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Session 3 Track 2: Translatability of CNS Safety and Pharmacology
Session Chair(s)
Aimee L. Jackson, PhD
, Miragen 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)
Hao Chen, PhD
Toxicologist, Ionis Pharmaceuticals, Inc., United States
Acute Transient Neurobehavioral Changes in NHPs
Katharine Bray-French, PhD, MS
Toxicology Project Lead / Distinguished Scientist, F. Hoffmann La-Roche, Switzerland
Rugonersen Pediatric Trial: How Did Nonclinical Studies (not) Translate?
Kendall Frazier, DVM, PhD
, Retired, United States
Safety and Biodistribution of both IT/ICV and Conjugated IV ASOs and siRNAs targeting CNS indications