LACTATE DEHYDROGENASE A (LDHA) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
AssigneesAlnylam Pharmaceuticals, Inc., The UAB Research Foundation
InventorsDavid Erbe, Abigail Liebow, Kevin Fitzgerald, Gregory Hinkle, Kyle David Wood, Ross Philip Holmes, John Knight
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Abstract
The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the LDHA gene, as well as methods of inhibiting expression of LDHA, methods of inhibiting LDHA and HAO1, and methods of treating subjects that would benefit from reduction in expression of LDHA, such as subjects having an oxalate pathway-associated disease, disorder, or condition, using such dsRNA compositions.
Claims27
A double stranded ribonucleic acid (dsRNA) agent for inhibiting expression of lactic acid dehydrogenase A (LDHA) in a cell, wherein the dsRNA agent comprises a sense strand and an antisense strand forming a double stranded region,
- wherein the antisense strand comprises at least 15 contiguous nucleotides differing by no more than 3 nucleotides from any one of the antisense sequences listed in any one of Tables 2-5,
- wherein all of the nucleotides of the sense strand and all of the nucleotides of the antisense comprise a nucleotide modification, and
- wherein a ligand comprising one or more N-acetylgalactosamine (GalNAc) derivatives attached through a monovalent, bivalent, or trivalent branched linker is conjugated to at least one strand of the agent.
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Dependencies are inferred from the claim text.