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
Abstract: The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the TRAF6 gene, as well as methods of inhibiting expression of TRAF6, and methods of treating subjects that would benefit from reduction in expression of TRAF6, such as subjects having a TRAF6-associated disease, disorder, or condition, using such dsRNA compositions.
Type:
Application
Filed:
June 8, 2021
Publication date:
October 12, 2023
Applicant:
ALNYLAM PHARMACEUTICALS, INC.
Inventors:
ARLIN ROGERS, MELISSA MOBLEY, JAMES D. MCININCH
Abstract: The disclosure relates to double-stranded ribonucleic acid (dsRNA) compositions targeting MYOC, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of MYOC.
Abstract: The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the G protein-coupled receptor 146 (GPR146). The invention also relates to methods of using such RNAi agents to inhibit expression of a GPR146 gene and to methods of preventing and treating a GPR146-associated disorders, e.g., hypercholesterolemia and atherosclerosis.
Type:
Application
Filed:
February 26, 2021
Publication date:
September 14, 2023
Applicant:
ALNYLAM PHARMACEUTICALS, INC.
Inventors:
KEVIN FITZGERALD, FREDERIC TREMBLAY, JAMES D. MCININCH
Abstract: One aspect of the present invention relates to double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene. The antisense strand of the dsRNA molecule comprises at least one thermally destabilizing nucleotide occurring at a seed region; the dsRNA comprises at least four 2?-fluoro modifications, and the sense strand of the dsRNA molecule comprises ligand, wherein the ligand is an ASGPR ligand. 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.
Type:
Application
Filed:
September 30, 2022
Publication date:
August 17, 2023
Applicant:
ALNYLAM PHARMACEUTICALS, INC.
Inventors:
Mark K. SCHLEGEL, Maja JANAS, Vasant R. JADHAV, Muthiah MANOHARAN, Kallanthottathil G. RAJEEV, Muthusamy JAYARAMAN, Alexander V. KEL'IN, Shigeo MATSUDA, Klaus CHARISSE, Jayaprakash K. NAIR, Martin A. MAIER, Alfica SEHGAL, Christopher BROWN, Christopher THEILE, Stuart MILSTEIN
Abstract: 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.
Type:
Grant
Filed:
March 8, 2021
Date of Patent:
August 15, 2023
Assignee:
Alnylam Pharmaceuticals, Inc.
Inventors:
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
Abstract: 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 ?-thalassemia or hemochromatosis.
Type:
Grant
Filed:
October 5, 2020
Date of Patent:
August 15, 2023
Assignee:
Alnylam Pharmaceuticals, Inc.
Inventors:
James Butler, Martin A. Maier, Kevin Fitzgerald, Shannon Fishman, Donald Foster, Vasant R. Jadhav, Stuart Milstein
Abstract: The disclosure relates to double stranded ribonucleic acid interference (dsRNAi) agents and compositions targeting a microtubule-associated protein tau (MAPT) gene, as well as methods of inhibiting expression of a MAPT gene and methods of treating subjects having a MAPT-associated disease or disorder, e.g., Alzheimer's disease, frontotemporal dementia, progressive supranuclear palsy, or other tauopathies, using such dsRNAi agents and compositions.
Type:
Application
Filed:
March 30, 2021
Publication date:
June 29, 2023
Applicant:
ALNYLAM PHARMACEUTICALS, INC.
Inventors:
MANGALA MEENAKSHI SOUNDARAPANDIAN, JAMES D. MCININCH, ELANE FISHILEVICH, ADAM CASTORENO, CHARALAMBOS KAITTANIS, MARK K. SCHLEGEL, JONATHAN EDWARD FARLEY, JEFFREY ZUBER
Abstract: The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the GPAM gene, as well as methods of inhibiting expression of GPAM, and methods of treating subjects that would benefit from reduction in expression of GPAM, such as subjects having a GPAM-associated disease, disorder, or condition, using such dsRNA compositions.
Type:
Application
Filed:
June 9, 2021
Publication date:
June 29, 2023
Applicant:
ALNYLAM PHARMACEUTICALS, INC.
Inventors:
JAMES D. MCININCH, LUCAS BONDURANT, AIMEE DEATON
Abstract: 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.
Abstract: The disclosure relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the DNAJC15 gene, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of DNAJC15.
Abstract: 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. These cationic lipids may be incorporated into a lipid particle for delivering an active agent, such as a nucleic acid. The invention also relates to lipid particles comprising a neutral lipid, a lipid capable of reducing aggregation, a cationic lipid of the present invention, and optionally, a sterol. The lipid particle may further include a therapeutic agent such as a nucleic acid.
Type:
Grant
Filed:
December 17, 2021
Date of Patent:
June 20, 2023
Assignee:
ALNYLAM PHARMACEUTICALS, INC.
Inventors:
Martin Maier, Muthusamy Jayaraman, Akin Akinc, Shigeo Matsuda, Pachamuthu Kandasamy, Kallanthottathil G. Rajeev, Muthiah Manoharan
Abstract: The disclosure relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the MARC1 gene, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of MARC1.
Abstract: The present invention provides a modified RNAi agent comprising a sense strand and an antisense strand, wherein the antisense strand targets an HBV mRNA; all of the nucleotide sugars of the RNAi agent are 2?-modified; and at least one of the sense and antisense strands is conjugated to at least one targeting ligand that comprises two or more building blocks connected to a biantennary or triantennary branched linker, optionally via one or more linking groups.
Type:
Grant
Filed:
November 23, 2021
Date of Patent:
June 6, 2023
Assignee:
ALNYLAM PHARMACEUTICALS, INC.
Inventors:
Muthiah Manoharan, Kallanthottathil G. Rajeev, Jayaprakash K. Nair, Martin Maier
Abstract: The disclosure relates to double-stranded ribonucleic acid (dsRNA) compositions targeting SCN9A, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of SCN9A.
Type:
Application
Filed:
April 6, 2021
Publication date:
May 25, 2023
Applicant:
ALNYLAM PHARMACEUTICALS, INC.
Inventors:
WILLIAM CANTLEY, JAMES D. MCININCH, ADAM CASTORENO, CHARALAMBOS KAITTANIS, MARK K. SCHLEGEL
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:
Grant
Filed:
June 29, 2022
Date of Patent:
May 16, 2023
Assignee:
Alnylam Pharmaceuticals, Inc.
Inventors:
Adam Castoreno, Jason Gilbert, Charalambos Kaittanis, James D. McIninch, Stuart Milstein, Mark K. Schlegel
Abstract: The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting an alpha-2A adrenergic receptor (ADRA2A) gene, as well as methods of inhibiting expression of an ADRA2A gene and methods of treating subjects having an ADRA2A-associated disease or disorder, e.g., a primary tauopathy or Alzheimer's disease, using such dsRNAi agents and compositions.
Type:
Application
Filed:
April 1, 2021
Publication date:
May 4, 2023
Applicant:
ALNYLAM PHARMACEUTICALS, INC.
Inventors:
BRET L. BOSTWICK, JAMES D. MCININCH, ADAM CASTORENO
Abstract: The disclosure relates to double-stranded ribonucleic acid (dsRNA) compositions targeting VEGF-A, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of VEGF-A.
Type:
Application
Filed:
February 9, 2021
Publication date:
May 4, 2023
Applicant:
ALNYLAM PHARMACEUTICALS, INC.
Inventors:
JAMES D. MCININCH, MARK KEATING, MARK K. SCHLEGEL, ADAM CASTORENO, VASANT R. JADHAV, CHARALAMBOS KAITTANIS, ELENA CASTELLANOS-RIZALDOS, BHAUMIK A. PANDYA