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Tuesday, June 26 • 8:00am - 9:00am
#207: Novel Approaches for Accessing the CNS: Nonclinical and Clinical Challenges

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Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-18-560-L04-P; CME 1.00; IACET 1.00; RN 1.00

This session will highlight new approaches in CNS-targeted therapeutics. These approaches to identify and develop therapies able to reach the CNS and address specific genetic perturbations are being pioneered in rare disease, and require disciplined approaches to study designs and execution.

Learning Objectives

Design studies for advanced treatment modalities, with focus on CNS therapeutics. Identify and address hurdles in pre-clinical evaluation, identification of appropriate patients, and administration of treatments crossing or bypassing the blood brain barrier: Describe target engagement and biologic impact.


Richard Scheyer, MD


Nonclinical Models Supporting Orphan Drug Designations in Rare Neurodegenerative Conditions
Dinah Duarte

Novel Central Nervous System Delivery Methods in the Era of Targeted Therapeutics
William Elmquist, PharmD, PhD

Novel Approaches to Confirming CNS Penetration and Target Engagement
Richard Scheyer, MD

avatar for Dinah Duarte

Dinah Duarte

Scientific Evaluation Unit, Directorate of Medicinal Products, INFARMED, Portugal
Dr. Dinah Duarte is a senior assessor at the Scientific Evaluation Unit at the Directorate of Medicinal Products, in the Portuguese regulatory authority for medicines and health products (INFARMED). She is an expert member at the European Medicines Agency (EMA); the current Committee... Read More →
avatar for William Elmquist

William Elmquist

Director, Brain Barriers Research; Distinguished Prof, Dept. of Pharmaceutics, Univerity of Minnesota
His research has studied the influence of active efflux transporters in the blood-brain barrier (BBB) on CNS drug distribution. An important project currently underway is examining the determinants of anticancer drug permeability in the blood-brain barrier to improve the treatment... Read More →
avatar for Richard Scheyer

Richard Scheyer

Vice President, Medical, Medpace
Dr. Richard Scheyer is VP Medical at Medpace. Prior to Medpace, he led Experimental Medicine, BM, and PGx functions at Daiichi Sankyo. He served in leadership roles at Sanofi-Aventis and CMO at Neurotrope Bioscience. Dr. Scheyer received his BS Physics from Stanford, MD from SUNY... Read More →

Tuesday June 26, 2018 8:00am - 9:00am EDT
Room 156ABC Boston Convention and Exhibition Center 415 Summer Street, Boston, MA 02210 USA
  06: PreClin Dev-EarlyPhaseCR, Session