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Session 3 Track A: Oligonucleotide Conjugate Approaches in the Clinic
Session Chair(s)
Saraswathy V. Nochur, PhD, MSc
Chief Regulatory Officer
Alnylam Pharmaceuticals, United States
Jessica Hawes, PhD
Deputy Director
FDA, United States
Conjugation of oligonucleotides with various ligands provides opportunities for targeted delivery of these molecules to specific tissues. Conjugate approaches with siRNAs and antisense oligonucleotides will be discussed and clinical data will be showcased.
Learning Objective : At the conclusion of this session, participants should be able to:- Understand various chemistries and conjugate ligand approaches for delivery of oligonucleotides
- Realize the clinical data including pharmacological effect and safety in the clinic to date
- Understand the potential for this technology to target specific diseases
Speaker(s)
Erin Morgan
Ionis Pharmaceuticals, Inc, United States
Executive Director, Clinical Development
Clinical Experience with Lumasiran, an Investigational RNAi Therapeutic for the Treatment of Adult and Pediatric Patients with Primatry Hyperoxaluria 1
Tracy McGregor, MD
Alnylam Pharmaceuticals, Inc., United States
Director, Clinical Development
The Emerging Safety and Activity of TRiM™ Platform Based siRNA Drugs
James Hamilton Hamilton, MD, MBA
Arrowhead Pharmaceuticals, United States
Senior Vice President, Discovery and Transitional Medicine
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