Ligand conjugated antisense oligonucleotide for the treatment of transthyretin amyloidosis: preclinical and phase 1 data

Ligand conjugated antisense oligonucleotide for the treatment of transthyretin amyloidosis: preclinical and phase 1 data

Abstract Aims Amyloidogenic transthyretin (ATTR) amyloidosis is a fatal disease characterized by progressive cardiomyopathy and/or polyneuropathy. AKCEA‐TTR‐LRx (ION‐682884) is a ligand‐conjugated antisense drug designed for receptor‐mediated uptake by hepatocytes, the primary source of circulating...

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Bibliographic Details
Main Authors: Nicholas J. Viney, Shuling Guo, Li‐Jung Tai, Brenda F. Baker, Mariam Aghajan, Shiangtung W. Jung, Rosie Z. Yu, Sheri Booten, Heather Murray, Todd Machemer, Sebastien Burel, Sue Murray, Gustavo Buchele, Sotirios Tsimikas, Eugene Schneider, Richard S. Geary, Merrill D. Benson, Brett P. Monia
Format: Article
Language:English
Published: Wiley 2021-02-01
Series:ESC Heart Failure
Subjects:
Transthyretin
Amyloidosis
Cardiomyopathy
Polyneuropathy
Antisense
Ligand‐conjugated
Online Access:https://doi.org/10.1002/ehf2.13154

Internet

https://doi.org/10.1002/ehf2.13154

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