公开(公告)号：US20240261438A1

公开(公告)日：2024-08-08

申请号：US18435561

申请日：2024-02-07

申请人： Ginkgo Bioworks, Inc.

发明人： Kevin Sheets ,  Erin Borchardt

IPC分类号： A61K48/00 ,  A61K38/53 ,  A61P25/28 ,  C12N7/00 ,  C12N15/86

CPC分类号： A61K48/0058 ,  A61K38/53 ,  A61P25/28 ,  C12N7/00 ,  C12N15/86 ,  C12Y603/02019 ,  C12N2750/14143 ,  C12N2750/14151

摘要： The disclosure provides nucleic acids (comprising AAV expression cassettes), AAV vectors, and compositions for use in methods for treating and/or delaying the onset of diseases associated with mutations in genes, such as UBE3A, associated with Angelman syndrome. Also, provided herein are methods for treating and/or delaying the onset of Angelman syndrome.

公开(公告)号：US20240197843A1

公开(公告)日：2024-06-20

申请号：US18283268

申请日：2022-03-24

申请人： Synlogic Operating Company, Inc.

发明人： Vincent M. Isabella ,  David Lubkowicz ,  Michael James ,  Caroline Kurtz

IPC分类号： A61K38/53 ,  A61K31/4439 ,  A61K35/00 ,  A61K35/741 ,  A61K38/45 ,  A61K38/51 ,  A61P13/04 ,  C12N9/00 ,  C12N9/10 ,  C12N9/88 ,  C12N15/52 ,  C12N15/70

CPC分类号： A61K38/53 ,  A61K31/4439 ,  A61K35/741 ,  A61K38/45 ,  A61K38/51 ,  A61P13/04 ,  C12N9/13 ,  C12N9/88 ,  C12N9/93 ,  C12N15/52 ,  C12N15/70 ,  C12Y208/03016 ,  C12Y401/01008 ,  C12Y602/01008 ,  A61K2035/115 ,  C12N2800/101 ,  C12N2830/003

摘要： The present invention provides recombinant bacterial cells comprising at least one heterologous gene encoding at least one oxalate catabolism enzyme. In another aspect, the recombinant bacterial cells further comprise at least one heterologous gene encoding an importer of oxalate. The invention further provides pharmaceutical compositions comprising the recombinant bacteria, and methods for treating disorders in which oxalate is detrimental, such as hyperoxaluria, using the pharmaceutical compositions of the invention.

公开(公告)号：US11932885B2

公开(公告)日：2024-03-19

申请号：US17832562

申请日：2022-06-03

申请人： Thoeris GmbH

发明人： Tamara Nicolson

IPC分类号： C12N9/00 ,  A61K31/216 ,  A61K38/43 ,  A61K38/53 ,  A61K47/60 ,  A61P3/00 ,  A61P7/00 ,  C07K14/00 ,  C07K19/00

CPC分类号： C12N9/93 ,  C12Y603/01002 ,  A61K31/216 ,  A61K38/43 ,  A61K38/53 ,  A61K47/60 ,  A61P3/00 ,  A61P7/00 ,  C07K14/00 ,  C07K19/00

摘要： The present invention relates to the use of glutamine synthetase as a protein therapy (such as enzyme replacement protein therapy) for the treatment of hyperammonemia. In particular the invention relates to the systemic administration of glutamine synthetase. The glutamine synthetase may be provided in conjugated or fusion form, to increase its half-life in the circulation. Also provided is a pharmaceutical composition comprising glutamine synthetase. The invention also relates to the uses, methods, and compositions involving a combination of the glutamine synthetase protein and an ammonia lowering agent, such as a nitrogen scavenger.

公开(公告)号：US20230140435A1

公开(公告)日：2023-05-04

申请号：US17906522

申请日：2021-03-16

申请人： The Trustees of the University of Pennsylvania

发明人： Benjamin L. Prosser ,  Kenneth B. Margulies ,  Alexey Bogush

IPC分类号： C12N15/113 ,  A61K38/53 ,  A61P9/04 ,  A61K45/06 ,  C12N15/86 ,  C12N15/85

摘要： A recombinant viral vector comprising an expression cassette which comprises a coding sequence for an shRNA inhibitor of vasohibin (VASH)-small vasohibin binding protein (SVBP) complex operably linked to regulatory sequences which direct expression thereof is provided. Further provided are compositions containing such viral vectors formulated for delivery to a human patient. Also provided are methods using these vectors and compositions for improving or stabilizing cardiac function.

公开(公告)号：US20230024893A1

公开(公告)日：2023-01-26

申请号：US17952990

申请日：2022-09-26

申请人： KOREA RESEARCH INSTITUTE OF BIOSCIENCE AND BIOTECHNOLOGY ,  SEOUL NATIONAL UNIVERSITY R&DB FOUNDATION

发明人： Myung Hee KIM ,  Won-Jae LEE ,  Su-Man KIM ,  Shinhye PARK ,  Kyung-Ah LEE ,  Eun-Young LEE ,  Byoung Chan KIM ,  Chul-Ho LEE ,  Jungwon HWANG

IPC分类号： A61K38/16 ,  A61K38/53 ,  A61P29/00

摘要： The present invention relates to uses of threonyl-tRNA synthetase (TARS) derived from Akkermansia muciniphila or a fragment thereof as an active ingredient for prevention, alleviation, and treatment of an inflammatory disease. The Akkermansia muciniphila TARS or a fragment thereof according to the present invention promotes the differentiation of M2 macrophages, which are anti-inflammatory macrophages, to multiply macrophages, thereby increasing the secretion of IL-10 as an anti-inflammatory cytokine, leading to the proliferation of B cells to result in an anti-inflammatory effect, and thus was verified to show alleviation effects of not only inflammatory diseases but also immune diseases, infectious diseases, and metabolic diseases accompanied by inflammation, and to have excellent effects in the prevention or treatment of an inflammatory bowel disease (IBD).

公开(公告)号：US20220401532A1

公开(公告)日：2022-12-22

申请号：US17832564

申请日：2022-06-03

申请人： Thoeris GmbH

发明人： Tamara NICOLSON

IPC分类号： A61K38/53 ,  A61K47/60 ,  A61P7/00 ,  A61P3/00 ,  A61K31/216 ,  A61K38/43 ,  C07K14/00 ,  C07K19/00 ,  C12N9/00

摘要： The present invention relates to the use of glutamine synthetase as a protein therapy (such as enzyme replacement protein therapy) for the treatment of hyperammonemia. In particular the invention relates to the systemic administration of glutamine synthetase. The glutamine synthetase may be provided in conjugated or fusion form, to increase its half-life in the circulation. Also provided is a pharmaceutical composition comprising glutamine synthetase. The invention also relates to the uses, methods, and compositions involving a combination of the glutamine synthetase protein and an ammonia lowering agent, such as a nitrogen scavenger.

公开(公告)号：US20220401531A1

公开(公告)日：2022-12-22

申请号：US17832562

申请日：2022-06-03

申请人： Thoeris GmbH

发明人： Tamara NICOLSON

IPC分类号： A61K38/53 ,  A61K47/60 ,  A61P7/00 ,  A61P3/00 ,  A61K31/216 ,  A61K38/43 ,  C07K14/00 ,  C07K19/00 ,  C12N9/00

摘要： The present invention relates to the use of glutamine synthetase as a protein therapy (such as enzyme replacement protein therapy) for the treatment of hyperammonemia. In particular the invention relates to the systemic administration of glutamine synthetase. The glutamine synthetase may be provided in conjugated or fusion form, to increase its half-life in the circulation. Also provided is a pharmaceutical composition comprising glutamine synthetase. The invention also relates to the uses, methods, and compositions involving a combination of the glutamine synthetase protein and an ammonia lowering agent, such as a nitrogen scavenger.

公开(公告)号：US20220331297A1

公开(公告)日：2022-10-20

申请号：US17196695

申请日：2021-03-09

申请人： Arvinas Operations, Inc.

发明人： Andrew P. Crew ,  Erika Araujo

IPC分类号： A61K31/437 ,  A61P25/00 ,  C07D487/04 ,  C07D417/14 ,  A61P29/00 ,  C07D401/14 ,  A61K47/64 ,  A61P21/04 ,  A61P37/06 ,  A61P35/00 ,  A61K31/422 ,  A61K31/427 ,  A61K31/551 ,  A61K38/20 ,  A61K38/53

摘要： The present disclosure relates to bifunctional compounds, which find utility as modulators of Interleukin-1 Receptor-Associated Kinase 4 (IRAK-4); the target protein). In particular, the present disclosure is directed to bifunctional compounds, which contain on one end a Von Hppel-Lindau, cereblon, ligand which binds to the E3 ubiquitin ligase and on the other end a moiety which binds the target protein, such that the target protein is placed in proximity to the ubiquitin ligase to effect degradation (and inhibition) of target protein. The present disclosure exhibits a broad range of pharmacological activities associated with degradation/inhibition of target protein. Diseases or disorders that result from aggregation or accumulation of the target protein are treated or prevented with compounds and compositions of the present disclosure.

公开(公告)号：US11098364B2

公开(公告)日：2021-08-24

申请号：US16405812

申请日：2019-05-07

申请人： The University Of Tokyo

发明人： Hirotaka Matsuo ,  Nariyoshi Shinomiya ,  Tappei Takada

IPC分类号： C12Q1/68 ,  C12P19/34 ,  C12Q1/6883 ,  A61K38/00 ,  C07K14/47 ,  G01N33/68 ,  A61K38/17 ,  A61K38/44 ,  A61K38/45 ,  A61K38/53 ,  C12N9/00 ,  A61K48/00

摘要： To specify a molecule associated with the onset of gout so as to provide a method for evaluating a diathesis of uric acid-related diseases and a diathesis of inflammation-related diseases, an evaluation kit for carrying out the method, an inspection object, and a drug, on the basis of the molecule specified above, for contributing to the early treatment and prevention of the uric acid-related diseases and inflammation-related diseases. The molecule includes any one protein and cDNA of CNIH2-PACS1, ALDH2, MYL2-CUX2, GCKR, MAP3K11, NPT4, ABCG2, HIST1H2BF/HIST1H4E, HIST1H2BE/HIST1H4D and FAM35A, or proteins of combination thereof with GLUT9, NPT1, URAT1, or NXRN2, and is capable of selectively inducing gout. A molecule includes protein and cDNA of an ABCG2 variant and is capable of selectively and ATP-dependently decreasing urate excretion.

公开(公告)号：US11065231B2

公开(公告)日：2021-07-20

申请号：US16194094

申请日：2018-11-16

申请人： Arvinas Operations, Inc.

发明人： Andrew P. Crew ,  Erika Araujo

IPC分类号： A61K31/437 ,  A61P25/00 ,  C07D487/04 ,  C07D417/14 ,  A61P29/00 ,  C07D401/14 ,  A61K47/64 ,  A61P21/04 ,  A61P37/06 ,  A61P35/00 ,  A61K31/422 ,  A61K31/427 ,  A61K31/551 ,  A61K38/20 ,  A61K38/53

摘要： The present disclosure relates to bifunctional compounds, which find utility as modulators of Interleukin-1 Receptor-Associated Kinase 4 (IRAK-4); the target protein). In particular, the present disclosure is directed to bifunctional compounds, which contain on one end a Von Hppel-Lindau, cereblon, ligand which binds to the E3 ubiquitin ligase and on the other end a moiety which binds the target protein, such that the target protein is placed in proximity to the ubiquitin ligase to effect degradation (and inhibition) of target protein. The present disclosure exhibits a broad range of pharmacological activities associated with degradation/inhibition of target protein. Diseases or disorders that result from aggregation or accumulation of the target protein are treated or prevented with compounds and compositions of the present disclosure.