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Bethesda North Marriott Hotel and Conference Center

Oct 28, 2019 7:00 AM - Oct 30, 2019 12:30 PM

5701 Marinelli Road, , North Bethesda, MD 20852 , USA

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


Erin  Morgan

Erin Morgan

Ionis Pharmaceuticals, Inc, United States

Executive Director, Clinical Development

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

Alnylam Pharmaceuticals, Inc., United States

Director, Clinical Development

James Hamilton Hamilton, MD, MBA

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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