354 patents
Page 15 of 18
Utility
Compositions and Methods for Inhibiting Expression of the PCSK9 Gene
7 Oct 20
The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the PCSK9 gene (PCSK9 gene), comprising an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19-25 nucleotides in length, and which is substantially complementary to at least a part of the PCSK9 gene.
Pamela Tan, Birgit Bramlage, Maria Frank-Kamenetsky, Kevin Fitzgerald, Akin Akinc, Victor E. Kotelianski
Filed: 4 Nov 19
Utility
High Mobility Group BOX-1 (HMGB1) Irna Compositions and Methods of Use Thereof
30 Sep 20
The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the HMGB 1 gene.
Gregory Hinkle, Frederic Tremblay, James D. McIninch
Filed: 17 Dec 18
Utility
Carbohydrate Conjugates As Delivery Agents for Oligonucleotides
23 Sep 20
Muthiah MANOHARAN, Kallanthottathil G. RAJEEV, Jayaprakash NAIR, Martin MAIER
Filed: 28 Apr 20
Utility
Xanthine Dehydrogenase (XDH) Irna Compositions and Methods of Use Thereof
23 Sep 20
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: 26 Jan 20
Utility
HEPATITIS D VIRUS (HDV) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
16 Sep 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 R. Jadhav, Martin A. Maier, Muthiah Manoharan, Stuart Milstein, Svetlana Shulga Morskaya, Kallanthottathil G. Rajeev, Huilei Xu
Filed: 25 Feb 20
Utility
Methods and Compositions for Detecting Anti-drug Antibodies
16 Sep 20
Assays, methods, reagents and kits for evaluating the level of an antibody against a nucleic acid molecule, e.g., a double-stranded oligonucleotide or RNA molecule (e.g., dsRNA), are disclosed herein.
Kallanthottathil G. Rajeev, Jayaprakash Nair, Muthiah Manoharan, Renta Hutabarat, Minggeng Gao
Filed: 27 Feb 20
Utility
Compositions and Methods for Inhibiting Expression of Transthyretin
9 Sep 20
The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting a transthyretin (TTR) gene, and methods of using the dsRNA to inhibit expression of TTR.
Dinah Wen-Yee Sah, Gregory Hinkle, Rene Alvarez, Stuart Milstein, Qingmin Chen
Filed: 22 Jan 20
Utility
Sterol regulatory element binding protein (SREBP) chaperone (SCAP) iRNA compositions and methods of use thereof
7 Sep 20
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: 6 Jun 18
Utility
INSULIN-LIKE GROWTH FACTOR BINDING PROTEIN, ACID LABILE SUBUNIT (IGFALS) AND INSULIN-LIKE GROWTH FACTOR 1 (IGF-1) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
2 Sep 20
The present invention relates to RNAi agents, e.g., double stranded RNAi agents, targeting the insulin-like growth factor binding protein, acid labile subunit (IGFALS) gene or the insulin-like growth factor 1 (IGF-1) gene, methods of using such double stranded RNAi agents to inhibit expression of an IGFALS gene or an IGF-1 gene, and methods of treating subjects having an IGF system-associated disorder.
James Butler, Kevin Fitzgerald, Gregory Hinkle, Brian Bettencourt, Huilei Xu
Filed: 14 Oct 19
Utility
Compositions and Methods for Inhibition of Expression of Apolipoprotein C-III (APOC3) Genes
19 Aug 20
The invention relates to double-stranded ribonucleic acid (dsRNA) targeting an APOC3 gene, and methods of using the dsRNA to inhibit expression of APOC3.
Brian Bettencourt, Kevin Fitzgerald, Stuart Milstein, Martin A. Maier, Klaus Charisse, Kallanthottathil Rajeev, Satyanarayana Kuchimanchi, Muthiah Manoharan, Tuyen Nguyen
Filed: 1 Dec 19
Utility
COMPLEMENT COMPONENT iRNA COMPOSITIONS AND METHODS OF USE THEREOF
19 Aug 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: 17 Sep 19
Utility
Compositions and methods for inhibiting expression of the LECT2 gene
17 Aug 20
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: 7 Apr 16
Utility
Compositions and methods for inhibiting expression of CD274/PD-L1 gene
17 Aug 20
The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the CD274/PD-L1 gene, and methods of using such dsRNA compositions to inhibit expression of CD274/PD-L1.
Antonin DeFougerolles, Tatiana Novobrantseva, Brian Bettencourt
Filed: 26 Feb 18
Utility
Therapeutic Compositions
12 Aug 20
This application relates to therapeutic siRNA agents and methods of making and using the agents.
Muthiah MANOHARAN, Kallanthottathil G. RAJEEV, David BUMCROT
Filed: 21 Apr 20
Utility
Polynucleotide Agents Targeting Patatin-like Phospholipase Domain Containing 3 (PNPLA3) and Methods of Use Thereof
5 Aug 20
The invention relates to polynucleotide agents targeting a Patatin-Like Phospholipase Domain Containing 3 (PNPLA3) gene, and methods of using such polynucleotide agents to inhibit expression of a PNPLA3 gene and methods of treating subjects having Nonalcoholic Fatty Liver Disease (NAFLD) and/or a PNPLA3-associated disorder.
Gregory Hinkle
Filed: 13 Feb 20
Utility
Lipid Formulated Compositions and Methods for Inhibiting Expression of Serum Amyloid a Gene
29 Jul 20
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: 2 Oct 19
Utility
Compositions and Methods for Inhibitiion of Expression of Protein C (Proc) Genes
29 Jul 20
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: 10 Nov 19
Utility
Compositions and Methods for Inhibiting Expression of Factor V
22 Jul 20
The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of Factor V, comprising an antisense strand having a nucleotide sequence which is less that 25 nucleotides in length and which is substantially complementary to at least a part of Factor V.
Hans-Peter Vornlocher, John M. Maraganore
Filed: 3 Nov 19
Utility
Angiopoietin-like 3 (ANGPTL3) Irna Compositions and Methods of Use Thereof
15 Jul 20
The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the ANGPTL3 gene, as well as methods of inhibiting epression 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: 11 Dec 19
Utility
Polynucleotide agents targeting angiotensinogen (AGT) and methods of use thereof
13 Jul 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: 24 May 16