118 patents
Page 3 of 6
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
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
Methods for inhibition of HAO1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression
1 Mar 22
The invention relates methods of using RNAi agents to inhibit expression of HAO1 and methods of treating subjects having, e.g., PH1.
David V. Erbe, Tracy L. McGregor
Filed: 12 Jul 18
Utility
Biodegradable lipids for the delivery of active agents
15 Feb 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: 29 Apr 21
Utility
Serum amyloid P component (APCS) iRNA compositions and methods of use thereof
25 Jan 22
The invention relates to iRNA, e.g., double stranded ribonucleic acid (dsRNA), compositions targeting the serum amyloid P component (APCS) gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of an APCS gene and to treat subjects having an APCS-associated disease, e.g., amyloidosis, Alzheimer's disease or coronary atherosclerotic heart disease.
Kevin Fitzgerald, Gregory Hinkle
Filed: 19 May 20
Utility
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
Utility
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
Utility
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
Utility
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
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
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
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
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
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
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
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
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