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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：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：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：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：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：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：US20240174710A1
公开(公告)日：2024-05-30
申请号：US18316932
申请日：2023-05-12
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 , C07C213/00 , C07C215/20 , C07C215/44 , C07D207/08 , C07D211/22 , C07F7/08 , C07F9/6564 , C07H19/06
CPC分类号： C07H21/04 , C07C213/00 , C07C215/20 , C07C215/44 , C07D207/08 , C07D211/22 , C07F7/0812 , C07F7/083 , 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：US20220186217A1
公开(公告)日：2022-06-16
申请号：US17311285
申请日：2019-12-06
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 , Khoa Ngoc Dang Luu
IPC分类号： C12N15/113 , C12N15/11
摘要： Among other things, the present disclosure provides designed DMD oligonucleotides, compositions, and methods of use thereof. In some embodiments, the present disclosure provides technologies useful for repairing mutant DMD transcripts by skipping exon 51, so that the transcript can be translated into an internally truncated but at least partially functional Dystrophin protein variant. In some embodiments, the present disclosure provides technologies useful for modulating DMD transcript splicing. In some embodiments, provided technologies can alter splicing of a dystrophin (DMD) DMD transcript. In some embodiments, the present disclosure provides methods for treating diseases, such as muscular dystrophy, including but not limited to Duchenne muscular dystrophy, Becker's muscular dystrophy, etc.
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公开(公告)号：US20210254062A1
公开(公告)日：2021-08-19
申请号：US17054452
申请日：2019-05-10
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： C12N15/113
摘要： Among other things, the present disclosure provides designed DMD oligonucleotides, compositions, and methods of use GC thereof. In some embodiments, the present disclosure provides technologies useful for repairing mutant DMD transcripts by skipping exon 51 or exon 53, so that the transcript can be translated into an internally truncated but at least partially functional Dystrophin protein variant. In some embodiments, the present disclosure provides technologies useful for modulating DMD transcript splicing. In some embodiments, provided technologies can alter splicing of a dystrophin (DMD) DMD transcript. In some embodiments, the present disclosure provides methods for treating diseases, such as muscular dystrophy, including but not limited to Duchenne muscular dystrophy, Becker's muscular dystrophy, etc.
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公开(公告)号：US11873316B2
公开(公告)日：2024-01-16
申请号：US16463328
申请日：2017-11-22
发明人： David Charles Donnell Butler , Pachamuthu Kandasamy , Subramanian Marappan , Ik-Hyeon Paik , Jayakanthan Kumarasamy , Gopal Reddy Bommineni
IPC分类号： C07H1/06 , C07H19/10 , C07H19/207 , C07H21/00
CPC分类号： C07H1/06 , C07H19/10 , C07H19/207 , C07H21/00
摘要： The present disclosure, among other things, provides technologies for oligonucleotide synthesis. In some embodiments, the present disclosure provides phosphoramidites and methods for synthesis thereof. In some embodiments, provided methods provides higher yields and/or purities. In some embodiments, provided methods remove byproducts without contact with an aqueous solution.
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