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公开(公告)号：US11634710B2
公开(公告)日：2023-04-25
申请号：US16782021
申请日：2020-02-04
发明人： Maria David Frank-Kamenetsky , Hailin Yang , Aaron Jay Morris , Chandra Vargeese , Christopher J. Francis
IPC分类号： C07H21/02 , C12N15/113 , C12N15/115 , A61P25/28
摘要： Among other things, the present disclosure relates to designed oligonucleotides, compositions, and methods thereof. In some embodiments, provided oligonucleotide compositions provide altered splicing of a transcript. In some embodiments, provided oligonucleotide compositions have low toxicity. In some embodiments, provided oligonucleotide compositions provide improved protein binding profiles. In some embodiments, provided oligonucleotide compositions have improved delivery. In some embodiments, provided oligonucleotide compositions have improved uptake. In some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions.
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公开(公告)号：US20230089442A1
公开(公告)日：2023-03-23
申请号：US17439755
申请日：2020-03-19
申请人： Pachamuthu KANDASAMY , Mamoru SHIMIZU , David Charles Donnell BUTLER , Jayakanthan KUMARASAMY , Gopal Reddy BOMMINENI , Mohammed Rowshon ALAM , Sethumadhavan DIVAKARAMENON , Bijay Tilak BHATTARAI , Chandra VARGEESE , Keith Andrew BOWMAN , Stephany Michelle STANDLEY , WAVE LIFE SCIENCES LTD.
发明人： Pachamuthu Kandasamy , Mamoru Shimizu , David Charles Donnell Butler , Jayakanthan Kumarasamy , Gopal Reddy Bommineni , Mohammed Rowshon Alam , Sethumadhavan Divakaramenon , Bijay Tilak Bhattarai , Chandra Vargeese , Keith Andrew Bowman , Stephany Michelle Standley
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：US11608355B2
公开(公告)日：2023-03-21
申请号：US16648146
申请日：2018-09-17
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：US11597927B2
公开(公告)日：2023-03-07
申请号：US16618001
申请日：2018-06-01
发明人： Chandra Vargeese , Naoki Iwamoto , David Charles Donnell Butler , Subramanian Marappan , Genliang Lu , Jason Jingxin Zhang , Vinod Vathipadiekal , Luciano Henrique Apponi , Hanna Maria Wisniewska , Xiayun Cheng , Young Jin Cho
IPC分类号： C12N15/113 , A61K47/54 , A61K47/55 , A61K45/06
摘要： Among other things, the present disclosure provides designed PNPLA3 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 N 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：US10724035B2
公开(公告)日：2020-07-28
申请号：US16098836
申请日：2017-05-03
发明人： Chandra Vargeese , Meena , Nenad Svrzikapa , Susovan Mohapatra , Christopher J. Francis , Gregory L. Verdine , Anna Sokolovska
IPC分类号： A61K31/712 , A61K31/7125 , C12N15/11 , C12N15/113
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：US20190249173A1
公开(公告)日：2019-08-15
申请号：US16098658
申请日：2017-05-03
申请人： Chandra VARGEESE , Jason Jingxin ZHANG , Sethumadhavan DIVAKARAMENON , David Charles Donnell BUTLER , Genliang LU , Naoki IWAMOTO , Hailin YANG , Maria David FRANK-KAMENETSKY , Subramanian MARAPPAN , WAVE LIFE SCIENCES LTD.
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 , A61K31/7088 , A61K47/54
CPC分类号： C12N15/111 , A61K31/7088 , A61K47/542 , C12N2310/11 , C12N2310/14 , C12N2310/141 , C12N2310/16 , C12N2310/20 , C12N2310/315 , C12N2310/3515 , C12N2320/32
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, docosa-hexaenoic 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：US20190127733A1
公开(公告)日：2019-05-02
申请号：US15766578
申请日：2016-10-07
发明人： David Charles Donnell Butler , Sethumadhavan Divakaramenon , Christopher J. Francis , Maria David Frank-Kamenetsky , Naoki Iwamoto , Genliang Lu , Subramanian Marappan , Meena . , Chandra Vargeese , Gregory L. Verdine , Hailin Yang , Jason Jingxin Zhang
摘要： Among other things, the present disclosure relates to designed oligonucleotides, compositions, and methods thereof. In some embodiments, provided oligonucleotide compositions provide altered splicing of a transcript. In some embodiments, provided oligonucleotide compositions have low toxicity. In some embodiments, provided oligonucleotide compositions provide improved protein binding profiles. In some embodiments, provided oligonucleotide compositions have improved delivery. In some embodiments, provided oligonucleotide compositions have improved uptake. In some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions.
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公开(公告)号：US20240026358A1
公开(公告)日：2024-01-25
申请号：US18252029
申请日：2022-03-11
发明人： Prashant Monian , Chikdu Shakti Shivalila , Subramanian Marappan , Chandra Vargeese , Pachamuthu Kandasamy , Genliang Lu , Hui Yu , David Charles Donnell Butler , Luciano Henrique Apponi , Mamoru Shimizu , Stephany Michelle Standley , David John Boulay , Andrew Guzior Hoss , Jigar Desai , Jack David Godfrey , Hailin Yang , Naoki Iwamoto , Jayakanthan Kumarasamy , Anthony Lamattina , Tom Liantang Pu
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , C12N2310/322 , C12N2310/321 , C12N2310/315 , C12N2320/34
摘要： Among other things, the present disclosure provides oligonucleotides and compositions thereof. In some embodiments, provided oligonucleotides and compositions are useful for adenosine modification. In some embodiments, the present disclosure provides methods for treating various conditions, disorders or diseases that can benefit from adenosine modification.
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公开(公告)号：US20230392137A1
公开(公告)日：2023-12-07
申请号：US17953292
申请日：2022-09-26
发明人： Prashant Monian , Chikdu Shakti Shivalila , Subramanian Marappan , Chandra Vargeese , Pachamuthu Kandasamy , Genliang Lu , Hui Yu , David Charles Donnell Butler , Luciano Henrique Apponi , Mamoru Shimizu , Stephany Michelle Standley , David John Boulay , Andrew Guzior Hoss , Jigar Desai , Jack David Godfrey , Hailin Yang , Naoki Iwamoto , Jayakanthan Kumarasamy , Anthony Lamattina , Tom Liantang Pu
IPC分类号： C12N15/10 , C12N15/113
CPC分类号： C12N15/102 , C12N15/1135
摘要： Among other things, the present disclosure provides oligonucleotides and compositions thereof. In some embodiments, provided oligonucleotides and compositions are useful for adenosine modification. In some embodiments, the present disclosure provides methods for treating various conditions, disorders or diseases that can benefit from adenosine modification.
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公开(公告)号：US20230203087A1
公开(公告)日：2023-06-29
申请号：US17926987
申请日：2021-05-24
申请人： Pachamuthu Kandasamy , Jayakanthan Kumarasamy , Chandra Vargeese , Subramanian Marappan , Gopal Bommineni , Mamoru Shimizu , Naoki Iwamoto , Stephany Standley , Yuanjing Liu , Amy Jada Andreucci , Genliang LU , Onanong Chivatakarn , Akbar Husain Khan , WAVE LIFE SCIENCES LTD.
发明人： Pachamuthu Kandasamy , Jayakanthan Kumarasamy , Chandra Vargeese , Subramanian Marappan , Gopal Reddy Bommineni , Mamoru Shimizu , Naoki Iwamoto , Stephany Michelle Standley , Yuanjing Liu , Amy Jada Andreucci , Genliang Lu , Onanong Chivatakarn , Akbar Husain Khan
IPC分类号： C07H21/04 , C07F9/165 , C07D295/02
CPC分类号： C07H21/04 , C07F9/165 , C07D295/02
摘要： The present disclosure provides modified oligonucleotides and compositions and methods thereof. In some embodiments, provided technologies comprise modified sugars and/or modified internucleotidic linkages. In some embodiments, the present disclosure provides technologies for preparing modified oligonucleotides. In some embodiments, the present disclosure provides chirally controlled oligonucleotide compositions and methods for their preparation and uses.
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