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：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：US20240209372A1

公开(公告)日：2024-06-27

申请号：US18373397

申请日：2023-09-27

Applicant: Alnylam Pharmaceuticals, Inc.

Inventor： David Erbe ,  James D. McIninch

IPC: C12N15/113 ,  A61K47/54

CPC classification number: C12N15/1137 ,  A61K47/549 ,  C12N2310/11 ,  C12N2310/3125 ,  C12N2310/315 ,  C12N2310/351

Abstract: The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the proline dehydrogenase 2 (PRODH2) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a PRODH2 gene and to methods of preventing and treating a PRODH2-associated disorder, e.g., primary hyperoxalurias and/or kidney stone diseases.

公开(公告)号：US11859185B2

公开(公告)日：2024-01-02

申请号：US17069907

申请日：2020-10-14

Applicant: Alnylam Pharmaceuticals, Inc.

Inventor： Kevin Fitzgerald ,  David Erbe ,  Gregory Hinkle

IPC: C07H21/04 ,  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：US20230183706A1

公开(公告)日：2023-06-15

申请号：US17945151

申请日：2022-09-15

Applicant: Alnylam Pharmaceuticals, Inc.

Inventor： Aimee M. Deaton ,  David Erbe

IPC: C12N15/113 ,  C12N9/10 ,  A61P13/04

CPC classification number: C12N15/1137 ,  C12Y101/01027 ,  C12Y101/03015 ,  C12N9/1096 ,  C12Y206/01044 ,  A61P13/04 ,  C12N2310/11 ,  C12N2310/315

Abstract: The present invention provides methods for treating subjects suffering from a kidney stone disease carrying a heterozygous AGXT gene variant, methods for identifying such subjects, and compositions comprising nucleic acid inhibitors, e.g., double stranded ribonucleic acid (dsRNA) agents or single stranded antisense polynucleotide agents targeting lactate dehydrogenase A (LDHA) and/or hydroxyacid oxidase (HAO1), for treating such subjects.

公开(公告)号：US20210171956A1

公开(公告)日：2021-06-10

申请号：US17069907

申请日：2020-10-14

Applicant: Alnylam Pharmaceuticals, Inc.

Inventor： Kevin Fitzgerald ,  David Erbe ,  Gregory Hinkle

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

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：US20240344070A1

公开(公告)日：2024-10-17

申请号：US18403862

申请日：2024-01-04

Applicant: Alnylam Pharmaceuticals, Inc.

Inventor： John M. Gansner ,  David Erbe

IPC: C12N15/113

CPC classification number: C12N15/1137 ,  C12Y101/03015 ,  C12N2310/14 ,  C12N2310/351

Abstract: The present invention provides methods for treating subjects having or at risk of developing a non-primary hyperoxaluria disease or disorder that would benefit from reduction in oxalate, and compositions comprising nucleic acid inhibitors, e.g., double stranded ribonucleic acid (dsRNA) agents or single stranded antisense polynucleotide agents targeting lactate dehydrogenase A (LDHA), hydroxyacid oxidase (HAO1) and/or proline dehydrogenase 2 (PRODH2), for treating such subjects.

公开(公告)号：US20240309375A1

公开(公告)日：2024-09-19

申请号：US18531914

申请日：2023-12-07

Applicant: Alnylam Pharmaceuticals, Inc.

Inventor： Jared A. Gollob ,  Kevin Fitzgerald ,  David Erbe ,  Simina Ticau ,  Jeffrey Zuber

IPC: C12N15/113 ,  A61P27/02

CPC classification number: C12N15/113 ,  A61P27/02 ,  C12N2310/11 ,  C12N2310/351

Abstract: The present invention relates to agents which inhibit the expression and/or activity of transthyretin (TTR), e.g., a double stranded RNA (dsRNA) agent, or salt thereof, or an antisense oligonucleotide or a gene therapy targeting TTR, and the use of these agents in methods of treating or preventing Startgardt's disease, methods of decreasing Vitamin A levels or formation of toxic Vitamin A metabolites, and/or methods of halting progression of vision loss in a subject.
The present invention also relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the retinal binding protein 4 (RBP4) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of an RBP4 gene. The invention further provides the use of RNAi agent targeting RBP4 and/or nucleic acid agents targeting TTR in methods of preventing and treating an RBP4-associated disorder, e.g., an ocular disease, e.g., Stargardt's disease, diabetic retinopathy, age-related macular degeneration (AMD), e.g., dry AMD and wet AMD; or a metabolic disorder, e.g., a disorder of glucose and lipid homeostasis, e.g., insulin resistance associated with type II diabetes, or a cardiovascular disease.

公开(公告)号：US20210228614A1

公开(公告)日：2021-07-29

申请号：US17106259

申请日：2020-11-30

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：US20200206258A1

公开(公告)日：2020-07-02

申请号：US16811476

申请日：2020-03-06

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.