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Session 3C (Clinical Development Track): Liver Targets
Session Chair(s)
Richard Stephen Geary, PhD
Senior Vice President, Drug Development
Ionis Pharmaceuticals, Inc., United States
This session will provide updates on antisense and siRNA development programs focused on liver targets. Included in this session are two second generation antisense drugs targeting apoC-III and C-reactive protein (CRP), respectively, as well as two approaches to delivering siRNA for targeting transthyretin in TTR amyloidosis. All of these disease associated targets are primarily synthesized in the liver. This session will also provide insights into clinical study designs and development plans as well as proof of concept and safety assessments for two platform approaches to targeting mRNA transcripts in the liver.
Speaker(s)
Targeting APOC-III in Severe Hypertriglyceridemia
Walter Singleton, MD
Isis Pharmaceuticals Inc., United States
Chief Medical Officer
Clinical Trials of ALN-TTR02, an siRNA Therapeutic for Transthyretin Amyloidosis
Jared A. Gollob, MD
Alnylam Pharmaceuticals, Inc., United States
Vice President, Clinical Research
Isis CRPRx: Pharmacokinetics, Pharmacodynamics, Safety and Tolerability
Marshelle Smith Warren, MD
Isis Pharmaceuticals Inc., United States
Executive Director
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