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公开(公告)号：US20240279658A1
公开(公告)日：2024-08-22
申请号：US18612623
申请日：2024-03-21
发明人： Chandra Vargeese , Naoki IWAMOTO , Wei LIU , Ngoc Dang Khoa LUU , Pachamuthu KANDASAMY , Subramanian MARAPPAN , Snehlata TRIPATHI
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , C12N2310/14 , C12N2310/314 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/332 , C12N2310/351
摘要： The present disclosure provides double stranded oligonucleotides, compositions, and methods relating thereto. The present disclosure encompasses the recognition that structural elements of double stranded oligonucleotides, such as base sequence, chemical modifications (e.g., modifications of sugar, base, and/or internucleotidic linkages) or patterns thereof, 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. The present disclosure also provides methods for treatment of diseases using provided double stranded oligonucleotide compositions, for example, in RNA interference.
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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：US20220162598A1
公开(公告)日：2022-05-26
申请号：US16869126
申请日：2020-05-07
发明人： Chandra Vargeese , Meena , Nenad Svrzikapa , Susovan Mohapatra , Christopher J. Francis , Gregory L. Verdine , Anna Sokolovska
IPC分类号： 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：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：US10696711B2
公开(公告)日：2020-06-30
申请号：US16182302
申请日：2018-11-06
发明人： Mamoru Shimizu , Takeshi Wada
IPC分类号： C07H23/00 , C07B53/00 , C07H21/04 , C07H1/00 , C07H19/10 , C07H19/207 , C07D405/04 , C07D473/18 , C07D473/34 , C07H19/11 , C07H19/213 , C07D207/08 , C07F7/08
摘要： To provide a chiral reagent or a salt thereof.The chiral reagent has following chemical formula (I). In the formula (I), G1 and G2 are independently a hydrogen atom, a nitro group (—NO2), a halogen atom, a cyano group (—CN), a group of formula (II) or (III), or both G1 and G2 taken together to form a group of formula (IV).
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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：US20240229026A1
公开(公告)日：2024-07-11
申请号：US18313032
申请日：2023-05-05
发明人： David Charles Donnell Butler , Naoki Iwamoto , Meena . , Nenad Svrzikapa , Gregory L. Verdine , Ivan Zlatev
IPC分类号： C12N15/113 , C07C317/28 , C07D295/088 , C07H21/00 , 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：US20240150756A1
公开(公告)日：2024-05-09
申请号：US18305195
申请日：2023-04-21
发明人： Maria David Frank-Kamenetsky , Hailin Yang , Aaron Jay Morris , Chandra Vargeese , Christopher J. Francis
IPC分类号： C12N15/113 , A61P25/28 , C12N15/115
CPC分类号： C12N15/113 , A61P25/28 , C12N15/115 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/341 , C12N2310/346
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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