Strategies for the Analytical Development of CRISPR-Based Therapies in Rare & Ultra-Rare Diseases
Time: 1:30 pm
day: Pre-Conference Day
Details:
This workshop will explore the unique analytical development challenges and strategic considerations involved in advancing CRISPR-based therapies for rare and ultra-rare diseases. Using the recent case of the first-ever personalized CRISPR medicine as a foundation, the session will examine how analytical teams can respond to accelerated timelines, limited patient populations, and evolving regulatory expectations.
Attendees will discuss how to design flexible yet robust analytical packages that can support rapid IND submissions, even when working with bespoke or n-of-1 therapies. Topics will include assay development under compressed timelines, balancing scientific rigor with clinical urgency, and navigating regulatory pathways for ultra-rare indications.
