are stable, produce high yields of the bioproduct and exhibit desired CQAs. Screening and process optimization activities are typically carried out at the small scale in bioreactor cultures and are aimed at ensuring that results translate to larger bioreactor…

Selecting the Best Transfection Method – When to Use Transfection Reagents, Viral Transduction or Electroporation

No single delivery method is ideal for all situations, but researchers may routinely employ a suboptimal approach for the sake of familiarity or to avoid any start-up costs associated with new methods. In this white paper, we will describe three methods (chemical, electroporation and viral transduction) and highlight the Mirus Bio TransIT® transfection reagents and Ingenio® EZporator® Electroporation System, which are both easy to use and cost-effective.

Cell and gene therapy processing: Iteration or innovation?

Cell and gene therapies represent the “next evolutionary step” in bioprocessing incorporating disruptive technologies on a well-proven manufacturing base, agree panelists at BPI Europe. “Is is cell and gene therapy really the shiny, bright object on the field that breaks all the rules and disrupts everything? Or is it just an iteration of the same principles that we had before, with slightly different practices?” That was the question Nadine Ritter, president and analytical advisor at Global Biotech Experts posed to…