Regulatory Toxicology Case Studies: An Interactive Session Involving Industry, CRO, Consultant and Regulatory Perspectives (CE1)

CE Course Chair(s)

• Simon Authier, DVM, MBA, PhD, DSP

  Principal Director
  Charles River Laboratories
  Laval, Quebec, Canada

• Katie Sokolowski, PhD, DABT

  Principle Toxicologist
  Safety Assessment / Development Sciences
  Denali Therapeutics
  South San Francisco, California, United States

CE Course Speaker(s)

• Denis Roy, PhD

  Vice President
  Nonclinical Safety & Development
  SciLucent, Inc.
  San Diego, California, United States

• Armaghan Emami, PharmD, PhD

  Pharmacologist
  US FDA
  Silver Spring, Maryland, United States

• Paramita Mookherjee, PhD, DABT

  Director, Toxicology
  Drug Safety and Pharmacometrics
  Regeneron
  Seattle, Washington, United States

• Wendy Haines, PhD, DABT, ERT

  Director of Toxicology & Quality Services
  PharmEng Technology
  Durham, North Carolina, United States

Educational Co-Support Provided by: Charles River and SciLucent, Inc.

This session will present interactive case studies from pharmaceutical development across the toxicology testing continuum. The aspects that will be presented will include CMC (i.e. formulation preparation), excipients, impurities/degradants, analytical chemistry/bioanalysis stability, study designs (species, exposure, metabolites, duration, route of administration, etc.), safety margins, novel modalities (e.g. cell and gene therapy), unexpected toxicity, regulatory meetings, in the context of the targeted patient population/indication and regulatory jurisdiction. The moderators in this session include representation from pharmaceutical industry, CRO, consultants, and regulatory agency toxicologists. Moderators will interact and discuss with participants for each case study presented as the various perspectives are considered.

Presentations:

• 8:00 AM - 8:10 AM CT

  Introduction
  

  CE Course Speaker: Katie B. Sokolowski, PhD, DABT – Denali Therapeutics
• 8:10 AM - 8:50 AM CT

  Case Study Example #1: Advancing Gene Therapy: A Case Study on AAV Program Design for Central Nervous System Delivery in Orphan Indications
  

  CE Course Speaker: Simon Authier, DVM, MBA, PhD, DSP – Charles River Laboratories
• 8:50 AM - 9:30 AM CT

  Case Study Example #2: Leveraging the 505(b)(2) Pathway for an Investigational New Drug Formulation Using New Administration Modalities
  

  CE Course Speaker: Denis Roy, PhD – SciLucent, Inc.
• 9:30 AM - 10:00 AM CT

  Break
• 10:00 AM - 10:30 AM CT

  Case Study Example #3: Local Safety Margins for Intra-articular Products
  

  CE Course Speaker: Armaghan Emami, PharmD, PhD – US FDA
• 10:30 AM - 11:00 AM CT

  Case Study Example #4: Residual Solvent Toxicity in Products
  

  CE Course Speaker: Wendy Haines, PhD, DABT, ERT – PharmEng Technology
• 11:00 AM - 11:30 AM CT

  Case Study Example #5: Applying Nonclinical Data in Requesting Study Waivers for NMEs
  

  CE Course Speaker: Paramita Mookherjee, PhD, DABT – Regeneron