118 patents
Utility
Complement component C5 iRNA compositions and methods of use thereof
16 Jan 24
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.
Kevin Fitzgerald, James Butler, Brian Bettencourt, Anna Borodovsky, Satyanarayana Kuchimanchi, Klaus Charisse, Muthiah Manoharan, Martin A. Maier, Kallanthottathil G. Rajeev, Donald Foster
Filed: 12 Jul 21
Utility
Complement component C3 iRNA compositions and methods of use thereof
9 Jan 24
The invention relates to iRNA, e.g., double stranded ribonucleic acid (dsRNA), compositions targeting the complement factor C3 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of a C3 gene and to treat subjects having a complement component C3-associated disease, e.g., paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), atypical hemolytic uremic syndrome (aHUS), neuromyelitis optica (NMO), multifocal motor neuropathy (MMN), myasthenia gravis (MG), and C3 glomerulonephritis.
Gregory Hinkle, Anna Borodovsky
Filed: 1 Nov 18
Utility
Angiopoietin-like 3 (ANGPTL3) iRNA compositions and methods of use thereof
9 Jan 24
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: 22 Nov 21
Utility
Transmembrane protease, serine 6 (TMPRSS6) iRNA compositions and methods of use thereof
9 Jan 24
The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the Transmembrane protease, serine 6 (TMPRSS6) gene.
Aimee M. Deaton, John Michael Gansner, James D. McIninch, Mark K. Schlegel, Benjamin P. Garfinkel
Filed: 6 Jan 23
Utility
Glucokinase (GCK) iRNA compositions and methods of use thereof
2 Jan 24
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
Angiopoietin-like 3 (ANGPTL3) iRNA compositions and methods of use thereof
12 Dec 23
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: 22 Nov 21
Utility
Angiotensinogen (AGT) iRNA compositions and methods of use thereof
5 Dec 23
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: 7 Apr 21
Utility
Angiopoietin-like 3 (ANGPTL3) iRNA compositions and methods of use thereof
5 Dec 23
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: 22 Nov 21
Utility
Dosages and methods for delivering lipid formulated nucleic acid molecules
28 Nov 23
Methods, kits and devices for dosing a subject to reduce a hypersensitivy response to a lipid-formulated nucleic acid (e.g., RNA) molecule are disclosed.
Pia Kasperkovitz, Jared Gollob
Filed: 8 Apr 21
Utility
Compositions and methods for treating transthyretin (TTR) mediated amyloidosis
7 Nov 23
Disclosed herein are methods for treating hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in a human patient in need thereof by administering an effective amount of a transthyretin (TTR)-inhibiting composition.
Jared Gollob
Filed: 19 Sep 18
Utility
Serpina1 iRNA compositions and methods of use thereof
15 Aug 23
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: 8 Mar 21
Utility
TMPRSS6 iRNA compositions and methods of use thereof
15 Aug 23
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
Programmed cell death 1 ligand 1 (PD-L1) iRNA compositions and methods of use thereof
27 Jun 23
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
Superoxide dismutase 1 (SOD1) iRNA compositions and methods of use thereof for treating or preventing superoxide dismutase 1-(SOD1-) associated neurodegenerative diseases
16 May 23
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
Biodegradable lipids for the delivery of active agents
25 Apr 23
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: 14 Feb 22
Utility
Biodegradable lipids for the delivery of active agents
25 Apr 23
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: 14 Feb 22
Utility
Biodegradable lipids for the delivery of active agents
28 Mar 23
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: 14 Feb 22
Utility
Angiopoietin-like 3 (ANGPTL3) iRNA compositions and methods of use thereof
28 Mar 23
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
Chirally-enriched double-stranded RNA agents
7 Mar 23
The present invention relates to a chirally-modified dsRNA agent capable of inhibiting the expression of a target gene.
Muthiah Manoharan, Nate Taneja, Hartmut Ingo Jahns, Shigeo Matsuda, Klaus Charisse, Guo He, Jayaprakash K. Nair, Christopher Brown, Mark K. Schlegel, Vasant Jadhav, Martin Maier
Filed: 21 Dec 18
Utility
Biodegradable lipids for the delivery of active agents
28 Feb 23
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: 14 Feb 22