Course Description:
The success of autologous CAR-T therapies such as Kymriah® and YESCARTA® marked a turning point in cell and gene therapy (CGT). These breakthroughs showcase decades of scientific discovery and technological innovation, translating from research concepts into real therapeutic options. However, despite the excitement and investment following these approvals, the “manufacturing problem” has emerged as a critical bottleneck to broader commercial success for this class of highly personalized medicines. This webinar will explore CMC strategies in general, and control strategies in particular, as essential tools to achieving manufacturing success with confidence.
Discussion will include:
- Developing a control strategy tailored to a specific CGT modality
- Addressing challenges with raw materials and analytical methods
- Linking control strategy with Quality Management Systems (QMS)
Together, these approaches support compliance with cGMP and regulatory expectations, while enhancing the value of CGT assets across all stages of development.Key Topics:
Manufacturing Challenges Unique to CGT: Why CGT manufacturing brings added complexity and how to navigate those challenges.
Finding Commonality in High Variability: Applying Quality by Design (QbD) principles to define an early control strategy and evolve it into a formal approach suitable for BLA submissions. Guidance on avoiding common pitfalls.
Integrating Automation into Control Strategy: Automation technologies are increasingly reducing manual manipulations in CGT manufacturing. Learn how to adapt control strategies to automation while maintaining compliance and oversight.
Analytical Challenges and Emerging Solutions: Review of evolving regulatory guidance, analytical standards, and new tools that strengthen analytical components of CGT control strategies.
Connecting QMS with Control Strategy: Think of QMS as the infrastructure and control strategy as the map—together they increase the probability of product success.
Who should participate:
Pharmaceutical manufacturing, biomanufacturing, biologics organizations; Job function: process design, quality assurance (QA), quality control (QC), operations managers, quality management, pharmaceutical quality directors, MHA, MPH
Though this course is being made available on USP’s
Education site, the course content was developed by Pharmatech Associates, a
USP company. USP has not independently reviewed or verified the accuracy
of the course content.