公开(公告)号：US11597929B2

公开(公告)日：2023-03-07

申请号：US17453995

申请日：2021-11-08

申请人： Dicerna Pharmaceuticals, Inc.

发明人： Bob Dale Brown

IPC分类号： C07H21/04 ,  C12N15/113 ,  A61K31/713 ,  C12N15/11

摘要： The invention provides compositions and methods for reducing expression of a target gene in a cell, involving contacting a cell with an isolated double stranded nucleic acid (dsNA) in an amount effective to reduce expression of a target gene in a cell. The dsNAs of the invention possess a single stranded extension (in most embodiments, the single stranded extension comprises at least one modified nucleotide and/or phosphate back bone modification). Such single stranded extended Dicer-substrate siRNAs (DsiRNAs) were demonstrated to be effective RNA inhibitory agents compared to corresponding double stranded DsiRNAs.

公开(公告)号：US11414664B2

公开(公告)日：2022-08-16

申请号：US17407930

申请日：2021-08-20

申请人： Dicerna Pharmaceuticals, Inc.

发明人： Bob Dale Brown

IPC分类号： C07H21/04 ,  C07H21/02 ,  C12N15/113 ,  A61K31/713 ,  C12N15/11

摘要： The invention provides compositions and methods for reducing expression of a target gene in a cell, involving contacting a cell with an isolated double stranded nucleic acid (dsNA) in an amount effective to reduce expression of a target gene in a cell. The dsNAs of the invention possess a single stranded extension (in most embodiments, the single stranded extension comprises at least one modified nucleotide and/or phosphate back bone modification). Such single stranded extended Dicer-substrate siRNAs (DsiRNAs) were demonstrated to be effective RNA inhibitory agents compared to corresponding double stranded DsiRNAs.

公开(公告)号：US11408003B2

公开(公告)日：2022-08-09

申请号：US17407966

申请日：2021-08-20

申请人： Dicerna Pharmaceuticals, Inc.

发明人： Bob Dale Brown

IPC分类号： C07H21/04 ,  C07H21/02 ,  C12N15/113 ,  A61K31/713 ,  C12N15/11

摘要： The invention provides compositions and methods for reducing expression of a target gene in a cell, involving contacting a cell with an isolated double stranded nucleic acid (dsNA) in an amount effective to reduce expression of a target gene in a cell. The dsNAs of the invention possess a single stranded extension (in most embodiments, the single stranded extension comprises at least one modified nucleotide and/or phosphate back bone modification). Such single stranded extended Dicer-substrate siRNAs (DsiRNAs) were demonstrated to be effective RNA inhibitory agents compared to corresponding double stranded DsiRNAs.

公开(公告)号：US20210388358A1

公开(公告)日：2021-12-16

申请号：US17407966

申请日：2021-08-20

申请人： Dicerna Pharmaceuticals, Inc.

发明人： Bob Dale Brown

IPC分类号： C12N15/113 ,  A61K31/713 ,  C12N15/11

摘要： The invention provides compositions and methods for reducing expression of a target gene in a cell, involving contacting a cell with an isolated double stranded nucleic acid (dsNA) in an amount effective to reduce expression of a target gene in a cell. The dsNAs of the invention possess a single stranded extension (in most embodiments, the single stranded extension comprises at least one modified nucleotide and/or phosphate back bone modification). Such single stranded extended Dicer-substrate siRNAs (DsiRNAs) were demonstrated to be effective RNA inhibitory agents compared to corresponding double stranded DsiRNAs.

公开(公告)号：US11351265B2

公开(公告)日：2022-06-07

申请号：US16165760

申请日：2018-10-19

申请人： Dicerna Pharmaceuticals, Inc.

发明人： Bob Dale Brown ,  Bo Ying

IPC分类号： A61K47/60 ,  A61K47/54 ,  A61K9/127 ,  C12N15/11

摘要： Formulations comprising anionic agents such as nucleic acids within a lipid-containing particle methods of formulating a lipid-containing particle comprising an anionic agent such as a nucleic acid, methods for preparing a lipid-containing particle comprising an anionic agent such as a nucleic acid, methods for therapeutic delivery of an anionic agent to a patient in need thereof, where the anionic agent is formulated in a lipid-containing particle as described herein.

公开(公告)号：US20210292303A1

公开(公告)日：2021-09-23

申请号：US17342281

申请日：2021-06-08

申请人： Dicerna Pharmaceuticals, Inc.

发明人： Bob Dale Brown

IPC分类号： C07D403/06 ,  C07D233/61 ,  C07D233/64 ,  C07D239/42 ,  C07D241/04 ,  C07C237/06 ,  C07D295/03 ,  C07D211/36 ,  C07D295/13 ,  C07D295/15 ,  C07C211/21 ,  C12N15/11 ,  C12N15/113 ,  C12N15/88 ,  A61K9/127 ,  A61K31/713 ,  A61K47/18 ,  A61K47/22 ,  A61K9/107 ,  A61K9/14 ,  C07D243/08 ,  C07D401/04 ,  C07D403/04

摘要： The present invention relates to lipid compounds and uses thereof. In particular, the compounds include a class of cationic lipids having an amine moiety, such as an amino-amine or an amino-amide moiety. The lipid compounds are useful for in vivo or in vitro delivery of one or more agents (e.g., a polyanionic payload or an antisense payload, such as an RNAi agent).

公开(公告)号：US20210062189A1

公开(公告)日：2021-03-04

申请号：US17095945

申请日：2020-11-12

申请人： Dicerna Pharmaceuticals, Inc.

发明人： Bob Dale Brown

IPC分类号： C12N15/113 ,  A61K31/713 ,  C12N15/11

摘要： The invention provides compositions and methods for reducing expression of a target gene in a cell, involving contacting a cell with an isolated double stranded nucleic acid (dsNA) in an amount effective to reduce expression of a target gene in a cell. The dsNAs of the invention possess a single stranded extension (in most embodiments, the single stranded extension comprises at least one modified nucleotide and/or phosphate back bone modification). Such single stranded extended Dicer-substrate siRNAs (DsiRNAs) were demonstrated to be effective RNA inhibitory agents compared to corresponding double stranded DsiRNAs.

公开(公告)号：US20200031862A1

公开(公告)日：2020-01-30

申请号：US16555193

申请日：2019-08-29

申请人： Dicerna Pharmaceuticals, Inc.

发明人： Bob Dale Brown ,  Weimin Wang

IPC分类号： C07H21/04 ,  A61K47/54 ,  C12N15/11 ,  C12N15/113 ,  C07H1/00 ,  C07H99/00

摘要： The invention provides for double stranded nucleic acid molecules comprising a 5′ extension of the sense or antisense strand and further comprising a plurality of nucleotides that are conjugated to a ligand and methods of using the double-stranded nucleic acid molecules. Ligand-modified oligomers where the sense stands form a tetraloop provide new potent and stable RNA interference agents. These dsNA molecules are synthesized using a plurality of nucleotides that include ligand-modified monomers, nucleotide analog monomers, modified nucleotide monomers and the like, using standard nucleotide synthetic methods and systems.

公开(公告)号：US20150374842A1

公开(公告)日：2015-12-31

申请号：US14852481

申请日：2015-09-11

申请人： Dicerna Pharmaceuticals, Inc.

发明人： Bob Dale Brown ,  Bo Ying

IPC分类号： A61K47/48 ,  C12N15/11

CPC分类号： A61K47/60 ,  A61K9/1271 ,  A61K9/1272 ,  A61K9/1277 ,  A61K47/543 ,  C12N15/111 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2320/32

摘要： Formulations comprising anionic agents such as nucleic acids within a lipid-containing particle methods of formulating a lipid-containing particle comprising an anionic agent such as a nucleic acid, methods for preparing a lipid-containing particle comprising an anionic agent such as a nucleic acid, methods for therapeutic delivery of an anionic agent to a patient in need thereof, where the anionic agent is formulated in a lipid-containing particle as described herein.

摘要翻译： 包含阴离子试剂的制剂，例如含脂质颗粒内的核酸，配制包含阴离子试剂如核酸的含脂颗粒的方法，制备包含阴离子试剂如核酸的含脂颗粒的方法， 将阴离子剂治疗性递送至有需要的患者的方法，其中将阴离子剂配制在本文所述的含脂质颗粒中。

公开(公告)号：US11459566B1

公开(公告)日：2022-10-04

申请号：US17472227

申请日：2021-09-10

申请人： Dicerna Pharmaceuticals, Inc.

发明人： Bob Dale Brown ,  Henryk T. Dudek

IPC分类号： C12N15/11 ,  C12N15/113

摘要： This invention relates to compounds, compositions, and methods useful for reducing α-1 antitrypsin target RNA and protein levels via use of dsRNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.