Reference materials for cellular therapeutics

Area of Science:

• Biotechnology
• Analytical Chemistry
• Regulatory Science

Background:

• Cellular therapeutics (CTP) development is a lengthy process with long-term clinical use expectations.
• Manufacturing and quality consistency rely on complex analytical methods, posing a risk of process and method drift.
• Reference materials (RM) are a scientific and regulatory necessity for CTP development.

Purpose of the Study:

• To discuss the role and necessity of RM in CTP manufacturing and quality control.
• To highlight the challenges in developing suitable RM for living cell products and associated analytical methods.
• To propose strategies for ensuring the reliability of analytical measurements in CTP development.

Main Methods:

• Review of the established scientific and regulatory principles for using RM in biopharmaceutical development.
• Discussion of the application of RM to analytical methods commonly used in CTP, such as flow cytometry.
• Exploration of the characteristics and suitability of different types of RM, including consensus and product-specific RM.

Main Results:

• Few consensus RM are currently available for common CTP analytical methods.
• Consensus RM may require application to specific standardized methods rather than broad use.
• Product-specific RM are often the most suitable solution for ensuring CTP quality, given product deviations from starting cells.

Conclusions:

• Fit-for-purpose RM are critical for ensuring the reliability of analytical measurements in CTP manufacturing.
• Standardized methods and appropriate measurement RM are key for defining starting cell types.
• CTP developers must strategically implement RM to guarantee the consistency and quality of their products.