The landscape of therapeutic innovation continues to expand with groundbreaking modalities that redefine the boundaries of modern medicine. This session will explore the evolution of emerging therapeutic technologies and the enabling role of CMC and regulatory science as critical partners to advance innovation. A case study on the development and clinical application of customized CRISPR-based gene editing for the KJ Baby, treated at Children's Hospital of Philadelphia, will be featured, followed by a panel discussion with leaders from industry, academia, and regulatory agencies to foster dialogue on accelerating the safe and efficient translation of emerging modalities from bench to bedside. Attendees will examine how novel regulatory review processes and collaborative approaches can overcome manufacturing, characterization, and safety challenges. These innovative frameworks not only enable current therapeutic breakthroughs but also serve as models for accelerated development pathways, setting the stage for the next wave of transformative treatments.

Session Speakers:

Risk-Benefit Appropriate Platform Manufacturing for Personalized Gene Editing

Sadik Kassim, DH Life Sciences, LLC

Developing and Deploying Personalized Gene-Editing Therapies

Kiran Musunuru, University of Pennsylvania

Additional Panelists:

Paula Russell, Health Canada

Brian Stultz, CBER, FDA

Heidi Zhang, Tune Therapeutics

Tyler Goodwin

Appearances