Alnylam Pharmaceuticals, Inc.
Polynucleotide agents targeting aminolevulinic acid synthase-1 (ALAS1) and uses thereof

Abstract:

The invention relates to polynucleotide agents, e.g., antisense polunucleotide agents, targeting the ALAS1 gene, and methods of using such agents to alter (e.g., inhibit) expression of ALAS1 and to treat ALAS1 associated diseases, e.g., porphyria.