Alnylam Starts Phase II Study of Subcutaneous ATTR Drug | GenomeWeb

Alnylam Pharmaceuticals this week announced that it has begun a phase II trial of its subcutaneously delivered TTR amyloidosis treatment ALN-TTRsc.

ATTR is caused by mutations in the TTR gene, which triggers accumulation of abnormal amyloid proteins in the body. ALN-TTRsc is designed to inhibit both the wild-type and mutant forms of the protein and is formulated with the GalNAc technology, which facilitates liver delivery of siRNAs via uptake through the asialoglycoprotein receptors expressed on the surface of hepatocytes.

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