354 patents
Page 14 of 18
Utility
Polynucleotide agents targeting factor XII (hageman factor) (F12) and methods of use thereof
4 Jan 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: 5 Jan 17
Utility
TMPRSS6 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
16 Dec 20
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: 17 Aug 20
Utility
Kisspeptin 1 (KISS1) Irna Compositions and Methods of Use Thereof
9 Dec 20
The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the KISS1 gene.
Mangala Meenakshi Soundarapandian, Gregory Hinkle
Filed: 14 Nov 18
Utility
Polynucleotide Agents Targeting Angiotensinogen (Agt) and Methods of Use Thereof
9 Dec 20
The invention relates to polynucleotide agents, e.g., antisense polynucleotide agents, targeting an angiotensinogen (AGT) gene, and methods of using such polynucleotide agents to inhibit expression of AGT and to treat subjects having an AGT-associated disease, e.g., hypertension.
Gregory Hinkle
Filed: 19 Aug 20
Utility
TRANSTHYRETIN (TTR) iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING OR PREVENTING TTR-ASSOCIATED DISEASES
2 Dec 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: 30 Apr 20
Utility
Compositions and Methods for Inhibiting Expression of Mutant Egfr Gene
2 Dec 20
The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting a mutant Epidermal Growth Factor Receptor (EGFR), and methods of using the dsRNA to inhibit expression of mutant EGFR.
Dinah Sah, Pamela Tan, Webster Cavenee, Frank Furnari, Maria del Mar Inda Perez, Rudy Bonavia
Filed: 2 Mar 20
Utility
Methods and compositions for treating a proprotein convertase subtilisin kexin (PCSK9) gene-associated disorder
30 Nov 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
Glucokinase (GCK) iRNA compositions and methods of use thereof
23 Nov 20
The invention relates to double stranded ribonucleic acid (dsRNA) compositions targeting a glucokinase (GCK) gene, as well as methods of inhibiting expression of a glucokinase (GCK) gene, and methods of treating subjects having a glycogen storage disease (GSD), e.g., type Ia GSD.
Kevin Fitzgerald, David Erbe, Gregory Hinkle
Filed: 20 Dec 17
Utility
MODIFIED RNAi AGENTS
11 Nov 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: 15 Apr 20
Utility
TMPRSS6 iRNA compositions and methods of use thereof
9 Nov 20
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, Martin A. Maier, Kevin Fitzgerald, Shannon Fishman, Donald Foster, Vasant R. Jadhav, Stuart Milstein
Filed: 6 Feb 19
Utility
SERUM AMYLOID P COMPONENT (APCS) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
4 Nov 20
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: 18 May 20
Utility
AMYLOID PRECURSOR PROTEIN (APP) RNAi AGENT COMPOSITIONS AND METHODS OF USE THEREOF
28 Oct 20
The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting the APP gene, as well as methods of inhibiting expression of an APP gene and methods of treating subjects having an APP-associated disease or disorder, such as cerebral amyloid angiopathy (CAA) and early onset familial Alzheimer disease (EOFAD or eFAD), using such dsRNAi agents and compositions.
Stuart Milstein, Kirk Brown, Jayaprakash Nair, Martin Maier, Vasant Jadhav, Mark Keating, Adam Castoreno, Patrick Haslett, Mangala Meenakshi Soundarapandian, Kevin Fitzgerald
Filed: 8 Jul 20
Utility
COMPLEMENT COMPONENT iRNA COMPOSITIONS AND METHODS OF USE THEREOF
28 Oct 20
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: 9 Jul 20
Utility
Assays and Methods for Determining Expression of the LECT2 Gene
28 Oct 20
The invention relates to methods and assays for determining the activity or expression of LECT2 gene in a subject.
Gregory Hinkle, Ho-Chou Tu, Alfica Sehgal
Filed: 7 Nov 18
Utility
Angiotensinogen (AGT) iRNA compositions and methods of use thereof
26 Oct 20
The present invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the angiotensinogen (AGT) gene, and methods of using such RNAi agents to inhibit expression of AGT and methods of treating subjects having an AGT-associated disorder, e.g., hypertension.
Donald Foster, Brian Bettencourt, Klaus Charisse, Gregory Hinkle, Satyanarayana Kuchimanchi, Martin A. Maier, Stuart Milstein
Filed: 12 Feb 19
Utility
Dual Targeting siRNA Agents
21 Oct 20
The invention relates to dual targeting siRNA agents targeting a PCSK9 gene and a second gene, and methods of using dual targeting siRNA agents to inhibit expression of PCSK9 and to treat PCSK9 related disorders, e.g., hyperlipidemia.
Kevin Fitzgerald, Maria Frank-Kamenetsky, Klaus Charisse
Filed: 26 Feb 20
Utility
Oligonucleotide-ligand conjugates and process for their preparation
19 Oct 20
Muthiah Manoharan, Jayaprakash K. Nair, Pachamuthu Kandasamy, Shigeo Matsuda, Alexander V. Kelin, Muthusamy Jayaraman, Kallanthottathil G. Rajeev
Filed: 10 Jul 14
Utility
Carbohydrate conjugates as delivery agents for oligonucleotides
19 Oct 20
Muthiah Manoharan, Kallanthottathil G. Rajeev, Jayaprakash Nair, Martin Maier
Filed: 19 Nov 18
Utility
Compositions and Methods for Inhibition of HAO1 (Hydroxyacid Oxidase 1 (Glycolate Oxidase)) Gene Expression
7 Oct 20
The invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the HAO1 gene, and methods of using such RNAi agents to inhibit expression of HAO1 and methods of treating subjects having, e.g., PH1.
William Querbes, Kevin Fitzgerald, Brian Bettencourt, Abigail Liebow, David V. Erbe
Filed: 3 Nov 19
Utility
Rnai Agents, Compositions and Methods of Use Thereof for Treating Transthyretin (TTR) Associated Diseases
7 Oct 20
The present invention provides RNAi agents, e.g., double stranded RNAi agents, that target the transthyretin (TTR) gene and methods of using such RNAi agents for treating or preventing TTR-associated diseases.
Kallanthottathil G. Rajeev, Tracy Zimmermann, Muthiah Manoharan, Martin A. Maier, Satyanarayana Kuchimanchi, Klaus Charisse
Filed: 8 Jan 20