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公开(公告)号：US10450568B2
公开(公告)日：2019-10-22
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： A61K31/712 , A61K31/7125 , C12N15/113 , C07H21/02 , C07H21/00 , A61P25/14 , A61P21/00 , A61K31/7115 , A61K48/00 , A61P27/00 , C07F9/59
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：US10307434B2
公开(公告)日：2019-06-04
申请号：US15167669
申请日：2016-05-27
发明人： Gregory L. Verdine , Meena , Naoki Iwamoto
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.
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公开(公告)号：US09885037B2
公开(公告)日：2018-02-06
申请号：US14414614
申请日：2013-07-12
发明人： David Butler , Naoki Iwamoto , Meena , Nenad Svrzikapa , Gregory L. Verdine , Ivan Zlatev
IPC分类号： C07H21/04 , C12N15/113 , C07H21/00 , C07C317/28 , C07D295/088 , C12Q1/68 , C12N15/11
摘要： The present invention relates to chirally controlled oligonucleotides, chirally controlled oligonucleotide compositions, and the method of making and using the same. The invention specifically encompasses the identification of the source of certain problems with prior methodologies for preparing chiral oligonucleotides, including problems that prohibit preparation of fully chirally controlled compositions, particularly compositions comprising a plurality of oligonucleotide types. In some embodiments, the present invention provides chirally controlled oligonucleotide compositions. In some embodiments, the present invention provides methods of making chirally controlled oligonucleotides and chirally controlled oligonucleotide compositions.
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公开(公告)号：US20240175016A1
公开(公告)日：2024-05-30
申请号：US17766680
申请日：2020-10-05
发明人： Yuanjing Liu , Naoki Iwamoto , Chandra Vargeese , Zhong Zhong , Amy Jada Andreucci , Susovan Mohapatra
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/3341
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：US20240117347A1
公开(公告)日：2024-04-11
申请号：US18062422
申请日：2022-12-06
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： C12N15/113 , A61K31/7115 , A61K31/712 , A61K31/7125 , A61K48/00 , A61P21/00 , A61P25/14 , A61P27/00 , C07F9/59 , C07H21/00 , C07H21/02
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 , C12N2310/11 , C12N2310/315 , C12N2310/322 , C12N2310/343 , C12N2310/346 , C12N2320/33
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：US20240109931A1
公开(公告)日：2024-04-04
申请号：US17960090
申请日：2022-10-04
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 , Hanna Maria Wisniewska-wrona , Xiayun Cheng , Young Jin Cho
IPC分类号： C07H17/04 , C07H19/04 , C12N15/113
CPC分类号： C07H17/04 , C07H19/04 , C12N15/113 , C12N2310/14 , C12N2310/315
摘要： Among other things, the present disclosure provides designed APOC3 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：US20230145795A1
公开(公告)日：2023-05-11
申请号：US17605997
申请日：2020-04-24
发明人： Michael John Byrne , Vinod Vathipadiekal , Naoki Iwamoto , Chandra Vargeese , Lankai Guo , Andrew Guzior Hoss
IPC分类号： C12N15/113 , A61P27/00
CPC分类号： C12N15/1138 , A61P27/00 , C12N2310/315 , C12N2310/351
摘要： Among other things, the present disclosure provides RHO oligonucleotides, compositions, and methods. In some embodiments, provided oligonucleotides comprise nucleobase modifications, sugar modifications, internucleotidic linkage modifications and/or patterns thereof, and have improved properties, activities and/or selectivities. In some embodiments, the present disclosure provides RHO oligonucleotides, compositions and methods for preventing and/or treating RHO-related conditions, disorders or diseases, such as retinopathy (e.g, retinal degeneration, retinal degenerative disease, retinal degenerative disorder, inherited retinal degenerative disorder, retinitis pigmentosa, autosomal dominant retinitis pigmentosa, etc.).
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公开(公告)号：US11643657B2
公开(公告)日：2023-05-09
申请号：US16717986
申请日：2019-12-17
发明人： David Charles Donnell Butler , Naoki Iwamoto , Meena , Nenad Svrzikapa , Gregory L. Verdine , Ivan Zlatev
IPC分类号： C12N15/113 , C07H21/00 , C07C317/28 , C07D295/088 , C12N15/11 , C12Q1/6876
CPC分类号： C12N15/113 , C07C317/28 , C07D295/088 , C07H21/00 , C12N15/11 , C12Q1/6876 , C12N15/111 , C12N2310/11 , C12N2310/14 , C12N2310/31 , C12N2310/315 , C12N2330/30
摘要： The present invention relates to chirally controlled oligonucleotides, chirally controlled oligonucleotide compositions, and the method of making and using the same. The invention specifically encompasses the identification of the source of certain problems with prior methodologies for preparing chiral oligonucleotides, including problems that prohibit preparation of fully chirally controlled compositions, particularly compositions comprising a plurality of oligonucleotide types. In some embodiments, the present invention provides chirally controlled oligonucleotide compositions. In some embodiments, the present invention provides methods of making chirally controlled oligonucleotides and chirally controlled oligonucleotide compositions.
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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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