USP Biologics Stakeholder Forum 2022: Collaborating to solve CMC (Chemistry Manufacturing and Controls) challenges and support efficient development of lentiviral-mediated CAR T cell therapies

USP Biologistics Stakeholder Forum 2022: Collaborating to solve CMC challenges

 Agenda | Speaker Biographies | Event Presentations | Event Summary

We explored the industry's most pressing challenges at the intersection of biotherapeutic quality and innovation, each session of this forum focused on a single area of biotherapeutic quality that could be advanced through collaborations with USP – an independent scientific organization that develops quality standards used globally by industry.

We engaged with industry leaders, regulators, and USP to help shape future quality standards to support development and characterization of genetically modified cell-based therapies like chimeric antigen receptor (CAR) T cell therapies. Although multiple therapies in this class have been approved and a recent draft guidance proposed by the Food and Drug Administration (FDA), there are still many challenges that hinder development of these products, including:

  • donor cell apheresis variability
  • production and characterization of the viral vector used to deliver the appropriate CAR
  • development of suitable potency assays
  • ensuring sterility of the modified cells prior to delivery to the patient

The forum started at 9:15 a.m. EDT with talks by subject matter experts from FDA, industry, and USP in a hybrid meeting format (in person and virtual). These talks included case studies that focused on chemistry, manufacturing, and controls aspects that are often challenging.

For those who attended in person, the day included lunch, networking time, and moderated breakout sessions to discuss issues and challenges along with making recommendations for USP’s future work to advance the development and licensure of this important therapeutic class.

Questions? Please contact Maura Kibbey to learn more.

Event Presentations

  1. Introduction and Objectives for today's Biologics Stakeholder Forum, Edward Chess, Ph.D., USP Biologics Stakeholder Forum Planning Committee, Chair
  2. USP standards to support the development of cell and gene therapy products, Ben Clarke, Ph.D. USP
  3. Regulatory Considerations for the Development of Potency Assays during CAR T Cell Development, Andrew Timmons, Ph.D., U.S. Food and Drug Administration
  4. Approaches to Potency Testing for Chimeric Antigen Receptor T Cells, Shree Joshi, Janssen Pharmaceutical
  5. Implementation of AQbD principles in potency assay development and overcoming challenges on the road to commercialization, Kim Nguyen, Ph.D., Kite Pharma