Toxicologic Neuropathology of Novel Therapeutics (CE6)

CE Course Chair(s)

• Dinesh Bangari, PhD, DACVP

  Head of Discovery Pathology
  Sanofi
  Cambridge, Massachusetts, United States

• Lisa Lanigan, DVM, PhD, DACVP

  Senior Pathologist
  Charles River Laboratories

CE Course Speaker(s)

• Sarah Cramer, DVM, PhD, DACVP

  Senior Director, Medical Device Pathology and Neuropathology
  StageBio
  Frederick, Maryland, United States

• Elizabeth Galbreath, DVM, PhD, DACVP

  Founder, Principal
  Advanced Pathology Solutions
  Lexington, Massachusetts, United States

• Jessica Grieves, DVM, PhD, ACVP

  Director of Pathology
  Preclinical Development
  Ionis Pharmaceuticals
  Carlsbad, California, United States

• René Meisner, DVM, DACVP, DABT

  Vice President
  Nonclinical Development
  TCG Labs Soleil
  South San Francisco, California, United States

• Arlin Rogers, DVM, PhD, DACVP

  Senior Director
  Pathology
  Alnylam Pharmaceuticals
  Sudbury, Massachusetts, United States

• Brad Bolon, DVM, PhD

  President
  GEMpath, Inc.
  Longmont, Colorado, United States

Educational Support Provided by: Society of Toxicologic Pathology

We will present an overview of the mechanisms of action, toxicologic liabilities, and safety risk assessment approaches for novel biotherapeutics for use in treating nervous system disorders and neuromuscular diseases. These modalities include vector-based therapies such as lentiviral and adeno-associated viral vectors (AAVs), cell-based therapies such as stem cells and CAR-T cells, nucleic acid-based therapies such as antisense oligonucleotides (ASOs) and mRNAs, and novel antibody-based therapeutics. The session will begin with an overview of the mechanisms of action for each of these therapeutics, how they can specifically target the nervous system and the unique aspects of safety risk assessment for these novel biotherapeutics. Additionally, there will be a presentation on the methods for investigating biodistribution, pharmacokinetics and pharmacodynamics to enable clinical/human dose prediction of these novel therapeutics. Finally, there will be 2–4 short case studies to demonstrate the importance of an informed safety assessment for novel biotherapeutics that target the nervous system. The goal of this session is to provide a well-rounded overview of the rapidly emerging biopharmaceutical research that is focused on developing novel therapeutics for the treatment of nervous system disorders and neuromuscular diseases.

Presentations:

• 1:00 PM – 1:30 PM CT

  Vector-Based and Cell-Based Therapies
  

  CE Course Speaker: Sarah Cramer, DVM, PhD, DACVP – StageBio
• 1:30 PM – 2:00 PM CT

  Case Presentation: Routed by ROA—Effect of Route of Administration on AAV Neuropathology
  

  CE Course Speaker: Elizabeth Galbreath, DVM, PhD, DACVP – Advanced Pathology Solutions
• 2:00 PM – 2:30 PM CT

  Overview of Nucleic Acid-Based Therapies for Neurologic and Neuromuscular Diseases​
  

  CE Course Speaker: Jessica Grieves, DVM, PhD, ACVP – Ionis Pharmaceuticals
• 2:30 PM – 3:00 PM CT

  Break
• 3:00 PM – 3:30 PM CT

  Engineered Large Molecules for CNS Delivery
  

  CE Course Speaker: René Meisner, DVM, DACVP, DABT – TCG Labs Soleil
• 3:30 PM – 4:00 PM CT

  RNAi Pharmacokinetics and Activity Assessment in CNS
  

  CE Course Speaker: Arlin B. Rogers, DVM, PhD, DACVP – Alnylam Pharmaceuticals
• 4:00 PM – 4:30 PM CT

  Concept Presentation: Grappling with Gliosis in Assessing Adversity
  

  CE Course Speaker: Brad Bolon, DVM, PhD – GEMpath, Inc.