公开(公告)号：US20210228614A1

公开(公告)日：2021-07-29

申请号：US17106259

申请日：2020-11-30

申请人： Alnylam Pharmaceuticals, Inc. ,  The UAB Research Foundation

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

IPC分类号： A61K31/7105 ,  C12N15/113

摘要： 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

申请人： Alnylam Pharmaceuticals, Inc. ,  The UAB Research Foundation

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

IPC分类号： A61K31/7105 ,  C12N15/113

摘要： 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：US20210369683A1

公开(公告)日：2021-12-02

申请号：US17322260

申请日：2021-05-17

申请人： VANDERBILT UNIVERSITY ,  The UAB Research Foundation ,  The United States of America, as Represented by the Secretary, Department of Health and Human Servic

发明人： Matthew Hall ,  Daniel J. Urban ,  John Knight ,  Ross Holmes ,  Kyle David Wood ,  Alex Waterson ,  Victor M. Darley-Usmar ,  Leonard M. Neckers

IPC分类号： A61K31/427 ,  A61K31/4439 ,  A61K31/506

摘要： The disclosure provides methods of treating a patient having primary hyperoxaluria or idiopathic hyperoxaluria comprising administering a therapeutically effective amound of compound of the formula and pharmaceutically acceptable salts, solvates, and hydrates thereof to the patient. The variables, e.g. ring A, n, R, R3, R10, X, Y, and Z are defined herein. These compounds act as lactate dehydrogenase inhibitors and are useful inhibiting the conversion of glyoxylate to oxalate. When administered to a patient having a disease or disorder associated with elevated oxalate levels, such as PH type 1, type 2, or type 3 or idiopathic hyperoxaluria the compounds prevent or substantially reduce the amount and buildup of oxalate the patient's kidneys, bladder, urinary tract and other parts of the patient's body.

公开(公告)号：US11752138B2

公开(公告)日：2023-09-12

申请号：US17322260

申请日：2021-05-17

申请人： VANDERBILT UNIVERSITY ,  The UAB Research Foundation ,  The United States of America, as Represented by the Secretary, Department of Health and Human Services

发明人： Matthew Hall ,  Daniel J. Urban ,  John Knight ,  Ross Holmes ,  Kyle David Wood ,  Alex Waterson ,  Victor M. Darley-Usmar ,  Leonard M. Neckers

IPC分类号： A61K31/427 ,  A61K31/506 ,  A61K31/4439

CPC分类号： A61K31/427 ,  A61K31/4439 ,  A61K31/506

摘要： The disclosure provides methods of treating a patient having primary hyperoxaluria or idiopathic hyperoxaluria comprising administering a therapeutically effective amound of compound of the formula




and pharmaceutically acceptable salts, solvates, and hydrates thereof to the patient. The variables, e.g. ring A, n, R, R3, R10, X, Y, and Z are defined herein. These compounds act as lactate dehydrogenase inhibitors and are useful inhibiting the conversion of glyoxylate to oxalate. When administered to a patient having a disease or disorder associated with elevated oxalate levels, such as PH type 1, type 2, or type 3 or idiopathic hyperoxaluria the compounds prevent or substantially reduce the amount and buildup of oxalate the patient's kidneys, bladder, urinary tract and other parts of the patient's body.