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Capital Hilton

28 oct 2024 8:30 a.m. - 30 oct 2024 12:40 p.m.

1001 16th Street NW, Washington, DC 20036-5794

DIA/FDA Oligonucleotide-Based Therapeutics Conference

Session 3 Track 2: Translatability of CNS Safety and Pharmacology

Session Chair(s)

Aimee L. Jackson, PhD

Aimee L. Jackson, PhD

Chief Scientific Officer

Atalanta Therapeutics, United States

Lois  Freed, PhD

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
  • Understand safety considerations for oligonucleotide therapeutics in the CNS

Speaker(s)

Hao  Chen, PhD

Acute Transient Neurobehavioral Changes in NHPs

Hao Chen, PhD

Ionis Pharmaceuticals, Inc., United States

Toxicologist

Katharine  Bray-French, PhD, MS

Rugonersen Pediatric Trial: How Did Nonclinical Studies (not) Translate?

Katharine Bray-French, PhD, MS

F. Hoffmann La-Roche, Switzerland

Toxicology Project Lead / Distinguished Scientist

Kendall  Frazier, DVM, PhD

Safety and Biodistribution of both IT/ICV and Conjugated IV ASOs and siRNAs targeting CNS indications

Kendall Frazier, DVM, PhD

Retired, United States

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