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ALNY Alnylam Pharmaceuticals

181 patents

Page 4 of 10
APP
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
APP
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
APP
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
GRANT
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The invention relates to iRNA agents, which preferably include a monomer in which the ribose moiety has been replaced by a moiety other than ribose that further includes a tether having one or more linking groups, in which at least one of the linking groups is a cleavable linking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
GRANT
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
GRANT
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
GRANT
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
GRANT
Utility
Polynucleotide agents targeting aminolevulinic acid synthase-1 (ALAS1) and uses thereof
6 Apr 21
The invention relates to polynucleotide agents, e.g., antisense polunucleotide agents, targeting the ALAS1 gene, and methods of using such agents to alter (e.g., inhibit) expression of ALAS1 and to treat ALAS1 associated diseases, e.g., porphyria.
Gregory Hinkle
Filed: 30 Mar 17
APP
Utility
Compositions and Methods for Inhibiting Expression of the LECT2 Gene
1 Apr 21
The invention relates to antisense polynucleotide agents targeting the LECT2 gene, and methods of using such anti sense polynucleotide agents to inhibit expression of LECT2 and to treat subjects having a LECT2-associated disease, e.g., amyloidosis.
Gregory Hinkle
Filed: 13 Jul 20
APP
Utility
ANGIOTENSINOGEN (AGT) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
1 Apr 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
APP
Utility
Compositions and Methods Directed to Treating Liver Fibrosis
25 Mar 21
The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the COL1A1, TGF-β, and SMAD2/3 genes, and methods of using such dsRNA compositions to inhibit expression of COL1A1, TGF-β, and SMAD2/3.
Gregory Hinkle, Victor Kotelianski, Brian Bettencourt, Alfica Sehgal, Tatiana Novobrantseva
Filed: 6 May 20
APP
Utility
Oligonucleotides Comprising Acyclic and Abasic Nucleosides and Analogs
25 Mar 21
Muthiah MANOHARAN, Kallanthottathil G. RAJEEV, Jeremy LACKEY, Jayaprakash K. NAIR
Filed: 3 Dec 20
APP
Utility
Compositions and Methods for Inhibiting Expression of the ALAS1 Gene
25 Mar 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: 4 Jun 20
GRANT
Utility
GNAQ targeted dsRNA compositions and methods for inhibiting expression
23 Mar 21
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: 3 Apr 18
GRANT
Utility
Polynucleotide agents targeting hydroxyacid oxidase (glycolate oxidase, HAO1) and methods of use thereof
16 Mar 21
The invention relates to polynucleotide agents targeting an hydroxyacid oxidase (HAO1) gene, and methods of using such polynucleotide agents to inhibit expression of HAO1 and to treat subjects having an HAO1-associated disease, e.g., hyperoxaluria.
Gregory Hinkle
Filed: 14 Dec 17
GRANT
Utility
Myostatin iRNA compositions and methods of use thereof
2 Mar 21
The present invention provides methods comprising the in vivo delivery of small nucleic acid molecules capable of mediating RNA interference and reducing the expression of myostatin, wherein the small nucleic acid molecules are introduced to a subject by systemic administration.
Vasant R. Jadhav, Rubina Parmar, Laura Sepp-Lorenzino, Muthiah Manoharan
Filed: 20 Sep 17
GRANT
Utility
Factor XII (hageman factor) (F12), kallikrein B, plasma (fletcher factor) 1 (KLKB1), and kininogen 1 (KNG1) iRNA compositions and methods of use thereof
2 Mar 21
The present invention relates to RNAi agents, e.g., double stranded RNAi agents, targeting the Kallikrein B, Plasma (Fletcher Factor) 1 (KLKB1) gene, the Factor XII (Hageman Factor (F12) gene, or the Kininogen 1 (KNG1) gene, and methods of using such RNAi agents to inhibit expression of a KLKB1 gene, an F12 gene, and/or a KNG1 gene, and methods of treating subjects having an hereditary angioedema (HAE) and/or a contact activation pathway-associated disorder.
Akin Akinc, Gregory Hinkle, Martin A. Maier, James Butler, Jingxuan Liu
Filed: 24 May 19
GRANT
Utility
Angiopoietin-like 3 (ANGPTL3) iRNA compositions and methods of use thereof
2 Mar 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: 14 May 19
GRANT
Utility
Serpina1 siRNAs: compositions of matter and methods of treatment
2 Mar 21
The technology described herein relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the Serpina 1 gene, and methods of using such dsRNA compositions to inhibit expression of Serpina 1.
Alfica Sehgal, David Bumcrot, Brian Bettencourt
Filed: 17 Jul 19
APP
Utility
ANGIOTENSINOGEN (AGT) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
18 Feb 21
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
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