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公开(公告)号：US20240175018A1
公开(公告)日：2024-05-30
申请号：US18178470
申请日：2023-03-03
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 , A61K45/06 , A61K47/54 , A61K47/55
CPC分类号： C12N15/113 , A61K45/06 , A61K47/54 , A61K47/545 , A61K47/548 , A61K47/549 , A61K47/55 , C12N2310/14 , C12N2310/315 , C12N2310/32 , C12N2310/33 , C12N2310/352
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 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：US20220306573A1
公开(公告)日：2022-09-29
申请号：US17046752
申请日：2019-04-11
申请人： Jason Jingxi ZHANG , Chandra VARGEESE , Naoki IWAMOTO , Chikdu Shakti SHIVALILA , Nayantara KOTHARI , Ann Fiegen DURBIN , Selvi RAMASAMY , Pachamuthu KANDASAMY , Jayakanthan KUMARASAMY , Gopal Reddy BOMMINENI , Subramanian MARAPPAN , Sethumadhavan DIVAKARAMENON , David Charles Donnel BUTLER , Genliang LU , Hailin YANG , Mamoru SHIMIZU , Prashant MONIAN , WAVE LIFE SCIENCES LTD.
发明人： Jason Jingxin Zhang , Chandra Vargeese , Naoki Iwamoto , Chikdu Shakti Shivalila , Nayantara Kothari , Ann Fiegen Durbin , Selvi Ramasamy , Pachamuthu Kandasamy , Jayakanthan Kumarasamy , Gopal Reddy Bommineni , Subramanian Marappan , Sethumadhavan Divakaramenon , David Charles Donnell Butler , Genliang Lu , Hailin Yang , Mamoru Shimizu , Prashant Monian
IPC分类号： C07C317/28 , C07H21/02 , C07H21/04 , C12N15/113
摘要： Among other things, the present disclosure provides designed oligonucleotides, compositions, and methods of use thereof. In some embodiments, the present disclosure provides technologies useful for reducing levels of transcripts. In some embodiments, the present disclosure provides technologies useful for modulating transcript splicing. In some embodiments, provided technologies can alter splicing of a dystrophin (DMD) transcript. In some embodiments, the present disclosure provides methods for treating diseases, such as Duchenne muscular dystrophy, Becker's muscular dystrophy, etc.
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公开(公告)号：US20220145300A1
公开(公告)日：2022-05-12
申请号：US17609330
申请日：2020-05-08
IPC分类号： C12N15/113 , A61K31/713 , A61P25/28
摘要： Among other things, the present disclosure provides C9orf72 oligonucleotides, compositions, and methods thereof. In some embodiments, the present disclosure provides methods for treating C9orf72-associated conditions, disorders or diseases, such as amyotrophic lateral sclerosis and frontotemporal dementia.
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公开(公告)号：US20190008986A1
公开(公告)日：2019-01-10
申请号：US16139091
申请日：2018-09-23
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
IPC分类号： A61K48/00 , A61K31/7115 , A61K31/712 , A61K31/7125 , A61P21/00 , A61P25/14
CPC分类号： C12N15/113 , A61K31/7115 , A61K31/712 , A61K31/7125 , A61K48/0016 , A61K48/0066 , A61K48/0083 , A61P21/00 , A61P25/14 , A61P27/00 , C07F9/59 , C07H21/00 , C07H21/02
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：US20160347784A1
公开(公告)日：2016-12-01
申请号：US15167669
申请日：2016-05-27
发明人： Gregory L. Verdine , Meena , Naoki Iwamoto
IPC分类号： C07H21/00
CPC分类号： A61K31/70 , C07F9/65515 , C07H21/00
摘要： Described herein are nucleic acid prodrugs and nucleic acid prodrugs comprising chiral phosphorous moieties. Also described herein are methods of making and using nucleic acid prodrugs and nucleic acid prodrugs comprising chiral phosphorous moieties.
摘要翻译： 本文描述的是包含手性磷部分的核酸前药和核酸前药。 本文还描述了制备和使用包含手性磷部分的核酸前药和核酸前药的方法。
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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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公开(公告)号：US11603532B2
公开(公告)日：2023-03-14
申请号：US16618010
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 , Maria David Frank-Kamenetsky , Luciano Henrique Apponi , Young Jin Cho
IPC分类号： C12N15/11 , 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：US20220401467A1
公开(公告)日：2022-12-22
申请号：US17177111
申请日：2021-02-16
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 , A61P21/00 , C12N15/117 , A61K31/7088
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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