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Session 6 Track 2: Mind Over Matter: Cracking the Code of CNS Delivery
Session Chair(s)
Aimee L. Jackson, PhD
CXO, Curie Bio, United States
Chun-Ting (David) Lee, PhD
Pharmacology/Toxicology Reviewer, Office of New Drugs, CDER, FDA, United States
Considerations for the distribution and safety of oligonucleotide therapeutics for CNS applications.
Roshni Ramachandran, Denali Therapeutics
Hao Chen, Ionis Pharmaceuticals
An Atlas of Human Pathogenic Variants for Splice-Switching Oligonucleotide Therapy
Kim Jinkuk, KAIST (Korea Advanced Institute of Science and Technology)
Learning Objective : - Compare the distribution and safety profiles of oligos delivered systemically or directly into the CSF
- Identify the opportunities and challenges of different approaches to delivering oligonucleotides to the brain; when does systemic administration for BBB transit make sense and when is local administration the better choice?
- Apply stability, PK/PD, and safety information to identify the optimal approaches to targeting brain diseases
Speaker(s)
Safety Considerations for TfR-Mediated Oligonucleotide Delivery
Roshni Ramachandran, PhD
Director, Toxicology, Denali Therapeutics, United States
Acute Neuronal Inhibition Response from Intrathecally Administered Oligonucleotides
Hao Chen, PhD
Toxicologist, Ionis Pharmaceuticals, Inc., United States
An Atlas of Human Pathogenic Variants for Splice-Switching Oligonucleotide Therapy
Jinkuk Kim, PhD
Associate Professor, KAIST (Korea Advanced Institute of Science and Technology), Korea, Republic of