354 patents
Page 11 of 18
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
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
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
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
Utility
Carbohydrate conjugates as delivery agents for oligonucleotides
7 Sep 21
Muthiah Manoharan, Kallanthottathil G. Rajeev, Jayaprakash Nair, Martin Maier
Filed: 29 Apr 20
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
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
Utility
iRNA AGENTS WITH BIOCLEAVABLE TETHERS
19 Aug 21
The invention relates to iRNA agents, which preferably include a monomer in which the ribose moiety has been replaced by a moiety other than ribose that further includes a tether having one or more linking groups, in which at least one of the linking groups is a cleavable linking group.
Muthiah MANOHARAN, Kallanthottathil G. RAJEEV
Filed: 28 Apr 21
Utility
TMPRSS6 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
19 Aug 21
The invention relates to RNAi agents, e.g, double-stranded RNAi agents, targeting the TMPRSS6 gene, and methods of using such RNAi agents to inhibit expression of TMPRSS6 and methods of treating subjects having a TMPRSS6 associated disorder, e.g., an iron overload associated disorder, such as β-thalassemia or hemochromatosis.
James Butler, Brian Bettencourt, Kallanthottathil G. Rajeev, Martin A. Maier, Klaus Charisse
Filed: 15 Dec 20
Utility
Factor XII (Hageman Factor) (F12), KALLIKREIN B, PLASMA (FLETCHER FACTOR) 1 (KLKB1), and Kininogen 1 (KNG1) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
12 Aug 21
The present invention relates to RNAi agents, e.g., double stranded RNAi agents, targeting the Kallikrein B, Plasma (Fletcher Factor) 1 (KLKB1) gene, the Factor XII (Hageman Factor (F12) gene, or the Kininogen 1 (KNG1) gene, and methods of using such RNAi agents to inhibit expression of a KLKB1 gene, an F12 gene, and/or a KNG1 gene, and methods of treating subjects having an hereditary angioedema (HAE) and/or a contact activation pathway-associated disorder.
Akin Akinc, Gregory Hinkle, Martin A. Maier, James Butler, Jingxuan Liu
Filed: 15 Dec 20
Utility
Compositions and Methods for Improving Strand Biased
5 Aug 21
One aspect of the present invention relates to double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene.
Rubina G. PARMAR, Pawan KUMAR, Masaaki AKABANE-NAKATA, Shigeo MATSUDA, Muthiah MANOHARAN
Filed: 7 May 19
Utility
Modified Rna Agents with Reduced Off-target Effect
5 Aug 21
One aspect of the present invention relates to double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene.
Shigeo MATSUDA, Mark K. SCHLEGEL, Maja JANAS, Vasant R. JADHAV, Martin MAIER, Klaus CHARISSE, Muthiah MANOHARAN, Kallathotathil G. RAJEEV, Jayaprakash K. NAIR
Filed: 16 May 19
Utility
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
Utility
LACTATE DEHYDROGENASE A (LDHA) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
29 Jul 21
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.
David Erbe, Abigail Liebow, Kevin Fitzgerald, Gregory Hinkle, Kyle David Wood, Ross Philip Holmes, John Knight
Filed: 30 Nov 20
Utility
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
Utility
Polynucleotide Agents Targeting Factor XII (Hageman Factor) (F12) and Methods of Use Thereof
22 Jul 21
The invention relates to polynucleotide agents targeting Factor XII (F12) gene, and methods of using such polynucleotide agents to inhibit expression of Factor XII and to treat subjects having a Factor XII-associated disease, e.g., heredity angioedema (HAE), prekallikrein deficiency, malignant essential hypertension, hypertension, end stage renal disease, or Fletcher Factor Deficiency.
Gregory Hinkle
Filed: 24 Nov 20
Utility
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
Utility
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
Utility
Chirally-enriched Double-stranded Rna Agents
8 Jul 21
The present invention relates to a chirally-modified dsRNA agent capable of inhibiting the expression of a target gene.
Muthiah MANOHARAN, Nate TANEJA, Hartmut Ingo JAHNS, Shigeo MATSUDA, Klaus CHARISSE, Guo HE, Jayaprakash K. NAIR, Christopher BROWN, Mark K. SCHLEGEL, Vasant JADHAV, Martin MAIER
Filed: 21 Dec 18
Utility
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