Cambridge, MA, United States

Alnylam Pharmaceuticals

www.alnylam.com
Cambridge, MA, United States
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Patent
Alnylam Pharmaceuticals | Date: 2017-03-29

The present invention relates to RNAi agents, e.g., double- stranded RNAi agents, targeting the angiotensinogen (AGT) gene, and methods of using such RNAi agents to inhibit expression of AGT and methods of treating subjects having an AGT-associated disorder, e.g., hypertension.


Patent
Alnylam Pharmaceuticals | Date: 2017-07-26

The invention relates to iRNA, e.g., double- stranded ribonucleic acid (dsRNA), compositions targeting the complement component C5 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of C5 and to treat subjects having a complement component C5-associated disease, e.g., paroxysmal nocturnal hemoglobinuria.


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.


The invention relates to polynucleotide agents targeting the complement component C5 gene, and methods of using such polynucleotide agents to inhibit expression of C5 and to treat subjects having a complement component C5-associated disease, e.g., paroxysmal nocturnal hemoglobinuria.


Patent
Alnylam Pharmaceuticals | Date: 2017-07-05

One aspect of the present invention relates to double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene. The sense strand of the dsRNA agent comprises at least one thermally destabilizing nucleotide, and at least one said thermally destabilizing nucleotide occurring at a site opposite to the seed region (positions 2-8) of the antisense strand; and the antisense strand of the dsRNA agent comprises at least two modified nucleotides that provide the nucleotide a steric bulk that is less than or equal to the steric bulk of a 2-OMe modification, wherein said modified nucleotides are separated by 11 nucleotides in length. Other aspects of the invention relates to pharmaceutical compositions comprising these dsRNA agents suitable for therapeutic use, and methods of inhibiting the expression of a target gene by administering these dsRNA agents, e.g., for the treatment of various disease conditions.


Patent
Alnylam Pharmaceuticals | Date: 2017-03-15

The present invention provides iRNA agents comprising at least one subunit of the formula (I): wherein: A and B are each independently for each occurrence O, N(R^(N)) or S; X and Y are each independently for each occurrence H, OH, a hydroxyl protecting group, a phosphate group, a phosphodiester group, an activated phosphate group, an activated phosphite group, a phosphoramidite, a solid support, - P(Z)(Z)0- nucleoside, -P(Z)(Z)O-oligonucleotide, a lipid, a PEG, a steroid, a lipophile, a polymer, P(Z)(Z)O-Linker-OP(Z)(Z)O-oligonucleotide, a nucleotide, an oligonucleotide, - P(Z)(Z)-formula(I), -P(Z)(Z)- or -Linker-R; R is L^(G), -Linker-L^(G), or has the structure shown below: L^(G) is independently for each occurence a carbohydrate, e.g., monosaccharide, disaccharide, trisaccharide, tetrasaccharide, oligosaccharide, polysaccharide; R^(N) is independently for each occurrence H, methyl, ethyl, propyl, isopropyl, butyl, or benzyl; and Z, Z, Z and Z are each independently for each occurrence O or S.


Patent
Alnylam Pharmaceuticals | Date: 2017-07-05

Disclosed herein are methods for reducing or arresting an increase in a Neuropathy Impairment Score (NIS) or a modified NIS (mNIS+7) in a human subject by administering an effective amount of a transthyretin (TTR)-inhibiting composition.


The invention relates to polynucleotide agents targeting the complement component C5 gene, and methods of using such polynucleotide agents to inhibit expression of C5 and to treat subjects having a complement component C5-associated disease, e.g., paroxysmal nocturnal hemoglobinuria.


Patent
Alnylam Pharmaceuticals | Date: 2017-03-01

Assays, methods, reagents and kits for evaluating the level of an antibody against a nucleic acid molecule, e.g., a double- stranded oligonucleotide or RNA molecule (e.g., dsRNA), are disclosed herein.


Patent
Alnylam Pharmaceuticals | Date: 2017-01-06

The invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the Serpina1 gene, and methods of using such RNAi agents to inhibit expression of Serpina1 and methods of treating subjects having a Serpina1 associated disease, such as a liver disorder.

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