354 patents
Page 6 of 18
Utility
Complement Component C3 Irna Compositions and Methods of Use Thereof
17 Nov 22
The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the complement component C3 gene (C3).
Mark Keating, James D. McIninch, Elane Fishilevich, Kristina Yucius, Sarah Solomon, Mark K. Schlegel, Adam Castoreno, Charalambos Kaittanis
Filed: 15 Apr 22
Utility
SUPEROXIDE DISMUTASE 1 (SOD1) iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING OR PREVENTING SUPEROXIDE DISMUTASE 1- (SOD1-) ASSOCIATED NEURODEGENERATIVE DISEASES
10 Nov 22
The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting a SOD1 gene, as well as methods of inhibiting expression of a SOD1 gene and methods of treating subjects having a SOD1-associated neurodegenerative disease or disorder, e.g., Amyotrophic Lateral Sclerosis (ALS), Alzheimer's disease (AD), Parkinson's disease (PD), and Down's syndrome (DS), using such dsRNAi agents and compositions.
Adam Castoreno, Jason Gilbert, Charalambos Kaittanis, James D. McIninch, Stuart Milstein, Mark K. Schlegel
Filed: 29 Jun 22
Utility
Hepatitis B virus (HBV) dsRNA agent compositions and methods of use thereof
8 Nov 22
The present disclosure relates to double stranded RNA agents targeting the hepatitis B virus (HBV) genome, and methods of using such agents to inhibit expression of one or more HBV genes and methods of treating subjects having an HBV infection or HBV-associated disorder, e.g., chronic hepatitis B infection.
Vasant R. Jadhav, Martin A. Maier, Stuart Milstein, Mark K. Schlegel
Filed: 12 Aug 19
Utility
Biodegradable Lipids for the Delivery of Active Agents
3 Nov 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
Oligonucleotide-ligand Conjugates and Process for Their Preparation
3 Nov 22
The present invention relates to ligand conjugates of oligonucleotides (e.g., iRNA agents) and methods for their preparation.
Muthiah Manoharan, Jayaprakash K. Nair, Pachamuthu Kandasamy, Shigeo Matsuda, Alexander V. Kelin, Muthusamy Jayaraman, Kallanthottathil G. Rajeev
Filed: 2 Oct 20
Utility
GNAQ Targeted dsRNA Compositions and Methods for Inhibiting Expression
3 Nov 22
The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting a G-alpha q subunit (GNAQ) of a heterotrimeric G gene, and methods of using the dsRNA to inhibit expression of GNAQ.
Jared Gollob, Gregory Hinkle, Ivanka Toudjarska, David Bumcrot
Filed: 9 Feb 21
Utility
PATATIN-LIKE PHOSPHOLIPASE DOMAIN CONTAINING 3 (PNPLA3) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
3 Nov 22
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: 27 Apr 21
Utility
MODIFIED RNAi AGENTS
20 Oct 22
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: 24 Jun 22
Utility
TRANSTHYRETIN (TTR) iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING OR PREVENTING TTR-ASSOCIATED OCULAR DISEASES
20 Oct 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 ocular diseases.
Jayaprakash K. Nair, Martin A. Maier, Vasant R. Jadhav, Mark Keating, Kevin Fitzgerald, Stuart Milstein, Kirk Brown, Muthiah Manoharan
Filed: 26 Mar 21
Utility
Delivery of Oligonucleotides to the Striatum
29 Sep 22
One aspect of the present invention relates to a double stranded iRNA agent comprising an antisense strand which is complementary to a target gene; a sense strand which is complementary to said antisense strand; and one or more lipophilic moieties conjugated to one or more internal positions on at least one strand, optionally via a linker or carrier, which provides for targeting to, and uptake by, tissues and cells of the CNS, and in particular the striatum.
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: 16 Jun 20
Utility
SERUM AMYLOID P COMPONENT (APCS) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
29 Sep 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: 8 Dec 21
Utility
Assays and Methods for Determining Activity of a Therapeutic Agent In a Subject
29 Sep 22
The invention relates to methods and assays for determining the activity of a composition comprising a therapeutic gene administered to a subject.
Dinah Sah, David Bumcrot, Alfica Sehgal
Filed: 14 Feb 22
Utility
Compositions and methods for inhibition of HAO1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression
20 Sep 22
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: 4 Nov 19
Utility
Compositions and Methods for Inhibiting Expression of the LECT2 Gene
15 Sep 22
The disclosure relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the LECT2 gene, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of LECT2.
HO-CHOU TU, JAMES MCININCH
Filed: 2 Sep 20
Utility
Modified RNA Agents with Reduced Off-Target Effect
15 Sep 22
One aspect of the present invention relates to double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene.
Shigeo MATSUDA, Mark K. SCHLEGEL, Maja JANAS, Vasant R. JADHAV, Martin MAIER, Klaus CHARISSE, Muthiah MANOHARAN, Kallathotathil G. RAJEEV, Jayaprakash K. NAIR
Filed: 13 May 22
Utility
Angiopoietin-like 3 (ANGPTL3) Irna Compositions and Methods of Use Thereof
15 Sep 22
The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the Angiopoietin-like 3 (ANGPTL3) gene.
Lucas D. BonDurant, Mark K. Schlegel, Jeffrey Zuber, Lauren Blair Woods, Tyler Chickering
Filed: 8 Mar 22
Utility
Sterol regulatory element binding protein (SREBP) chaperone (SCAP) iRNA compositions and methods of use thereof
6 Sep 22
The invention relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting the SCAP gene, as well as methods of inhibiting expression of a SCAP gene and methods of treating subjects having a SCAP-associated disorder, such as nonalcoholic fatty liver disease (NAFLD) or nonalcoholic steatohepatitis (NASH), using such dsRNAi agents and compositions.
Kevin Fitzgerald, Huilei Xu, Gregory Hinkle
Filed: 27 Jul 20
Utility
Modified double-stranded RNA agents
30 Aug 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: 10 Nov 21
Utility
METHODS FOR TREATING OR PREVENTING CONTACT-ACTIVATION PATHWAY-ASSOCIATED DISEASES USING iRNA COMPOSITIONS TARGETING FACTOR XII (HAGEMAN FACTOR) (F12)
25 Aug 22
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: 28 Sep 21
Utility
Angiotensinogen (AGT) iRNA compositions and methods of use thereof
23 Aug 22
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: 15 Sep 20