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.

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.

公开(公告)号：US20230220384A1

公开(公告)日：2023-07-13

申请号：US17766677

申请日：2020-10-06

申请人： Prashant MONIAN ,  Chikdu Shakti SHIVAILA ,  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 ,  WAVE LIFE SCIENCES LTD.

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

IPC分类号： C12N15/113

CPC分类号： C12N15/113 ,  C12N2310/14 ,  C12N2310/351 ,  C12N2310/314 ,  C12N2310/322 ,  C12N2310/321 ,  C12N2310/315

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.

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.

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.

公开(公告)号：US20200157545A1

公开(公告)日：2020-05-21

申请号：US16618010

申请日：2018-06-01

申请人： Chandra VARGEESE ,  Naoki IWAMOTO ,  David Charles Donnell BUTLER ,  Subramanian MARAPPAN ,  Genliang LU ,  Jason Jingxin ZHANG ,  Vinod VATHIPADIEKAL ,  Maria David FRANK-KAMENETSKY ,  Luciano Henrique APPONI ,  WAVE LIFE SCIENCES LTD.

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： C12N15/113 ,  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.

公开(公告)号：US20230295619A1

公开(公告)日：2023-09-21

申请号：US17907895

申请日：2021-02-26

申请人： Abbie Madeline MAGUIRE ,  Priyanka Shiva PRAKASHA ,  Naoki IWAMOTO ,  Kenneth Allan LONGO ,  Chandra VARGEESE ,  Kevin KIM ,  Elena DALE ,  Pachamuthu KANDASAMY ,  Mamoru SHIMIZU ,  WAVE LIFE SCIENCES LTD.

发明人： Abbie Madeline Maguire ,  Priyanka Shiva Prakasha ,  Naoki Iwamoto ,  Kenneth Allan Longo ,  Chandra Vargeese ,  Kevin Kim ,  Elena Dale ,  Pachamuthu Kandasamy ,  Mamoru Shimizu

IPC分类号： C12N15/113 ,  A61P25/28

CPC分类号： C12N15/113 ,  A61P25/28 ,  C12N2310/14 ,  C12N2310/314 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322

摘要： Among other things, the present disclosure provides oligonucleotides, compositions, and methods for preventing and/ or treating various conditions, disorders or diseases. In some embodiments, provided technologies comprise nucleobase modifications, sugar modifications, intemucleotidic linkage modifications and/or patterns thereof, and have improved properties, activities and/or selectivities. In some embodiments, provided technologies target MAPT. In some embodiments, the present disclosure provides MAPT oligonucleotides, compositions and methods for preventing and/or treating MAPT-associated conditions, disorders or diseases, such as Alzheimer’s Disease (AD) or Frontotemporal Dementia (FTD).

公开(公告)号：US20240175018A1

公开(公告)日：2024-05-30

申请号：US18178470

申请日：2023-03-03

申请人： WAVE LIFE SCIENCES LTD.

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.

公开(公告)号：US20220145300A1

公开(公告)日：2022-05-12

申请号：US17609330

申请日：2020-05-08

申请人： WAVE LIFE SCIENCES LTD.

发明人： Yuanjing Liu ,  Naoki Iwamoto ,  Chandra Vargeese ,  Zhong Zhong ,  Amy Jada Andreucci

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.

公开(公告)号：US20190008986A1

公开(公告)日：2019-01-10

申请号：US16139091

申请日：2018-09-23

申请人： WAVE LIFE SCIENCES LTD.

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.