Advanced Therapies: Innovations in CMC Conference

Solutions for CMC challenges in the development and commercialization of cell therapy, gene therapy, and gene editing products.

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Session 5b: Case Studies on Challenges for Gene Editing Technology

Session Chair(s)

Eliana Clark, PHD

Senior Vice President Technical Operations and Quality, Intellia Therapeutics, Inc., United States

Kirsten Messmer, PHD, RAC

Owner, KM Intel LLC, United States

Genome edited products have recently entered the clinical development phase. These products face very specific CMC challenges due to the technology employed, e.g. off-target editing, translocation. This session will discuss case studies for 2-3 different gene edited products.

Learning Objective : At the conclusion of this session, participants should be able to:
Understand genome edited product specific CMC challenges during product development

Determine approaches to address the challenges to ensure regulatory compliance

Form strategies supporting development programs using genome editing technology

Speaker(s)

Kristy Wood, PHD

Vice President Nucleic Acid Therapeutics Development & Manufacturing, Intellia Therapeutics, United States

Speaker

Paul Kopesky, PHD

Senior Director, Process Development, Beam Therapeutics, United States