公开(公告)号：US20220306573A1

公开(公告)日：2022-09-29

申请号：US17046752

申请日：2019-04-11

Applicant: 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.

Inventor： 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: C07C317/28 ,  C07H21/02 ,  C07H21/04 ,  C12N15/113

Abstract: 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：US20190008986A1

公开(公告)日：2019-01-10

申请号：US16139091

申请日：2018-09-23

Applicant: WAVE LIFE SCIENCES LTD.

Inventor： 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: A61K48/00 ,  A61K31/7115 ,  A61K31/712 ,  A61K31/7125 ,  A61P21/00 ,  A61P25/14

CPC classification number: 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

Abstract: 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.

公开(公告)号：US20220401467A1

公开(公告)日：2022-12-22

申请号：US17177111

申请日：2021-02-16

Applicant: WAVE LIFE SCIENCES LTD.

Inventor： Jason Jingxin Zhang ,  Naoki Iwamoto ,  Christopher J. Francis ,  Chandra Vargeese ,  David Charles Donnell Butler ,  Sethumadhavan Divakaramenon ,  Genliang Lu ,  Subramanian Marappan

IPC: A61K31/7125 ,  A61P21/00 ,  C12N15/117 ,  A61K31/7088

Abstract: 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.

公开(公告)号：US20190390197A1

公开(公告)日：2019-12-26

申请号：US16087577

申请日：2017-10-06

Applicant: WAVE LIFE SCIENCES LTD.

Inventor： 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: C12N15/113 ,  A61P25/14 ,  A61P21/00 ,  A61K48/00 ,  A61K31/7125

Abstract: 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.

公开(公告)号：US10450568B2

公开(公告)日：2019-10-22

申请号：US16139091

申请日：2018-09-23

Applicant: WAVE LIFE SCIENCES LTD.

Inventor： 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: A61K31/712 ,  A61K31/7125 ,  C12N15/113 ,  C07H21/02 ,  C07H21/00 ,  A61P25/14 ,  A61P21/00 ,  A61K31/7115 ,  A61K48/00 ,  A61P27/00 ,  C07F9/59

Abstract: 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.

公开(公告)号：US20240117347A1

公开(公告)日：2024-04-11

申请号：US18062422

申请日：2022-12-06

Applicant: WAVE LIFE SCIENCES LTD.

Inventor： 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: C12N15/113 ,  A61K31/7115 ,  A61K31/712 ,  A61K31/7125 ,  A61K48/00 ,  A61P21/00 ,  A61P25/14 ,  A61P27/00 ,  C07F9/59 ,  C07H21/00 ,  C07H21/02

CPC classification number: 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

Abstract: 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.

公开(公告)号：US20230089442A1

公开(公告)日：2023-03-23

申请号：US17439755

申请日：2020-03-19

Applicant: 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.

Inventor： 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

IPC: C07H1/02 ,  C07H21/00 ,  C07H19/04

Abstract: 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.

公开(公告)号：US20190249173A1

公开(公告)日：2019-08-15

申请号：US16098658

申请日：2017-05-03

Applicant: 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.

Inventor： Chandra Vargeese ,  Jason Jingxin Zhang ,  Sethumadhavan Divakaramenon ,  David Charles Donnell Butler ,  Genliang Lu ,  Naoki Iwamoto ,  Hailin Yang ,  Maria David Frank-Kamenetsky ,  Subramanian Marappan

IPC: C12N15/11 ,  A61K31/7088 ,  A61K47/54

CPC classification number: C12N15/111 ,  A61K31/7088 ,  A61K47/542 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/141 ,  C12N2310/16 ,  C12N2310/20 ,  C12N2310/315 ,  C12N2310/3515 ,  C12N2320/32

Abstract: 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：US20190127733A1

公开(公告)日：2019-05-02

申请号：US15766578

申请日：2016-10-07

Applicant: WAVE LIFE SCIENCES LTD.

Inventor： 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: C12N15/113 ,  C07H21/02 ,  C07F9/59 ,  A61P27/00 ,  A61K48/00 ,  A61P21/00

Abstract: 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.

公开(公告)号：US20240174710A1

公开(公告)日：2024-05-30

申请号：US18316932

申请日：2023-05-12

Applicant: WAVE LIFE SCIENCES LTD.

Inventor： 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: C07H21/04 ,  C07C213/00 ,  C07C215/20 ,  C07C215/44 ,  C07D207/08 ,  C07D211/22 ,  C07F7/08 ,  C07F9/6564 ,  C07H19/06

CPC classification number: C07H21/04 ,  C07C213/00 ,  C07C215/20 ,  C07C215/44 ,  C07D207/08 ,  C07D211/22 ,  C07F7/0812 ,  C07F7/083 ,  C07F9/6564 ,  C07H19/06 ,  C07C2601/14

Abstract: 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.