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Session 3 Track 2: Translatability of CNS Safety and Pharmacology
Session Chair(s)
Aimee L. Jackson, PhD
Chief Scientific Officer
Atalanta Therapeutics, United States
Lois Freed, PhD
Director, Division of Pharmacology/Toxicology-Neuroscience (DPT-N), CDER
FDA, United States
This session will focus on current research efforts in the area of neurological diseases. The focus of the session will be improved oligonucleotide delivery to the CNS, pharmacokinetics/interspecies scaling for neurological assessment based on age and exposure levels, and safety observations following administration of oligonucleotides to the CNS.
Learning Objective : At the conclusion of this session, participants should be able to:- Describe the mechanisms and drivers for CNS uptake and pharmacokinetics
- Identify nonclinical study design considerations and translatability for nonclinical studies in the CNS
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Understand safety considerations for oligonucleotide therapeutics in the CNS
Speaker(s)
Acute Transient Neurobehavioral Changes in NHPs
Hao Chen, PhD
Ionis Pharmaceuticals, Inc., United States
Toxicologist
Rugonersen Pediatric Trial: How Did Nonclinical Studies (not) Translate?
Katharine Bray-French, PhD, MS
F. Hoffmann La-Roche, Switzerland
Toxicology Project Lead / Distinguished Scientist
Safety and Biodistribution of both IT/ICV and Conjugated IV ASOs and siRNAs targeting CNS indications
Kendall Frazier, DVM, PhD
Retired, United States