Session Chairs: Marcel Hoefnagel, Medicines Evaluation Board, Kevin Okimura, Orca Bio, and Cynthia Riggins, ElevateBio, LLC

Cell and gene therapies hold immense potential to transform clinical outcomes for patients with previously untreatable conditions. However, the individualized nature of process and analytical development introduces significant challenges, including increased time, resource demands, and complexity. Leveraging platform technologies offers a promising pathway to address these barriers, enabling broader clinical realization of these therapies. As demonstrated by last year’s draft guidance from FDA on the platform technology designation program, health authorities are working to provide a framework for advancing programs via use of platforms. However, maturation of the understanding and experience of platform development in action both within the US and worldwide would provide benefit.

This plenary session will delve into the evolving landscape of platform development, highlighting strategies and real-world case studies that illustrate its application in process and analytical development. Join us to explore how these innovations can drive efficiency and scalability in the field of cell and gene therapy.

Session Speakers:

Considerations in Development, Characterization, and Commercialization of Platform Processes

Stephen Madaras, Miltenyi Biotec

Accelerating Access to Life-Saving Cell Therapies

John Tomtishen, Cellares

Beyond “One Disease at a Time” : Genetic Therapy Platforms for Rare Monogenic Disease

PJ Brooks, National Center for Advancing Translational Sciences (NCATS), NIH

Additional Panelists:

Andreea Barbu, Swedish Medical Products Agency

Kathleen Francissen, Genentech, a Member of the Roche Group

Kevin Okimura

KObio Consulting, Inc.

Appearances