354 patents
Page 5 of 18
Utility
Rnai Agents, Compositions and Methods of Use Thereof for Treating Transthyretin (TTR) Associated Diseases
12 Jan 23
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: 9 Jul 21
Utility
Modified double-stranded RNA agents
10 Jan 23
One aspect of the present invention relates to double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene.
Martin Maier, Don Foster, Stuart Milstein, Satya Kuchimanchi, Vasant Jadhav, Kallanthottathil Rajeev, Muthiah Manoharan, Rubina Parmar
Filed: 23 Jun 22
Utility
Methods and Compositions for Treating an Angiotensinogen- (Agt-) Associated Disorder
5 Jan 23
The present invention relates to methods of inhibiting the expression of an AGT gene in a subject, as well as methods for treating subjects having an AGT-associated disorder, e.g., hypertension, using RNAi agents, e.g., double stranded RNAi agents, targeting the AGT gene.
Donald Foster, Sagar Agarwal, Stephen Albert Huang, Jae Kim
Filed: 13 May 22
Utility
PCSK9 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
5 Jan 23
The invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the PCSK9 gene, and methods of using such RNAi agents to inhibit expression of PCSK9 and methods of treating subjects having a lipid disorder, such as a hyperlipidemia.
Anna Borodovsky, Kallanthottathil G. Rajeev, Kevin Fitzgerald, Maria Frank-Kamenetsky, William Querbes, Martin A. Maier, Klaus Charisse, Satyanarayana Kuchimanchi, Muthiah Manoharan, Stuart Milstein
Filed: 28 Jan 22
Utility
XANTHINE DEHYDROGENASE (XDH) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
29 Dec 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: 1 Mar 22
Utility
MODIFIED iRNA AGENTS
22 Dec 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The invention relates to iRNA agents, which preferably include a monomer in which the ribose moiety has been replaced by a moiety other than ribose.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: 17 Mar 22
Utility
Complement Component C5 Irna Compositions and Methods of Use Thereof
22 Dec 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.
Anna Borodovsky, Andrew Sprague
Filed: 12 Jan 21
Utility
Compositions and Methods for Inhibiting Expression of the ALAS1 Gene
22 Dec 22
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: 8 Feb 21
Utility
Methods for Inhibition of HAO1 (Hydroxyacid Oxidase 1 (Glycolate Oxidase)) Gene Expression
22 Dec 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: 20 Jan 22
Utility
Therapeutic compositions
20 Dec 22
This application relates to therapeutic siRNA agents and methods of making and using the agents.
Muthiah Manoharan, Kallanthottathil G. Rajeev, David Bumcrot
Filed: 22 Apr 20
Utility
Angiopoietin-like 3 (ANGPTL3) iRNA compositions and methods of use thereof
13 Dec 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: 6 Apr 22
Utility
Modified Oligonucleotides
8 Dec 22
One aspect of the present invention relates to double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene.
Muthiah MANOHARAN, Pawan KUMAR, Dhrubajyoti DATTA
Filed: 13 Oct 20
Utility
Modified Double-stranded Rna Agents
8 Dec 22
One aspect of the present invention relates to double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene.
Martin MAIER, Don FOSTER, Stuart MILSTEIN, Satya KUCHIMANCHI, Vasant JADHAV, Kallanthottathil RAJEEV, Muthiah MANOHARAN, Rubina PARMAR
Filed: 23 Jun 22
Utility
Compositions and Methods for Silencing UGT1A1 Gene Expression
8 Dec 22
The disclosure relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the UGT1a1 gene, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of UGT1a1.
DONALD FOSTER, JAMES MCININCH
Filed: 2 Oct 20
Utility
Carbohydrate Conjugate Rna Agents and Process for Their Preparation
1 Dec 22
This disclosure relates to an improved process for the preparation of carbohydrate conjugates.
Jayaprakash K. Nair, Alexander V. Kel'in, Pachamuthu Kandasamy, Kallanthottathil G. Rajeev, Muthiah Manoharan
Filed: 5 Jul 22
Utility
HUNTINGTIN (HTT) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
1 Dec 22
The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting a Huntingtin (HTT) gene, e.g., exon 1 of an HTT gene, as well as methods of inhibiting expression of an HTT gene and methods of treating subjects having an HTT-associated disease or disorder, e.g., Huntington's disease, using such dsRNAi agents and compositions.
Mangala Meenakshi Soundarapandian, James D. McIninch, Bret Lee Bostwick, Mark K. Schlegel, Adam Castoreno, Jeffrey Zuber
Filed: 28 Apr 22
Utility
Xanthine Dehydrogenase (XDH) Irna Compositions and Methods of Use Thereof
1 Dec 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: 31 Mar 22
Utility
Compositions and Methods for Inhibiting Expression of the LECT2 Gene
24 Nov 22
The invention relates to antisense polynucleotide agents targeting the LECT2 gene, and methods of using such antisense polynucleotide agents to inhibit expression of LECT2 and to treat subjects having a LECT2-associated disease, e.g., amyloidosis.
Gregory Hinkle
Filed: 14 Mar 22
Utility
Biodegradable Lipids for the Delivery of Active Agents
24 Nov 22
Muthusamy Jayaraman, Guo He, Martin Maier
Filed: 27 Sep 19
Utility
Modified RNA agents with reduced off-target effect
22 Nov 22
One aspect of the present invention relates to double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene.
Mark K. Schlegel, Maja Janas, Vasant R. Jadhav, Donald Foster, Muthiah Manoharan, Kallanthottathil G. Rajeev, Muthusamy Jayaraman, Alexander V. Kel'in, Shigeo Matsuda, Klaus Charisse, Jayaprakash K. Nair, Martin A. Maier, Alfica Sehgal, Christopher Brown, Christopher Theile, Stuart Milstein
Filed: 22 Nov 17