公开(公告)号：US20240360454A1

公开(公告)日：2024-10-31

申请号：US18623281

申请日：2024-04-01

Applicant: Alnylam Pharmaceuticals, Inc.

Inventor： Simina Ticau ,  Anna Borodovsky

IPC: C12N15/113 ,  A61K31/555 ,  A61K31/7004 ,  A61P1/16 ,  G01N33/68

CPC classification number: C12N15/1137 ,  A61K31/555 ,  A61K31/7004 ,  A61P1/16 ,  G01N33/6893 ,  C12N2310/11 ,  C12N2310/14 ,  G01N2800/08 ,  G01N2800/60

Abstract: The disclosure provides biomarkers for diagnosis and monitoring of acute hepatic porphyria (AHP). The disclosure further provides methods for selection of agents for treatment of AHP using the biomarkers. The disclosure further provides kits for practicing the methods provided herein.

公开(公告)号：US11866701B2

公开(公告)日：2024-01-09

申请号：US16760593

申请日：2018-11-01

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：US20230136552A1

公开(公告)日：2023-05-04

申请号：US17873239

申请日：2022-07-26

Applicant: Alnylam Pharmaceuticals, Inc.

Inventor： Anna Borodovsky ,  Bret Lee Bostwick

IPC: C12N15/113 ,  A61P25/28

Abstract: The invention relates to iRNA, e.g., double-stranded ribonucleic acid (dsRNA), compositions targeting the complement component C5 gene for methods of using such iRNA to inhibit expression of C5 and to treat subjects having ALS.

公开(公告)号：US20210177884A1

公开(公告)日：2021-06-17

申请号：US17069931

申请日：2020-10-14

Applicant: Alnylam Pharmaceuticals, Inc.

Inventor： Anna Borodovsky

IPC: A61K31/7105 ,  C07K16/18 ,  C12N15/113 ,  A61K31/713 ,  A61K48/00 ,  C07K14/47

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.

公开(公告)号：US20200339998A1

公开(公告)日：2020-10-29

申请号：US16925463

申请日：2020-07-10

Applicant: Alnylam Pharmaceuticals, Inc.

Inventor： Anna Borodovsky ,  Brian Bettencourt

IPC: C12N15/113 ,  C07K16/18 ,  A61K39/395 ,  C12N15/11 ,  A61K38/12 ,  A61K31/713

Abstract: The invention relates to iRNA, e.g., double-stranded ribonucleic acid (dsRNA), compositions targeting the complement factor B (CFB) gene, the complement component C3 gene, and the complement component C9 gene and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of CFB, C9 and/or C3 and to treat subjects having a complement component-associated disease, e.g., paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome.

公开(公告)号：US20200032258A1

公开(公告)日：2020-01-30

申请号：US16378626

申请日：2019-04-09

Applicant: Alnylam Pharmaceuticals, Inc.

Inventor： Anna Borodovsky ,  Andrew Sprague

IPC: C12N15/113 ,  A61K9/00 ,  A61K31/713 ,  A61K39/395 ,  C07K16/18

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.

公开(公告)号：US20140243394A1

公开(公告)日：2014-08-28

申请号：US14279156

申请日：2014-05-15

Applicant: Alnylam Pharmaceuticals Inc. ,  The Government of the United States, as Represented by the Secretary of the Army

Inventor： Sina Bavari ,  Kelly Lyn Warfield ,  Pamela Tan ,  Anna Borodovsky ,  Tatiana Novobrantseva ,  Antonin de Fougerolles

IPC: C12N15/113

CPC classification number: C12N15/1131 ,  C07H21/02 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3521 ,  C12N2310/3531

Abstract: The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a gene from the Ebola virus.

Abstract translation: 本发明涉及用于抑制埃博拉病毒基因表达的双链核糖核酸（dsRNA）。

公开(公告)号：US11873491B2

公开(公告)日：2024-01-16

申请号：US17372710

申请日：2021-07-12

Applicant: Alnylam Pharmaceuticals, Inc.

Inventor： Kevin Fitzgerald ,  James Butler ,  Brian Bettencourt ,  Anna Borodovsky ,  Satyanarayana Kuchimanchi ,  Klaus Charisse ,  Muthiah Manoharan ,  Martin A. Maier ,  Kallanthottathil G. Rajeev ,  Donald Foster

IPC: C12N15/113 ,  A61K31/713 ,  A61K39/395 ,  C07K16/18

CPC classification number: C12N15/113 ,  A61K31/713 ,  A61K39/3955 ,  C07K16/18 ,  C07K2317/24 ,  C07K2317/76 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/3125 ,  C12N2310/321 ,  C12N2310/351 ,  C12N2310/3527 ,  C12N2320/30

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.

公开(公告)号：US20230390321A1

公开(公告)日：2023-12-07

申请号：US18180179

申请日：2023-03-08

Applicant: Alnylam Pharmaceuticals, Inc.

Inventor： Anna Borodovsky

IPC: A61K31/7105 ,  C07K16/18 ,  C12N15/113 ,  A61K31/713 ,  A61K48/00 ,  C07K14/47

CPC classification number: A61K31/7105 ,  C07K16/18 ,  C12N15/113 ,  A61K31/713 ,  A61K48/0066 ,  C07K14/472 ,  C12N2310/14 ,  C12N2310/322 ,  C12N2310/3515 ,  A61K9/0019

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.

公开(公告)号：US20200032259A1

公开(公告)日：2020-01-30

申请号：US16404862

申请日：2019-05-07

Applicant: Alnylam Pharmaceuticals, Inc.

Inventor： Kevin Fitzgerald ,  James Butler ,  Brian Bettencourt ,  Anna Borodovsky ,  Satyanarayana Kuchimanchi ,  Klaus Charisse ,  Muthiah Manoharan ,  Martin A. Maier ,  Kallanthottathil G. Rajeev ,  Donald Foster

IPC: C12N15/113 ,  A61K31/713 ,  A61K39/395 ,  C07K16/18

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.