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Session 8: Gene Editing: Driving CRISPR to the Clinic in Different Vehicles
Session Chair(s)
Gerald Cox, MD, PhD
Chief Medical Officer
Editas Medicine, Inc., United States
CRISPR is a powerful new technology whose genome editing versatility is being harnessed to usher in a new class of potential genetic therapies. While offering the promise of “one and done” treatments, human genome-based therapies also pose unique challenges for drug development. This session will examine two examples of early stage CRISPR-based medicines being developed to treat rare genetic diseases of the liver using systemic lipid nanoparticles and of the eye using subretinal delivery of AAV5. Preclinical evaluation of proof of concept, delivery, and specificity will be presented. As the CRISPR field evolves, so do the regulatory considerations regarding patient safety and risk-benefit. FDA’s perspective and insights on preclinical data needed to support the transition of CRISPR-based medicines into the clinic will be discussed.
Learning Objective : At the conclusion of this session, participants should be able to:
- Identify the unique preclinical challenges of developing CRISPR-based medicines
- Understand the regulatory expectations for the preclinical evaluation of gene editing-based products
Speaker(s)
Session Co-Chair
Iwen Wu, PhD
FDA, United States
Director, Office of Pharmacology and Toxicology Cell and Gene Therapies CBER
Robust In Vivo Gene Editing with Systemic Lipid Nanoparticle Delivery of CRISPR/Cas9 RNA Components
Amy Rhoden Smith, PhD
Intellia, United States
Principal Scientist
Preclinical Development of AAV5 Encoding CRISPR/SaCas9 for the Treatment of Infantile Blindness Caused by Leber Congenital Amaurosis Type 10
Gerald Cox, MD, PhD
Editas Medicine, Inc., United States
Chief Medical Officer
Preclinical Considerations for Gene Therapy Products Involving Gene Editing Technology: An FDA Perspective
Ying Huang, PhD
FDA, United States
Pharmacologist, Office of Cellular, Tissue and Gene Therapies, CBER
Panel Discussion
All Session Speakers, United States
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