Abstract: 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.
Type:
Application
Filed:
April 7, 2023
Publication date:
May 9, 2024
Applicant:
Alnylam Pharmaceuticals, Inc.
Inventors:
Adam Castoreno, Jason Gilbert, Charalambos Kaittanis, James D. McIninch, Stuart Milstein, Mark K. Schlegel
Abstract: The present invention relates to RNAi agents, e.g., dsRNA agents, targeting the complement factor B (CFB) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a CFB gene and to methods of treating or preventing a CFB-associated disease in a subject.
Type:
Grant
Filed:
March 22, 2023
Date of Patent:
April 23, 2024
Assignee:
Alnylam Pharmaceuticals, Inc.
Inventors:
James D. McIninch, Mark K. Schlegel, Adam Castoreno, Elane Fishilevich, Kristina Yucius
Abstract: The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the transthyretin (TTR) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of an TTR gene and to methods of preventing and treating an TTR-associated disorder, e.g., senile systemic amyloidosis (SSA), systemic familial amyloidosis, familial amyloidotic polyneuropathy (FAP), familial amyloidotic cardiomyopathy (FAC), leptomeningeal/Central Nervous System (CNS) amyloidosis, and hyperthyroxinemia.
Type:
Grant
Filed:
September 28, 2022
Date of Patent:
April 16, 2024
Assignee:
Alnylam Pharmaceuticals, Inc.
Inventors:
Mark K. Schlegel, Adam Castoreno, James D. McIninch
Abstract: One aspect of the present invention relates to double-stranded RNA (dsRNA) molecules comprising a 6-methyladenine nucleobase and capable of inhibiting the expression of a target gene. Other aspects of the invention relate to pharmaceutical compositions comprising these dsRNA molecules suitable for therapeutic use, and methods of inhibiting the expression of a target gene by administering these dsRNA molecules, e.g., for the treatment of various disease conditions.
Abstract: This disclosure relates to an improved process for the preparation of carbohydrate conjugates. The disclosure also relates to carbohydrate conjugated iRNA agents comprising these carbohydrate conjugates, which have improved purity and are advantageous for the in vivo delivery of the iRNA agents.
Type:
Grant
Filed:
July 5, 2022
Date of Patent:
April 2, 2024
Assignee:
ALNYLAM PHARMACEUTICALS, INC.
Inventors:
Jayaprakash K. Nair, Alexander V Kel'in, Pachamuthu Kandasamy, Kallanthottathil G Rajeev, Muthiah Manoharan
Abstract: The present invention relates to RNAi agents, e.g., dsRNA agents, targeting the ketohexokinase (KHK) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a KHK gene and to methods of treating or preventing a KHK-associated disorder in a subject.
Type:
Grant
Filed:
December 2, 2022
Date of Patent:
March 12, 2024
Assignee:
Alnylam Pharmaceuticals, Inc.
Inventors:
Leila Noetzli, James D. McIninch, Frederic Tremblay, Mark K. Schlegel, Adam Castoreno
Abstract: 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.
Type:
Grant
Filed:
September 14, 2021
Date of Patent:
January 30, 2024
Assignee:
Alnylam Pharmaceuticals, Inc.
Inventors:
Antonin de Fougerolles, Tatiana Novobrantseva, Gregory Hinkle
Abstract: 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.
Type:
Grant
Filed:
July 12, 2021
Date of Patent:
January 16, 2024
Assignee:
Alnylam Pharmaceuticals, Inc.
Inventors:
Kevin Fitzgerald, James Butler, Brian Bettencourt, Anna Borodovsky, Satyanarayana Kuchimanchi, Klaus Charisse, Muthiah Manoharan, Martin A. Maier, Kallanthottathil G. Rajeev, Donald Foster
Abstract: 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.
Type:
Grant
Filed:
November 22, 2021
Date of Patent:
January 9, 2024
Assignee:
Alnylam Pharmaceuticals, Inc.
Inventors:
Brian Bettencourt, William Querbes, Kevin Fitzgerald, Maria Frank-Kamenetsky, Stuart Milstein, Svetlana Shulga Morskaya
Abstract: 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.
Abstract: The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the Transmembrane protease, serine 6 (TMPRSS6) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a TMPRSS6 gene and to methods of preventing and treating a TMPRSS6-associated disorder, e.g., a disorder associated with iron overload and/or a disorder of ineffective erythropoiesis, e.g., hereditary hemochromatosis, ?-thalassemia (e.g., ?-thalassemia major and ?-thalassemia intermedia), polycythemia vera, myelodysplastic syndrome, congenital dyserythropoietic anemias, pyruvate kinase deficiency, erythropoietic porphyria, Parkinson's Disease, Alzheimer's Disease or Friedreich's Ataxia.
Type:
Grant
Filed:
January 6, 2023
Date of Patent:
January 9, 2024
Assignee:
Alnylam Pharmaceuticals, Inc.
Inventors:
Aimee M. Deaton, John Michael Gansner, James D. McIninch, Mark K. Schlegel, Benjamin P. Garfinkel
Abstract: 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.
Type:
Grant
Filed:
October 14, 2020
Date of Patent:
January 2, 2024
Assignee:
Alnylam Pharmaceuticals, Inc.
Inventors:
Kevin Fitzgerald, David Erbe, Gregory Hinkle
Abstract: 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.
Type:
Grant
Filed:
November 22, 2021
Date of Patent:
December 12, 2023
Assignee:
Alnylam Pharmaceuticals, Inc.
Inventors:
Brian Bettencourt, William Querbes, Kevin Fitzgerald, Maria Frank-Kamenetsky, Stuart Milstein, Svetlana Shulga Morskaya
Abstract: The present invention relates to RNAi agents, e.g., dsRNA agents, targeting the G-protein coupled receptor 75 (GPR75) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a GPR75 gene and to methods of treating or preventing a GPR75-associated disease, such as a body weight disorder, e.g., obesity, in a subject.
Type:
Application
Filed:
April 3, 2023
Publication date:
December 7, 2023
Applicants:
Alnylam Pharmaceuticals, Inc., Regeneron Pharmaceuticals, Inc.
Inventors:
James D. McIninch, Bret Lee Bostwick, Adam Castoreno
Abstract: The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the AGT gene. The invention also relates to methods of using such RNAi agents to inhibit expression of an AGT gene and to methods of preventing and treating an AGT-associated disorder, e.g., high blood pressure.
Type:
Grant
Filed:
April 7, 2021
Date of Patent:
December 5, 2023
Assignee:
Alnylam Pharmaceuticals, Inc.
Inventors:
Donald Foster, Gregory Hinkle, Mark K. Schlegel
Abstract: 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.
Type:
Grant
Filed:
November 22, 2021
Date of Patent:
December 5, 2023
Assignee:
Alnylam Pharmaceuticals, Inc.
Inventors:
Brian Bettencourt, William Querbes, Kevin Fitzgerald, Maria Frank-Kamenetsky, Stuart Milstein, Svetlana Shulga Morskaya
Abstract: 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.
Abstract: 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.
Abstract: The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting an anaplastic lymphoma kinase (ALK) gene, as well as methods of inhibiting expression of an ALK gene and methods of treating subjects having an ALK-associated disease or disorder, e.g., type 2 diabetes, obesity, or an obesity-associated disorder, using such dsRNAi agents and compositions.
Abstract: The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the GRB 10 or GRB 14 gene, as well as methods of inhibiting expression of GRB 10 or GRB 14, and methods of treating subjects that would benefit from reduction in expression of GRB 10 or GRB 14, such as subjects having a GRB 10- or GRB 14-associated disease, disorder, or condition, such as diabetes, using such dsRNA compositions.
Type:
Application
Filed:
September 9, 2021
Publication date:
October 19, 2023
Applicant:
ALNYLAM PHARMACEUTICALS, INC.
Inventors:
JAMES D. MCININCH, AIMEE DEATON, LUCAS BONDURANT, MARK K. SCHLEGEL, ADAM CASTORENO