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DIA/FDA Oligonucleotide-Based Therapeutics Conference


Session 3 Track A: Oligonucleotide Conjugate Approaches in the Clinic

Session Chair(s)

Saraswathy V. Nochur, PhD, MSc

Saraswathy V. Nochur, PhD, MSc

  • Chief Regulatory Officer
  • Alnylam Pharmaceuticals, United States
Jessica  Hawes, PhD

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

Erin Morgan

  • Executive Director, Clinical Development
  • Ionis Pharmaceuticals, Inc, United States
Tracy  McGregor, MD

Clinical Experience with Lumasiran, an Investigational RNAi Therapeutic for the Treatment of Adult and Pediatric Patients with Primatry Hyperoxaluria 1

Tracy McGregor, MD

  • Director, Clinical Development
  • Alnylam Pharmaceuticals, Inc., United States
James  Hamilton, MD, MBA

The Emerging Safety and Activity of TRiM™ Platform Based siRNA Drugs

James Hamilton, MD, MBA

  • Vice President, Clinical Development
  • Arrowhead Pharmaceuticals, United States