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ALNY Alnylam Pharmaceuticals

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High density lipoprotein binding protein (Hdlbp/Vigilin) iRNA compositions and methods of use thereof
28 Dec 21
The invention relates to double stranded ribonucleic acid (dsRNA) agents and compositions targeting a High Density Lipoprotein Binding Protein (Hdlbp/Vigilin) gene, as well as methods of inhibiting expression of Hdlbp/Vigilin and methods of treating subjects having a disorder of lipid metabolism, such as mixed hyperlipidemia, hypertriglyceridemia or hypercholesterolemia, using such dsRNA agents and compositions.
Muthiah Manoharan, Markus Stoffel, Mehrpouya Balaghy Mobin
Filed: 17 Oct 18
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Coronavirus iRNA compositions and methods of use thereof
28 Dec 21
The present invention relates to RNAi agents, e.g., dsRNA agents, targeting the coronavirus genome.
Akin Akinc, James D. McIninch, Yesseinia Anglero-Rodriguez, Mark K. Schlegel, Christy M. Hebner, Florian A. Lempp
Filed: 17 May 21
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Angiopoietin-like 3 (ANGPTL3) iRNA compositions and methods of use thereof
14 Dec 21
The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the ANGPTL3 gene, as well as methods of inhibiting expression of ANGPTL3 and methods of treating subjects having a disorder of lipid metabolism, such as hyperlipidemia or hypertriglyceridemia, using such dsRNA compositions.
William Querbes, Kevin Fitzgerald, James Butler, Stephanie Williams, Gregory Hinkle, Martin A. Maier
Filed: 12 Dec 19
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Compositions and methods for inhibiting expression of TMPRSS6 gene
14 Dec 21
The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the TMPRSS6 gene, and methods of using such dsRNA compositions to inhibit expression of TMPRSS6.
David Bumcrot, Brian Bettencourt, Ivanka Toudjarska
Filed: 4 Sep 18
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Complement component iRNA compositions and methods of use thereof
30 Nov 21
The invention relates to iRNA, e.g., double-stranded ribonucleic acid (dsRNA), compositions targeting the complement factor B (CFB) gene, the complement component C3 gene, and the complement component C9 gene and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of CFB, C9 and/or C3 and to treat subjects having a complement component-associated disease, e.g., paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome.
Anna Borodovsky, Brian Bettencourt
Filed: 18 Sep 19
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Complement component C5 iRNA compositions and methods of use thereof
2 Nov 21
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.
Kevin Fitzgerald, James Butler, Brian Bettencourt, Anna Borodovsky, Satyanarayana Kuchimanchi, Klaus Charisse, Muthiah Manoharan, Martin A. Maier, Kallanthottathil G. Rajeev, Donald Foster
Filed: 7 May 19
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Apolipoprotein C3 (APOC3) iRNA compositions and methods of use thereof
2 Nov 21
The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the apolipoprotein C3 gene (APOC3).
Frederic Tremblay, Lucas D. BonDurant, James D. McIninch, Adam Castoreno, Mark K. Schlegel, Charalambos Kaittanis
Filed: 22 Feb 21
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Lipid formulated compositions and methods for inhibiting expression of serum amyloid A gene
19 Oct 21
The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting a Serum Amyloid A (SAA) gene, and methods of using the dsRNA to inhibit expression of SAA
Antonin de Fougerolles, Tatiana Novobrantseva, Gregory Hinkle
Filed: 3 Oct 19
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Patatin-like phospholipase domain containing 3 (PNPLA3) iRNA compositions and methods of use thereof
19 Oct 21
The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the Patatin-Like Phospholipase Domain Containing 3 (PNPLA3) gene.
Frederic Tremblay, James D. McIninch, Adam Castoreno, Mark K. Schlegel, Charalambos Kaittanis
Filed: 23 Feb 21
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Apolipoprotein C3 (APOC3) iRNA compositions and methods of use thereof
12 Oct 21
The present invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the apolipoprotein C3 (APOC3) gene, and methods of using such RNAi agents to inhibit expression of APOC3 and methods of treating subjects having an APOC3 associated disorder, e.g., hypertriglyceridemia.
Kevin Fitzgerald, William Querbes, James Butler, Stephanie Williams, Abigail Liebow, Gregory Hinkle, Martin A. Maier, Stuart Milstein, Satyanarayana Kuchimanchi, Muthiah Manoharan
Filed: 7 Aug 19
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Ketohexokinase (KHK) iRNA compositions and methods of use thereof
5 Oct 21
The present invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the ketohexokinase (KHK) gene, and methods of using such RNAi agents to inhibit expression of KHK and methods of treating subjects having a KHK-associated disorder, e.g., liver disease (e.g., fatty liver, steatohepatitis), dyslipidemia (e.g., hyperlipidemia, high LDL cholesterol, low HDL cholesterol, hypertriglyceridemia, postprandial hypertriglyceridemia), disorders of glycemic control (e.g., insulin resistance, diabetes), cardiovascular disease (e.g., hypertension, endothelial cell dysfunction), kidney disease (e.g., acute kidney disorder, tubular dysfunction, proinflammatory changes to the proximal tubules), metabolic syndrome, adipocyte dysfunction, visceral adipose deposition, obesity, hyperuricemia, gout, eating disorders, and excessive sugar craving.
Kevin Fitzgerald, Brian Bettencourt, Gregory Hinkle, Jennifer Willoughby
Filed: 19 Jun 19
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Angiopoietin-like 3 (ANGPTL3) iRNA compositions and methods of use thereof
28 Sep 21
The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the ANGPTL3 gene, as well as methods of inhibiting expression of ANGPTL3 and methods of treating subjects having a disorder of lipid metabolism, such as hyperlipidemia or hypertriglyceridemia, using such dsRNA compositions.
Brian Bettencourt, William Querbes, Kevin Fitzgerald, Maria Frank-Kamenetsky, Stuart Milstein, Svetlana Shulga Morskaya
Filed: 26 Jan 21
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Compositions and methods for inhibition of expression of protein C (PROC) genes
14 Sep 21
The invention relates to double-stranded ribonucleic acid (dsRNA) targeting a PROC gene, and methods of using the dsRNA to inhibit expression of PROC.
Ivanka Toudjarska, John M. Maraganore, Brian Bettencourt, Stuart Milstein, Martin A. Maier, Klaus Charisse, Kallanthottathil Rajeev, Satyanarayana Kuchimanchi
Filed: 11 Nov 19
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Carbohydrate conjugates as delivery agents for oligonucleotides
7 Sep 21
Muthiah Manoharan, Kallanthottathil G. Rajeev, Jayaprakash Nair, Martin Maier
Filed: 29 Apr 20
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Methods of treating transthyretin (TTR) mediated amyloidosis
3 Aug 21
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.
Brian Bettencourt
Filed: 3 Aug 18
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Biodegradable lipids for the delivery of active agents
27 Jul 21
The present invention relates to a cationic lipid having one or more biodegradable groups located in a lipidic moiety (e.g., a hydrophobic chain) of the cationic lipid.
Martin Maier, Muthusamy Jayaraman, Akin Akinc, Shigeo Matsuda, Pachamuthu Kandasamy, Kallanthottathil G. Rajeev, Muthiah Manoharan
Filed: 23 Jul 19
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Insulin-like growth factor binding protein, acid labile subunit (IGFALS) and insulin-like growth factor 1 (IGF-1) iRNA compositions and methods of use thereof
20 Jul 21
The present invention relates to RNAi agents, e.g., double stranded RNAi agents, targeting the insulin-like growth factor binding protein, acid labile subunit (IGFALS) gene or the insulin-like growth factor 1 (IGF-1) gene, methods of using such double stranded RNAi agents to inhibit expression of an IGFALS gene or an IGF-1 gene, and methods of treating subjects having an IGF system-associated disorder.
James Butler, Kevin Fitzgerald, Gregory Hinkle, Brian Bettencourt, Huilei Xu
Filed: 15 Oct 19
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Hepatitis B virus (HBV) iRNA compositions and methods of use thereof
13 Jul 21
The present invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the hepatitis B virus (HBV) genome, and methods of using such RNAi agents to inhibit expression of one or more HBV genes and methods of treating subjects having an HBV infection and/or HBV-associated disorder, e.g., chronic hepatitis B infection.
Gregory Hinkle, Laura Sepp-Lorenzino, Vasant Jadhav, Martin Maier, Stuart Milstein, Muthiah Manoharan, Kallanthottathil G. Rajeev
Filed: 4 Nov 19
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Patatin-like phospholipase domain containing 3 (PNPLA3) iRNA compositions and methods of use thereof
6 Jul 21
The present invention relates to RNAi agents, e.g., double stranded RNAi agents, targeting the Patatin-Like Phospholipase Domain Containing 3 (PNPLA3) gene, and methods of using such RNAi agents to inhibit expression of a PNPLA3 gene and methods of treating subjects having Nonalcoholic Fatty Liver Disease (NAFLD) and/or a PNPLA3-associated disorder.
Kevin Fitzgerald, Gregory Hinkle
Filed: 5 Feb 19
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Multi-targeted single entity conjugates
6 Jul 21
The present invention relates, in general to, compounds, compositions and methods useful for modulating gene expression of multiple target nucleic acids by a single chemical entity.
Vasant Jadhav, Martin Maier, Ivan Zlatev, Akin Akinc, Kallanthottathil G. Rajeev, Jayaprakash K. Nair, Pachamuthu Kandasamy, Muthiah Manoharan
Filed: 15 Jul 16
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