9:00 am Registration & Networking
9:30 am – 12:30 pm | Workshop A
Regulatory Readiness for the Analytical Development of CRISPR-Edited Products
Synopsis
This workshop will delve into the regulatory landscape for CRISPR-based therapies, highlighting new trials, case studies, and lessons from IND submissions. Participants will gain valuable insights into FDA guidelines, quality assurance practices, and regulatory requirements for both in vivo and in vitro CRISPR-based therapeutics.
Discussions will focus on maintaining regulatory consistency, anticipating changes, and ensuring data packages meet regulatory standards. At the end of the workshop, attendees will understand the strategies needed to advance CRISPR-based therapies from development to commercial approval, ensuring compliance and accelerating market entry
12:30 pm Break
1:00 pm – 3:30 pm | Workshop B
Strategies for the Analytical Development of CRISPR-Based Therapies in Rare & Ultra-Rare Diseases
Synopsis
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.