354 patents
Page 8 of 18
Utility
Carbohydrate Conjugates As Delivery Agents for Oligonucleotides
12 May 22
The present invention provides a modified RNAi agent comprising a sense strand and an antisense strand, wherein the antisense strand targets an HBV mRNA; all of the nucleotide sugars of the RNAi agent are 2′-modified; and at least one of the sense and antisense strands is conjugated to at least one targeting ligand that comprises two or more building blocks connected to a biantennary or triantennary branched linker, optionally via one or more linking groups.
Muthiah MANOHARAN, Kallanthottathil G. RAJEEV, Jayaprakash K. NAIR, Martin MAIER
Filed: 23 Nov 21
Utility
Methods for the treatment of subjects having a hepatitis b virus (HBV) infection
10 May 22
The present invention provides methods for the treatment of a subject having a Hepatitis B virus (HBV) infection, e.g., a chronic HBV infection, using a combination of an RNAi agent that targets HBV and an HBV vaccine.
Laura Sepp-Lorenzino, Ulrike Protzer, Thomas Michler
Filed: 18 Apr 18
Utility
Xanthine dehydrogenase (XDH) iRNA compositions and methods of use thereof
10 May 22
The present invention relates to RNAi agents, e.g., dsRNA agents, targeting the xanthine dehydrogenase (XDH) gene.
James D. McIninch, Jingxuan Liu, Mark K. Schlegel, Adam Castoreno, Anna Borodovsky
Filed: 20 Oct 21
Utility
Xanthine dehydrogenase (XDH) IRNA compositions and methods of use thereof
3 May 22
The present invention relates to RNAi agents, e.g., double stranded RNAi agents, targeting a xanthine dehydrogenase (XDH) gene, and methods of using such double stranded RNAi agents to inhibit expression of an XDH gene and methods of treating subjects having an XDH-associated disease.
Kevin Fitzgerald, Gregory Hinkle, Timothy Ryan Mooney
Filed: 27 Jan 20
Utility
Extrahepatic Delivery
28 Apr 22
The invention relates to a method of gene silencing, comprising administering to a cell or a subject in need thereof a therapeutically effective amount of the lipophilic moieties-conjugated double-stranded iRNAs at one or more internal positions on at least one strand, optionally via a linker or carrier.
Jayaprakash K. NAIR, Martin MAIER, Vasant JADHAV, Stuart MILSTEIN, Kirk BROWN, Rubina G. PARMAR, Kallanthottathil G. RAJEEV, Muthiah MANOHARAN, Alexander V. KEL'IN, Muthusamy JAYARAMAN, Klaus CHARISSE, Adam CASTORENO, Christopher S. THEILE, Kevin FITZGERALD
Filed: 7 May 19
Utility
Reversir TM Compounds
28 Apr 22
The present invention relates, in general to agents that modulate the pharmacological activity of conjugated siRNAs.
Vasant JADHAV, John MARAGANORE, Martin MAIER, Kallanthottathil G. RAJEEV, Muthiah MANOHARAN, Akin AKINC, Ivan ZLATEV
Filed: 10 Aug 21
Utility
Biodegradable Lipids for the Delivery of Active Agents
28 Apr 22
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: 12 Jan 22
Utility
Coronavirus Irna Compositions and Methods of Use Thereof
28 Apr 22
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: 10 Nov 21
Utility
KETOHEXOKINASE (KHK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
28 Apr 22
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: 2 Sep 21
Utility
Modified iRNA agents
26 Apr 22
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.
Muthiah Manoharan, Venkitasamy Kesavan, Kallanthottathil Rajeev
Filed: 26 Jul 19
Utility
PATATIN-LIKE PHOSPHOLIPASE DOMAIN CONTAINING 3 (PNPLA3) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
21 Apr 22
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: 1 Jun 21
Utility
Angiopoietin-like 3 (ANGPTL3) Irna Compositions and Methods of Use Thereof
21 Apr 22
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: 3 Nov 21
Utility
Angiopoietin-like 3 (ANGPTL3) iRNA compositions and methods of use thereof
19 Apr 22
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: 22 Nov 21
Utility
Angiopoietin-like 3 (ANGPTL3) iRNA compositions and methods of use thereof
19 Apr 22
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: 22 Nov 21
Utility
Angiopoietin-like 3 (ANGPTL3) iRNA compositions and methods of use thereof
19 Apr 22
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: 22 Nov 21
Utility
Biodegradable Lipids for the Delivery of Active Agents
14 Apr 22
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: 17 Dec 21
Utility
Endosomal cleavable linkers
29 Mar 22
The present disclosure relates generally to cleavable linkers and uses thereof.
Jayaprakash K. Nair, Kallanthottathil G. Rajeev, Scott Lentini, Christopher S. Theile, Martin Maier, Ivan Zlatev
Filed: 18 Jan 18
Utility
Transthyretin (TTR) iRNA compositions and methods of use thereof for treating or preventing TTR-associated diseases
29 Mar 22
The present invention provides iRNA agents, e.g., double stranded iRNA agents, that target the transthyretin (TTR) gene and methods of using such iRNA agents for treating or preventing TTR-associated diseases.
Tracy Zimmermann, Amy Chan, Vasant R. Jadhav, Martin A. Maier, Kallanthottathil G. Rajeev
Filed: 1 May 20
Utility
COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
24 Mar 22
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: 12 Jul 21
Utility
ANGIOPOIETIN-LIKE 3 (ANGPTL3) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
10 Mar 22
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: 22 Nov 21