Alnylam Pharmaceuticals, Inc.
POLYNUCLEOTIDE AGENTS TARGETING AMINOLEVULINIC ACID SYNTHASE-1 (ALAS1) AND USES THEREOF
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Abstract:
The invention relates to polynucleotide agents, e.g., antisense polynucleotide 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.
Status:
Application
Type:
Utility
Filling date:
23 Feb 2021
Issue date:
6 Jan 2022