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ALNY Alnylam Pharmaceuticals

GRANT
Utility
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
APP
Utility
Compositions and Methods for Treating Transthyretin (TTR) Mediated Amyloidosis
25 Nov 21
Disclosed herein are methods for treating hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in a human patient in need thereof by administering an effective amount of a transthyretin (TTR)-inhibiting composition.
Jared Gollob
Filed: 19 Sep 18
APP
Utility
Compositions and Methods for Inhibiting Expression of the LECT2 Gene
11 Nov 21
The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the LECT2 gene, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of LECT2.
Gregory Hinkle, Huilei Xu
Filed: 15 Aug 19
GRANT
Utility
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
GRANT
Utility
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
APP
Utility
HEPATITIS B VIRUS (HBV) dsRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
28 Oct 21
The present disclosure relates to double stranded RNA agents targeting the hepatitis B virus (HBV) genome, and methods of using such agents to inhibit expression of one or more HBV genes and methods of treating subjects having an HBV infection or HBV-associated disorder, e.g., chronic hepatitis B infection.
Vasant R. JADHAV, Martin A. MAIER, Stuart MILSTEIN, Mark K. SCHLEGEL
Filed: 12 Aug 19
APP
Utility
KETOHEXOKINASE (KHK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
28 Oct 21
The present invention relates to RNAi agents, e.g., dsRNA agents, targeting the ketohexokinase (KHK) gene.
Gregory Hinkle
Filed: 17 Sep 19
GRANT
Utility
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
GRANT
Utility
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
APP
Utility
SERPINA1 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
14 Oct 21
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.
Alfica Sehgal, Klaus Charisse, Brian Bettencourt, Martin A. Maier, Kallanthottathil G. Rajeev, Gregory Hinkle, Muthiah Manoharan
Filed: 5 Jan 21
APP
Utility
Polynucleotide Agents Targeting Patatin-like Phospholipase Domain Containing 3 (PNPLA3) and Methods of Use Thereof
14 Oct 21
The invention relates to polynucleotide agents targeting a Patatin-Like Phospholipase Domain Containing 3 (PNPLA3) gene, and methods of using such polynucleotide 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.
Gregory Hinkle
Filed: 9 Nov 20
GRANT
Utility
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
APP
Utility
ANGIOTENSINOGEN (AGT) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
7 Oct 21
The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the AGT gene.
Donald Foster, Gregory Hinkle, Mark K. Schlegel
Filed: 7 Apr 21
GRANT
Utility
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
GRANT
Utility
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
APP
Utility
APOLIPOPROTEIN C3 (APOC3) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
23 Sep 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
GRANT
Utility
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
GRANT
Utility
Carbohydrate conjugates as delivery agents for oligonucleotides
7 Sep 21
Muthiah Manoharan, Kallanthottathil G. Rajeev, Jayaprakash Nair, Martin Maier
Filed: 29 Apr 20
APP
Utility
SERPINA1 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
2 Sep 21
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.
Mark K. Schlegel, Maja Janas, Vasant R. Jadhav, Donald Foster, Muthiah Manoharan, Kallanthottathil G. Rajeev, Alexander V. Kel'in, Klaus Charisse, Jayaprakash K. Nair, Martin A. Maier, Shigeo Matsuda, Muthusamy Jayaraman, Alfica Sehgal, Christopher Brown, Kevin Fitzgerald, Stuart Milstein
Filed: 8 Mar 21
APP
Utility
Complement Component C3 Irna Compositions and Methods of Use Thereof
26 Aug 21
The invention relates to iRNA, e.g., double stranded ribonucleic acid (dsRNA), compositions targeting the complement factor C3 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of a C3 gene and to treat subjects having a complement component C3-associated disease, e.g., paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), atypical hemolytic uremic syndrome (aHUS), neuromyelitis optica (NMO), multifocal motor neuropathy (MMN), myasthenia gravis (MG), and C3 glomerulonephritis.
Gregory Hinkle, Anna Borodovsky
Filed: 1 Nov 18
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