Session Details

Workshop 7 - Regulatory Updates – What You Need to Know When Filing Novel IND’s
Saturday, September 14, 2019 03:45 PM - 05:15 PM 
Ballroom A
This session will address some of the unique questions that come up when planning for a new trial and or IND filing in which some of the latest novel of cell and gene therapy technologies, such as CRISPR are being used. Hear from your colleagues and regulators how to best seek out information and guidance prior to filing.


Olive Sturtevant, MHP, MT(ASCP)SBB, Dana-Farber CMCF, USA


Regulatory Framework for Gene Therapies Incorporating Human Genome Editing - A CBER Perspective
Jessica Chery, PhD, Food and Drug Administration (FDA), USA
Learning objectives:
•      Overview of CBER's approach to regulation of gene therapies
•      Considerations in developing human genome editing products
•      CMC concerns for human genome editing/gene therapy products

Considerations when pursuing an iPSC-based clinical product
Amanda Mack, PhD, Fujifilm Cellular Dynamics, Inc, USA
Learning objectives:
The use of induced pluripotent stem cells (iPSC) for clinical product development represents a novel approach quickly on the rise. New questions have been encountered while preparing for IND submissions using iPSCs. These have reinvigorated discussions amongst iPSC communities aimed at establishing global standards to better enable comparisons across clinical results. The aim of this presentation is to share key highlights to consider when building an iPSC-based clinical product.

Synthesizing and Editing a CRISPR IND for Submission and Acceptance
Whitney Gladney, PhD, University of Pennsylvania, USA
Learning objectives:
•      Understand the nuances of an IND application for a novel T cell product with multiple modalities of gene editing, including CRISPR/Cas9.
•      Identify challenges associated with CRISPR-edited products in early-phase clinical trials.
•      Appreciate the value that experience with other products in your program has in facilitating a successful IND submission.

Olive Sturtevant MHP, MT(ASCP)SBB
Senior Director
Dana-Farber CMCF

Jessica Chery PhD
Gene & Cell Therapy Reviewer

Whitney Gladney PhD
Associate Director, Translational Sciences
University of Pennsylvania

Amanda Mack PhD
Director iPSC Technologies
FUJIFILM Cellular Dynamics, Inc