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Session 4: Rare Diseases
Session Chair(s)
Susan B. Sobolov, PhD
Executive Director, mRNA Portfolio Leader
Alexion Pharmaceuticals, United States
As a genetic technology, oligonucleotides can be specifically designed to modulate gene expression through multiple mechanisms and have a beneficial impact genetically defined rare diseases. The first speaker will provide an overview of RNA approaches to rare diseases and the expanding opportunity. The second speaker will discuss the FDA’s perspective on oligonucleotide-based therapeutics in rare disease. The next two speakers will focus on two clinical candidates in development for rare diseases. The first will present on ISIS- DMPK 2.5Rx an antisense oligonucleotide in Phase I/II for the treatment of myotonic dystrophy, and the last will discuss the Phase I results of ALN-AT3, an RNAi therapeutic for the treatment of hemophilia.
Speaker(s)
An Overview of Oligonucleotide in Rare Diseases
Susan B. Sobolov, PhD
Alexion Pharmaceuticals, United States
Executive Director, mRNA Portfolio Leader
FDA’s Perspective on Oligonucleotide-based Therapeutics in Rare Diseases
Richard Moscicki, MD
PhRMA, United States
Chief Medical Officer and Executive Vice President, Science and Regulatory
DM1 in Myotonic Dystrophy
Laurence Mignon, PhD
Ionis Pharmaceuticals, Inc., United States
Director, Clinical Development
A Subcutaneously Administered Investigational RNAi Therapeutic (ALN-AT3) Targeting Antithrombin for Treatment of Hemophilia: Phase 1 Study Results
Benny Sorensen, MD, PhD
Alnylam Pharmaceuticals, United States
Senior Director, Clinical Development
Q&A Panel Discussion
United States
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