Tracy S Zimmermann
Affiliation: Alnylam Pharmaceuticals
Zimmermann T, Karsten V, Chan A, Chiesa J, Boyce M, Bettencourt B, et al
. Clinical Proof of Concept for a Novel Hepatocyte-Targeting GalNAc-siRNA Conjugate. Mol Ther. 2017;25:71-78 pubmed publisher
..These results demonstrate translation of this novel delivery platform, enabling clinical development of subcutaneously administered GalNAc-siRNAs for liver-based diseases. ..
Willoughby J, Chan A, Sehgal A, Butler J, Nair J, Racie T, et al
. Evaluation of GalNAc-siRNA Conjugate Activity in Pre-clinical Animal Models with Reduced Asialoglycoprotein Receptor Expression. Mol Ther. 2018;26:105-114 pubmed publisher
..Collectively, our data support a broad application of the GalNAc-siRNA technology for hepatic targeting, including disease states where ASGPR expression may be reduced. ..