Specification setting for Advanced Therapy Medicinal Products (ATMPs) is built on the principles of ICH Q6B: Specifications for Biotechnological/Biological Products. The challenge for ATMP developers is the integration of regulatory principles, scientific understanding, pre-defined specifications by regulators, and platform experience for a wide variety of therapeutic ATMP modalities. As ATMP modalities become increasingly complex and diverse, sponsors face growing difficulty defining specifications that are development-stage appropriate, and adaptable across ATMP modalities and platforms.

This session will begin with an introduction to the fundamentals of specification setting for ATMPs. Speakers will discuss how traditional concepts such as critical quality attributes, acceptance criteria, and control strategies can be applied—and appropriately adapted—to advanced modalities. The discussion will then examine practical approaches to building specifications across three major advanced modality categories—cell therapies, viral vector-based gene therapies, and nucleic acid-based products—highlighting both commonalities and modality-specific challenges.

The session will then explore how platform and prior knowledge can be effectively leveraged to inform specification development. Drawing on experience from existing products and related platforms, strategies for extrapolating quality attributes, setting initial acceptance criteria, and managing uncertainty while maintaining regulatory flexibility will be explored.

Finally, the session will address decision-making around differentiating release methods (with defined acceptance criteria) from characterization methods. Speakers will discuss how to determine which quality attributes must be controlled at release versus those better suited for characterization, platform understanding, or lifecycle management, and how this distinction evolves across development stages. Data and evaluation criteria needed to support these decisions will be discussed, including assay performance, attribute relevance, risk assessment, and links to clinical and non-clinical outcomes. The importance of cross-functional collaboration—between clinical, non-clinical, and analytical stakeholders—will be emphasized as a key enabler of robust and defensible specification strategies.

Session Speakers:

Regulatory Perspectives on Establishing Specifications for Cell and Gene Therapy Products: From Speculation to Specification

Graeme Price, CBER, FDA

Evergreen Specification Setting Practices

Julia O’Neil, Direxa Consulting

Lifecycle‑Aligned Specifications: Ensuring Quality and Consistency in Cell and Gene Therapy Development

Tiffany Lucas, Eliquent Life Sciences

Additional Panelist:

Christopher Storbeck, Health Canada

Sarmitha Sathiamoorthy

Appearances