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公开(公告)号：US20230295617A1
公开(公告)日：2023-09-21
申请号：US18072296
申请日：2022-11-30
发明人： Chandra Vargeese , Jason Jingxin Zhang , Sethumadhavan Divakaramenon , David Charles Donnell Butler , Genliang Lu , Naoki Iwamoto , Hailin Yang , Maria David Frank-Kamenetsky , Subramanian Marappan
IPC分类号： C12N15/11 , A61K47/54 , A61K31/7088
CPC分类号： C12N15/111 , A61K47/542 , A61K31/7088 , C12N2310/141 , C12N2310/14 , C12N2310/20 , C12N2320/32 , C12N2310/11 , C12N2310/16 , C12N2310/315 , C12N2310/3515
摘要： In some embodiments, the present disclosure pertains to compositions and methods related to delivery of a biologically active agent, wherein the compositions comprise a biologically active agent and a lipid. In various embodiments, the lipid is selected from: lauric acid, myristic acid, palmitic acid, stearic acid, oleic acid, linoleic acid, alpha-linolenic acid, gamma-linolenic acid, docosahexaenoic acid (cis-DHA), turbinaric acid and dilinoleyl. In some embodiments, a composition and method are useful for delivery of a biologically active agent to a particular cell or tissue, e.g., a muscle cell or tissue.
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公开(公告)号：US11718638B2
公开(公告)日：2023-08-08
申请号：US16624896
申请日：2018-06-21
发明人： David Charles Donnell Butler , Christopher P. Hencken , Naoki Iwamoto , Pachamuthu Kandasamy , Alvaro Andres Lanao , Genliang Lu , Mamoru Shimizu , Sethumadhavan Divakaramenon , Chandra Vargeese , Gopal Reddy Bommineni , Subramanian Marappan
IPC分类号： C07H21/04 , C07H19/06 , C07C213/00 , C07C215/20 , C07C215/44 , C07D207/08 , C07D211/22 , C07F7/08 , C07F9/6564
CPC分类号： C07H21/04 , C07C213/00 , C07C215/20 , C07C215/44 , C07D207/08 , C07D211/22 , C07F7/083 , C07F7/0812 , C07F9/6564 , C07H19/06 , C07C2601/14
摘要： The present disclosure, among other things, provides technologies for synthesis, including reagents and methods for stereoselective synthesis. In some embodiments, the present disclosure provides compounds useful as chiral auxiliaries. In some embodiments, the present disclosure provides reagents and methods for oligonucleotide synthesis. In some embodiments, the present disclosure provides reagents and methods for chirally controlled preparation of oligonucleotides. In some embodiments, technologies of the present disclosure are particularly useful for constructing challenging internucleotidic linkages, providing high yields and stereoselectivity.
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公开(公告)号：US11596646B2
公开(公告)日：2023-03-07
申请号：US16755544
申请日：2018-10-12
IPC分类号： C07H21/04 , A61K31/7125 , C12N15/113
摘要： Among other things, the present disclosure provides technologies for altering splicing, particularly for increasing inclusion of exons in splicing products. In some embodiments, the present disclosure provides SMN2 oligonucleotides, compositions, and methods thereof. In some embodiments, the present disclosure provides chirally controlled SMN2 oligonucleotide compositions. In some embodiments, provided oligonucleotides and compositions can increase level of an exon 7-containing SMN2 splicing product and/or a gene product thereof. In some embodiments, the present disclosure provides methods for treatment of splicing-related conditions, disorders and diseases. In some embodiments, the present disclosure provides methods for treating SMN2-related conditions, disorders and diseases such as SMA (spinal muscular atrophy) and ALS (amyotrophic lateral sclerosis).
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公开(公告)号：US20220356204A1
公开(公告)日：2022-11-10
申请号：US16624896
申请日：2018-06-21
申请人： David Charles Donnell BUTLER , Christopher P. HENCKEN , Nao-ki IWAMOTO , Pachamuthu KANDASAMY , Alvaro Andres LANAO , Genliang LU , Mamoru SHIMIZU , Sethumadhavan DIVAKARAMENON , Chandra VARGEESE , Gopal Reddy BOMMINENI , Subramanian MARAPPAN , WAVE LIFE SCIENCES LTD.
发明人： David Charles Donnell Butler , Christopher P. Hencken , Naoki Iwamoto , Pachamuthu Kandasamy , Alvaro Andres Lanao , Genliang Lu , Mamoru Shimizu , Sethumadhavan Divakaramenon , Chandra Vargeese , Gopal Reddy Bommineni , Subramanian Marappan
IPC分类号： C07H21/04 , C07F7/08 , C07D207/08 , C07H19/06 , C07C215/20 , C07C213/00 , C07D211/22 , C07C215/44
摘要： The present disclosure, among other things, provides technologies for synthesis, including reagents and methods for stereoselective synthesis. In some embodiments, the present disclosure provides compounds useful as chiral auxiliaries. In some embodiments, the present disclosure provides reagents and methods for oligonucleotide synthesis. In some embodiments, the present disclosure provides reagents and methods for chirally controlled preparation of oligonucleotides. In some embodiments, technologies of the present disclosure are particularly useful for constructing challenging internucleotidic linkages, providing high yields and stereoselectivity.
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公开(公告)号：US20220195429A1
公开(公告)日：2022-06-23
申请号：US17375658
申请日：2021-07-14
发明人： Chandra Vargeese , Meena , Nenad Svrzikapa , Susovan Mohapatra , Christopher J. Francis , Gregory L. Verdine , Anna Sokolovska
IPC分类号： C12N15/113 , C12N15/115 , A61P25/28
摘要： Among other things, the present disclosure relates to chirally controlled oligonucleotides of select designs, chirally controlled oligonucleotide compositions, and methods of making and using the same. In some embodiments, a provided chirally controlled oligonucleotide composition provides different cleavage patterns of a nucleic acid polymer than a reference oligonucleotide composition. In some embodiments, a provided chirally controlled oligonucleotide composition provides single site cleavage within a complementary sequence of a nucleic acid polymer. In some embodiments, a chirally controlled oligonucleotide composition has any sequence of bases, and/or pattern or base modifications, sugar modifications, backbone modifications and/or stereochemistry, or combination of these elements, described herein.
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公开(公告)号：US20210032620A1
公开(公告)日：2021-02-04
申请号：US16636902
申请日：2018-08-07
发明人： Chandra Vargeese , Zhong Zhong , Naoki Iwamoto , Jason Jingxin Zhang , Jean-Cosme Dodart , Yuanjing Liu , Pachamuthu Kandasamy , Sethumadhavan Divakaramenon , Genliang Lu , Subramanian Marappan
IPC分类号： C12N15/11
摘要： Among other things, the present disclosure provides oligonucleotides, compositions, and methods thereof. Among other things, the present disclosure encompasses the recognition that structural elements of oligonucleotides, such as base sequence, chemical modifications (e.g., modifications of sugar, base, and/or internucleotidic linkages) or patterns thereof, conjugation with additional chemical moieties, and/or stereochemistry [e.g., stereochemistry of backbone chiral centers (chiral internucleotidic linkages)], and/or patterns thereof, can have significant impact on oligonucleotide properties and activities, e.g., knockdown ability, stability, delivery, etc. In some embodiments, the oligonucleotides decrease the expression, activity and/or level of a C9orf72 gene, including but not limited to, one comprising a repeat expansion, or a gene product thereof. In some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions, for example, in treatment of C9orf72-related disorders.
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公开(公告)号：US20200231620A1
公开(公告)日：2020-07-23
申请号：US16648146
申请日：2018-09-17
申请人： Keith Andrew BOWMAN , Chandra VARGEESE , David Charles Donnell BUTLER , Pachamuthu KANDASAMY , Mohammed Rowshon ALAM , Mamoru SHIMIZU , Stephany Michelle STANDLEY , Vincent ADUDA , Gopal Reddy BOMMINENI , Snehlata TRIPATHI , Ilia KORBOUKH , WAVE LIFE SCIENCES LTD.
发明人： Keith Andrew Bowman , Chandra Vargeese , David Charles Donnell Butler , Pachamuthu Kandasamy , Mohammed Rowshon Alam , Mamoru Shimizu , Stephany Michelle Standley , Vincent Aduda , Gopal Reddy Bommineni , Snehlata Tripathi , Ilia Korboukh
摘要： Among other things, the present disclosure provides technologies for oligonucleotide preparation, particularly chirally controlled oligonucleotide preparation, which technologies provide greatly improved crude purity and yield, and significantly reduce manufacturing costs.
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公开(公告)号：US20200157545A1
公开(公告)日：2020-05-21
申请号：US16618010
申请日：2018-06-01
申请人： Chandra VARGEESE , Naoki IWAMOTO , David Charles Donnell BUTLER , Subramanian MARAPPAN , Genliang LU , Jason Jingxin ZHANG , Vinod VATHIPADIEKAL , Maria David FRANK-KAMENETSKY , Luciano Henrique APPONI , WAVE LIFE SCIENCES LTD.
发明人： Chandra Vargeese , Naoki Iwamoto , David Charles Donnell Butler , Subramanian Marappan , Genliang Lu , Jason Jingxin Zhang , Vinod Vathipadiekal , Maria David Frank-Kamenetsky , Luciano Henrique Apponi , Young Jin Cho
IPC分类号： C12N15/113 , A61K31/712
摘要： Among other things, the present disclosure provides designed oligonucleotides, compositions, and methods thereof. In some embodiments, provided oligonucleotide compositions provide improved single-stranded RNA interference and/or RNase H-mediated knockdown. Among other things, the present disclosure encompasses the recognition that structural elements of oligonucleotides, such as base sequence, chemical modifications (e.g., modifications of sugar, base, and/or internucleotidic linkages) or patterns thereof, conjugation with additional chemical moieties, and/or stereochemistry [e.g., stereochemistry of backbone chiral centers (chiral internucleotidic linkages)], and/or patterns thereof, can have significant impact on oligonucleotide properties and activities, e.g., RNA interference (RNAi) activity, stability, delivery, etc. In some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions, for example, in RNA interference and/or RNase H-mediated knockdown.
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公开(公告)号：US20200080083A1
公开(公告)日：2020-03-12
申请号：US16551503
申请日：2019-08-26
发明人： Chandra Vargeese , Meena . , Nenad Svrzikapa , Susovan Mohapatra , Christopher J. Francis , Gregory L. Verdine , Anna Sokolovska
IPC分类号： C12N15/113 , A61P25/28 , C12N15/115
摘要： Among other things, the present disclosure relates to chirally controlled oligonucleotides of select designs, chirally controlled oligonucleotide compositions, and methods of making and using the same. In some embodiments, a provided chirally controlled oligonucleotide composition provides different cleavage patterns of a nucleic acid polymer than a reference oligonucleotide composition. In some embodiments, a provided chirally controlled oligonucleotide composition provides single site cleavage within a complementary sequence of a nucleic acid polymer. In some embodiments, a chirally controlled oligonucleotide composition has any sequence of bases, and/or pattern or base modifications, sugar modifications, backbone modifications and/or stereochemistry, or combination of these elements, described herein.
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公开(公告)号：US20190209604A1
公开(公告)日：2019-07-11
申请号：US16305937
申请日：2017-06-02
发明人： Jason Jingxin Zhang , Naoki Iwamoto , Christopher J. Francis , Chandra Vargeese , David Charles Donnell Butler , Sethumadhavan Divakaramenon , Genliang Lu , Subramanian Marappan
IPC分类号： A61K31/7125 , C12N15/117 , A61P21/00
CPC分类号： A61K31/7125 , A61K31/7088 , A61P21/00 , C12N15/117 , C12N2310/17 , C12N2310/31 , C12N2310/315 , C12N2310/351 , C12N2310/3515
摘要： The present disclosure pertains to the recognition that immune responses mediated by CpG oligonucleotides can be affected by the stereochemistry of modified internucleotidic linkages such as phosphorothioates. In some embodiments, the present disclosure relates to chirally controlled CpG oligonucleotide compositions comprising CpG oligonucleotides comprising multiple modified internucleotidic linkages such as phosphorothioate linkages, wherein the oligonucleotides comprise one or more CpG region motifs having defined stereochemistry patterns of chiral internucleotidic linkages. In some embodiments, CpG oligonucleotides comprising one or more CpG region motifs are capable of agonizing an immune response. In some embodiments, CpG oligonucleotides comprising one or more CpG region motifs are antagonistic. Methods for making and using chirally controlled CpG oligonucleotide compositions are also described. In some embodiments, no immune modulation is desired, and the present disclosure provides methods of identifying chirally controlled oligonucleotide compositions which have decreased immune modulation.
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