In today's featured presentation from
TIDES 2014, Jared A. Gollob, M.D., Vice President of Clinical Research at
Alnylam Pharmaceuticals, Inc. presents how explains how Transthyretin Amyloidosis is formed and how it develops throughout the body. Transthyretin (TTR) amyloidosis is caused by the deposition of liver-derived mutant and/or wild-type TTR in peripheral nerves, GI tract and the heart. Patisiran (ALN-TTR02), which is being developed for familial amyloidotic polyneuropathy (FAP), is a systemically administered lipid nanoparticle (LNP) formulation of a small interfering RNA (siRNA) targeting wild-type and all mutant forms of TTR that is currently in Phase 3. ALN-TTRsc, which is being developed to treat familial amyloidotic cardiomyopathy (FAC), is a subcutaneously administered N-acetylgalactosamine (GalNAc)-conjugated siRNA also targeting all forms of TTR that is currently in Phase 2. This presentation will review the clinical results to date with both patisiran and ALN-TTRsc. To learn more, watch the actual presentation:
TIDES 2015 will take place May 3-6, 2015 in San Diego, California. This year, Ravi S. Harapanhalli, Ph.D., Vice President, Technical, PAREXEL International and Former Branch Chief, Office of New Drug Quality Assessment, US FDA, will be on hand to present Strategies and Challenges in the Development of Oligonucleotide and Peptide Drugs: Perspectives on DrugDevice Combinations and Drug Product. For more information about this session and the rest of the program, download the agenda. As a reader of this blog, when you register to join us and mention code XB15180BLOG, you can save 20% off standard rates.
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