354 patents
Page 12 of 18
Utility
Multi-targeted single entity conjugates
6 Jul 21
The present invention relates, in general to, compounds, compositions and methods useful for modulating gene expression of multiple target nucleic acids by a single chemical entity.
Vasant Jadhav, Martin Maier, Ivan Zlatev, Akin Akinc, Kallanthottathil G. Rajeev, Jayaprakash K. Nair, Pachamuthu Kandasamy, Muthiah Manoharan
Filed: 15 Jul 16
Utility
PROGRAMMED CELL DEATH 1 LIGAND 1 (PD-L1) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
24 Jun 21
The present invention relates to RNAi agents, e.g., double stranded RNAi agents, targeting the programmed cell death 1 ligand 1 (PD-L1) gene, and methods of using such RNAi agents to inhibit expression of a PD-L1 gene and methods of treating subjects having a PD-L1-associated disorder.
Gregory Hinkle
Filed: 6 Nov 20
Utility
Compositions and Methods for Inhibiting Expression of Factor V
24 Jun 21
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: 23 Feb 21
Utility
PATATIN-LIKE PHOSPHOLIPASE DOMAIN CONTAINING 3 (PNPLA3) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
24 Jun 21
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: 23 Feb 21
Utility
COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
17 Jun 21
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
Filed: 14 Oct 20
Utility
Amyloid precursor protein (APP) RNAi agent compositions and methods of use thereof
15 Jun 21
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: 9 Jul 20
Utility
Apolipoprotein C3 (APOC3) iRNA compositions and methods of use thereof
15 Jun 21
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: 21 Dec 20
Utility
Polynucleotide Agents Targeting Angiotensinogen (Agt) and Methods of Use Thereof
10 Jun 21
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: 3 Jun 20
Utility
COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
10 Jun 21
The invention relates to methods for treating subjects having a complement component C5-associated disease, e.g., paroxysmal nocturnal hemoglobinuria, using iRNA, e.g., double stranded ribonucleic acid (dsRNA), compositions targeting the complement component C5 gene, and anti-C5 antibodies, e.g., eculizumab.
Nader Najafian, Jae Kim, Mustafa Varoglu, Gabriel Robbie
Filed: 14 Oct 20
Utility
ANGIOPOIETIN-LIKE 3 (ANGPTL3) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
10 Jun 21
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: 5 Nov 20
Utility
ANGIOPOIETIN-LIKE 3 (ANGPTL3) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
10 Jun 21
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: 26 Jan 21
Utility
GLUCOKINASE (GCK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
10 Jun 21
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: 14 Oct 20
Utility
Compositions and methods for inhibiting expression of the ALAS1 gene
8 Jun 21
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: 20 Sep 18
Utility
Methods and Compositions for Treating a Proprotein Convertase Subtilisin Kexin (PCSK9) Gene-associated Disorder
3 Jun 21
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 Jul 20
Utility
TMPRSS6 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
3 Jun 21
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: 5 Oct 20
Utility
Compositions and Methods for Inhibiting Expression of CD274/PD-L1 Gene
27 May 21
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: 7 Jul 20
Utility
iRNA agents with biocleavable tethers
25 May 21
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 that further includes a tether having one or more linking groups, in which at least one of the linking groups is a cleavable linking group.
Muthiah Manoharan, Kallanthottathil G. Rajeev
Filed: 23 Jul 18
Utility
Angiotensinogen (AGT) iRNA compositions and methods of use thereof
25 May 21
The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the AGT gene.
Donald Foster, Gregory Hinkle, Mark K. Schlegel
Filed: 10 Nov 20
Utility
Serpina1 iRNA compositions and methods of use thereof
4 May 21
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.
Mark K. Schlegel, Maja Janas, Vasant R. Jadhav, Donald Foster, Muthiah Manoharan, Kallanthottathil G. Rajeev, Alexander V. Kel'in, Klaus Charisse, Jayaprakash K. Nair, Martin A. Maier, Shigeo Matsuda, Muthusamy Jayaraman, Alfica Sehgal, Christopher Brown, Kevin Fitzgerald, Stuart Milstein
Filed: 23 May 19
Utility
TMPRSS6 iRNA compositions and methods of use thereof
27 Apr 21
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: 18 Aug 20