354 patents
Page 16 of 18
Utility
LACTATE DEHYDROGENASE A (LDHA) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
1 Jul 20
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: 5 Mar 20
Utility
METHODS FOR TREATING OR PREVENTING CONTACT-ACTIVATION PATHWAY-ASSOCIATED DISEASES USING iRNA COMPOSITIONS TARGETING FACTOR XII (HAGEMAN FACTOR) (F12)
1 Jul 20
The present invention relates to methods of use of RNAi agents, e.g., double stranded RNAi agents, targeting a Factor XII (Hageman Factor (F12) gene, for treating subjects having a contact activation pathway-associated disease, such as a thrombophilia or hereditary angioedema (HAE), methods for preventing at least one symptom in a subject having a contact activation pathway-associated disease, such as a thrombus formation or an angioedema attack, and RNAi agents targeting an F12 gene, for use in the methods of the invention.
James Butler, Jingxuan Liu
Filed: 5 Jul 18
Utility
APOLIPOPROTEIN C3 (APOC3) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
24 Jun 20
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: 6 Aug 19
Utility
siRNA Therapy for Transthyretin (TTR) Related Ocular Amyloidosis
24 Jun 20
The invention relates to a method of treating ocular amyloidosis by reducing TTR expression in a subject by administering a double-stranded ribonucleic acid (dsRNA) that targets a TTR gene to the retinal pigment epithelium of the subject.
Yukio Ando, Hirofumi Jono, Rene Alvarez, Dinah Wen-Yee Sah
Filed: 8 Sep 19
Utility
Transthyretin (TTR) iRNA compositions and methods of use thereof for treating or preventing TTR-associated diseases
15 Jun 20
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: 17 Dec 18
Utility
Serpina1 iRNA compositions and methods of use thereof
15 Jun 20
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: 10 Nov 19
Utility
Compositions and Methods for Inhibiting Expression of the ALAS1 Gene
10 Jun 20
The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the ALAS1 gene, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of ALAS1.
Brian Bettencourt, Kevin Fitzgerald, William Querbes, Robert J. Desnick, Makiko Yasuda
Filed: 16 Jul 19
Utility
Modified iRNA agents
8 Jun 20
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 Feb 18
Utility
KETOHEXOKINASE (KHK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
3 Jun 20
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: 18 Jun 19
Utility
Modified RNAi agents
1 Jun 20
One aspect of the present invention relates to double-stranded RNAi (dsRNA) duplex agent capable of inhibiting the expression of a target gene.
Kallanthottathil G. Rajeev, Tracy Zimmermann, Muthiah Manoharan, Martin Maier, Satyanarayana Kuchimanchi, Klaus Charisse
Filed: 18 Oct 18
Utility
Compositions and methods directed to treating liver fibrosis
1 Jun 20
The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the COL1A1, TGF-β, and SMAD2/3 genes, and methods of using such dsRNA compositions to inhibit expression of COL1A1, TGF-β, and SMAD2/3.
Gregory Hinkle, Victor Kotelianski, Brian Bettencourt, Alfica Sehgal, Tatiana Novobrantseva
Filed: 25 Feb 18
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Therapeutic compositions
1 Jun 20
This application relates to therapeutic siRNA agents and methods of making and using the agents.
Muthiah Manoharan, Kallanthottathil G. Rajeev, David Bumcrot
Filed: 13 Mar 19
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Methods For Inhibition Of HAO1 (Hydroxyacid Oxidase 1 (Glycolate Oxidase)) Gene Expression
27 May 20
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
Filed: 11 Jul 18
Utility
Myostatin Irna Compositions and Methods of Use Thereof
6 May 20
The present invention provides methods comprising the in vivo delivery of small nucleic acid molecules capable of mediating RNA interference and reducing the expression of myostatin, wherein the small nucleic acid molecules are introduced to a subject by systemic administration.
Vasant R. Jadhav, Rubina Parmar, Laura Sepp-Lorenzino, Muthiah Manoharan
Filed: 19 Sep 17
Utility
ANGIOPOIETIN-LIKE 3 (ANGPTL3) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
6 May 20
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: 13 May 19
Utility
Methods and Compositions for Treating a Proprotein Convertase Subtilisin Kexin (PCSK9) Gene-associated Disorder
6 May 20
The invention relates to methods of inhibiting the expression of a PCSK9 gene in a subject, as well as therapeutic and prophylactic methods for treating subjects having a lipid disorder, such as a hyperlipidemia using RNAi agents, e.g., double-stranded RNAi agents, targeting the PCSK9 gene.
Kevin Fitzgerald
Filed: 15 Jan 20
Utility
HEPATITIS B VIRUS (HBV) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
6 May 20
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: 3 Nov 19
Utility
Hepatitis D virus (HDV) iRNA compositions and methods of use thereof
4 May 20
The present invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the hepatitis D virus (HDV) genome, and methods of using such RNAi agents to inhibit expression of one or more HBV genes and methods of treating subjects having an HDV infection and/or HDV-associated disorder.
Gregory Hinkle, Laura Sepp-Lorenzino, Vasant Jadhav, Martin Maier, Muthiah Manoharan, Stuart Milstein, Svetlana Shulga Morskaya, Kallanthottathil G. Rajeev, Huilei Xu
Filed: 9 May 17
Utility
FACTOR XII (HAGEMAN FACTOR) (F12), KALLIKREIN B, PLASMA (FLETCHER FACTOR) 1 (KLKB1), AND KININOGEN 1 (KNG1) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
29 Apr 20
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: 23 May 19
Utility
LACTATE DEHYDROGENASE A (LDHA) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
15 Apr 20
The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the LDHA gene, as well as methods of inhibiting epression 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: 16 Dec 19