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Friday, June 18 • 12:00pm - 1:00pm
#521 OD: Continuing the Conversation: What’s New in Gene Therapy and Gene Editing

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Component Type: Session
Level: Intermediate

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 Objectives

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.

Chair

Philip (P.J.) Brooks, PhD

Speaker

Nonclinical Safety Evaluation Strategies Enabling IND and Marketing for Gene Therapy Products
Zeck Huang, MD, PhD

Immunogenicity Issues Related to CRISPR Cas-Mediated Gene Editing
Zuben Erach Sauna, PhD, MS



Speakers
avatar for Zuben Sauna

Zuben Sauna

Principal Investigator, OTAT, CBER, FDA, United States
Zuben E. Sauna is a Principal Investigator and also a CMC Reviewer at the US Food and Drug Administration. His research interests lie in understanding the pharmacogenetic basis of the immune response to proteins used in therapeutic interventions as these affect efficacy and safety... Read More →
avatar for Philip (P.J.) Brooks

Philip (P.J.) Brooks

Program Director, Office of Rare Diseases Research, National Center for Advancing Translational Sciences (NCATS), NIH, United States
Philip John (P.J.) Brooks is a Program Director in the NCATS Office of Rare Diseases Research. He is also the Working Group Coordinator for the NIH Common Fund program on Somatic Cell Genome Editing. He earned his Ph.D. in neurobiology from the University of North Carolina at Chapel... Read More →
avatar for Zeck Huang

Zeck Huang

Director, Topalliance Bioscience, United States
Zeck has over 20 years experience in drug discovery and development, especially in the therapeutic areas of oncology, CNS, autoimmune diseases (RA, SLE, MS, etc), infectious diseases (COVID-19, HIV, HBV, HVC, etc). He provided regulatory support for Cell and Gene therapeutic products... Read More →


Friday June 18, 2021 12:00pm - 1:00pm EDT
TBD Virtual Event Horsham, PA 19044
  06: PreClin Dev-EarlyPhaseCR, Session |   20: Early Access Content, Session