公开(公告)号：US20250017953A1

公开(公告)日：2025-01-16

申请号：US18637500

申请日：2024-04-17

Applicant: Alnylam Pharmaceuticals, Inc. ,  The UAB Research Foundation

Inventor： David Erbe ,  Abigail Liebow ,  Kevin Fitzgerald ,  Gregory Hinkle ,  Kyle David Wood ,  Ross Philip Holmes ,  John Knight

IPC: A61K31/7105 ,  C12N15/113

Abstract: The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the LDHA gene, as well as methods of inhibiting expression of LDHA, methods of inhibiting LDHA and HAO1, and methods of treating subjects that would benefit from reduction in expression of LDHA, such as subjects having an oxalate pathway-associated disease, disorder, or condition, using such dsRNA compositions.

公开(公告)号：US20240294912A1

公开(公告)日：2024-09-05

申请号：US18519295

申请日：2023-11-27

Applicant: Alnylam Pharmaceuticals, Inc.

Inventor： Gregory Hinkle ,  Anna Borodovsky

IPC: C12N15/113 ,  A61K31/7115

CPC classification number: C12N15/113 ,  A61K31/7115 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3235 ,  C12N2310/351

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.

公开(公告)号：US20240287524A1

公开(公告)日：2024-08-29

申请号：US18509583

申请日：2023-11-15

Applicant: Alnylam Pharmaceuticals, Inc.

Inventor： Kevin Fitzgerald ,  David Erbe ,  Gregory Hinkle

IPC: C12N15/113 ,  A61K31/713 ,  A61K45/06

CPC classification number: C12N15/1137 ,  A61K31/713 ,  A61K45/06 ,  C12Y207/01002 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/351 ,  C12N2310/3521 ,  C12N2310/3533

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.

公开(公告)号：US20240200075A1

公开(公告)日：2024-06-20

申请号：US18337495

申请日：2023-06-20

Applicant: Alnylam Pharmaceuticals, Inc.

Inventor： William Querbes ,  Kevin Fitzgerald ,  James Butler ,  Stephanie Williams ,  Gregory Hinkle ,  Martin A. Maier

IPC: C12N15/113 ,  A61K31/713 ,  A61K47/54

CPC classification number: C12N15/1136 ,  A61K31/713 ,  A61K47/549 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/34 ,  C12N2310/344 ,  C12N2310/346 ,  C12N2310/351 ,  C12N2310/3515 ,  C12N2320/32 ,  C12N2320/35 ,  C12N2320/51

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.

公开(公告)号：US20240148773A1

公开(公告)日：2024-05-09

申请号：US18204441

申请日：2023-06-01

Applicant: Alnylam Pharmaceuticals, Inc.

Inventor： Kevin Fitzgerald ,  Gregory Hinkle

IPC: A61K31/7125 ,  C07H21/02 ,  C12N15/113 ,  C12P19/34

CPC classification number: A61K31/7125 ,  C07H21/02 ,  C12N15/113 ,  C12N15/1137 ,  C12P19/34 ,  C07H19/067 ,  C12N2310/14 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3515

Abstract: The present invention relates to RNAi agents, e.g., double stranded RNAi agents, targeting the Patatin-Like Phospholipase Domain Containing 3 (PNPLA3) gene, and methods of using such RNAi agents to inhibit expression of a PNPLA3 gene and methods of treating subjects having Nonalcoholic Fatty Liver Disease (NAFLD) and/or a PNPLA3-associated disorder.

公开(公告)号：US20230032974A1

公开(公告)日：2023-02-02

申请号：US17668413

申请日：2022-02-10

Applicant: Alnylam Pharmaceuticals, Inc.

Inventor： Bret Lee Bostwick ,  Mangala Meenakshi Soundarapandian ,  James D. McIninch ,  Gregory Hinkle

IPC: C12N15/113 ,  A61P21/00

Abstract: The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting an RPS25 gene, as well as methods of inhibiting expression of an RPS25 gene and methods of treating subjects having an RPS25-associated disease or disorder, such as a nucleotide repeat expansion disorder, e.g., c9orf72 amyotrophic lateral sclerosis (ALS)/frontotemporal demential (FTD) and Huntington-Like Syndrome Due To C9orf72 Expansions, using such dsRNAi agents and compositions.

公开(公告)号：US20220305045A1

公开(公告)日：2022-09-29

申请号：US17544984

申请日：2021-12-08

Applicant: Alnylam Pharmaceuticals, Inc.

Inventor： Kevin Fitzgerald ,  Gregory Hinkle

IPC: A61K31/713 ,  A61K47/54 ,  C12N15/113

Abstract: The invention relates to iRNA, e.g., double stranded ribonucleic acid (dsRNA), compositions targeting the serum amyloid P component (APCS) gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of an APCS gene and to treat subjects having an APCS-associated disease, e.g., amyloidosis, Alzheimer's disease or coronary atherosclerotic heart disease.

公开(公告)号：US20220213473A1

公开(公告)日：2022-07-07

申请号：US17392122

申请日：2021-08-02

Applicant: Alnylam Pharmaceuticals, Inc.

Inventor： Brian Bettencourt ,  Kevin Fitzgerald ,  Gregory Hinkle ,  Alfica Sehgal

IPC: C12N15/113

Abstract: The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the LECT2 gene, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of LECT2.

公开(公告)号：US11229663B2

公开(公告)日：2022-01-25

申请号：US16877586

申请日：2020-05-19

Applicant: Alnylam Pharmaceuticals, Inc.

Inventor： Kevin Fitzgerald ,  Gregory Hinkle

IPC: C12N15/11 ,  A61K31/713 ,  A61K47/54 ,  C12N15/113

Abstract: The invention relates to iRNA, e.g., double stranded ribonucleic acid (dsRNA), compositions targeting the serum amyloid P component (APCS) gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of an APCS gene and to treat subjects having an APCS-associated disease, e.g., amyloidosis, Alzheimer's disease or coronary atherosclerotic heart disease.

公开(公告)号：US20210317452A1

公开(公告)日：2021-10-14

申请号：US17141275

申请日：2021-01-05

Applicant: Alnylam Pharmaceuticals, Inc.

Inventor： Alfica Sehgal ,  Klaus Charisse ,  Brian Bettencourt ,  Martin A. Maier ,  Kallanthottathil G. Rajeev ,  Gregory Hinkle ,  Muthiah Manoharan

IPC: C12N15/113 ,  A61K31/712 ,  A61K31/713

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.