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DIA 2021 Global Annual Meeting


Continuing the Conversation: What’s New in Gene Therapy and Gene Editing

Session Chair(s)

Philip (P.J.)  Brooks, PhD

Philip (P.J.) Brooks, PhD

  • Program Director, Office of Rare Diseases Research
  • National Center for Advancing Translational Sciences (NCATS), NIH, United States
This session will cover specific considerations for the nonclinical evaluation of GTP from a US regulatory perspective. In addition, we will present specific case studies to demonstrate the unique variability in nonclinical testing strategies for GTP.
Learning Objective : Describe the nonclinical safety requirements for gene therapy products (GTP)(oncolytic virus, CRISPR/Cas9, therapeutic vaccine); Discuss specific considerations for the nonclinical evaluation of GTP from a US regulatory perspective; Identify specific case studies to demonstrate the unique variability in nonclinical testing strategies for unique GTP.

Speaker(s)

Zeck  Huang, MD, PhD

Nonclinical Safety Evaluation Strategies Enabling IND and Marketing for Gene Therapy Products

Zeck Huang, MD, PhD

  • Director
  • Topalliance Bioscience, United States
Zuben Erach Sauna, PhD, MS

Immunogenicity Issues Related to CRISPR Cas-Mediated Gene Editing

Zuben Erach Sauna, PhD, MS

  • Principal Investigator, OTAT, CBER
  • FDA, United States