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Bethesda North Marriott Hotel and Conference Center

25 oct 2017 7:00 a.m. - 27 oct 2017 3:00 p.m.

5701 Marinelli Road, , North Bethesda, MD 20852 , USA

DIA/FDA Oligonucleotide-Based Therapeutics Conference

Convening industry and health authorities to inform, educate, and share advancements in oligonucleotide-based therapeutic product development.

Session 8: Gene Editing: Driving CRISPR to the Clinic in Different Vehicles

Session Chair(s)

Gerald  Cox, MD, PhD

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)

Iwen  Wu, PhD

Session Co-Chair

Iwen Wu, PhD

FDA, United States

Director, Office of Pharmacology and Toxicology Cell and Gene Therapies CBER

Amy  Rhoden Smith, PhD

Robust In Vivo Gene Editing with Systemic Lipid Nanoparticle Delivery of CRISPR/Cas9 RNA Components

Amy Rhoden Smith, PhD

Intellia, United States

Principal Scientist

Gerald  Cox, MD, PhD

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

Ying  Huang, PhD

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

Panel Discussion

All Session Speakers, United States

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