公开(公告)号：US20240150756A1

公开(公告)日：2024-05-09

申请号：US18305195

申请日：2023-04-21

Applicant: WAVE LIFE SCIENCES LTD.

Inventor： Maria David Frank-Kamenetsky ,  Hailin Yang ,  Aaron Jay Morris ,  Chandra Vargeese ,  Christopher J. Francis

IPC: C12N15/113 ,  A61P25/28 ,  C12N15/115

CPC classification number: C12N15/113 ,  A61P25/28 ,  C12N15/115 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/341 ,  C12N2310/346

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：US20230295617A1

公开(公告)日：2023-09-21

申请号：US18072296

申请日：2022-11-30

Applicant: 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 ,  A61K47/54 ,  A61K31/7088

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

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, docosahexaenoic 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：US20250135036A1

公开(公告)日：2025-05-01

申请号：US18695348

申请日：2022-09-26

Applicant: WAVE LIFE SCIENCES LTD.

Inventor： Christopher Michael Acker ,  Onanong Chivatakarn ,  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 ,  Jayakanthan Kumarasamy ,  Anthony Lamattina ,  Ian Chandler Harding ,  Jesse Turner

IPC: A61K48/00 ,  C12N9/78 ,  C12N15/113 ,  C12N15/87

Abstract: Among other things, the present disclosure provides oligonucleotides, compositions and methods thereof that can bring about specific editing of a target adenosine in a target RNA molecule. Such oligonucleotides, compositions and methods are useful to treat, prevent, or ameliorate MECP2 associated disorders, diseases and syndromes that can benefit from adenosine modification.

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：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：US11634710B2

公开(公告)日：2023-04-25

申请号：US16782021

申请日：2020-02-04

Applicant: WAVE LIFE SCIENCES LTD.

Inventor： Maria David Frank-Kamenetsky ,  Hailin Yang ,  Aaron Jay Morris ,  Chandra Vargeese ,  Christopher J. Francis

IPC: C07H21/02 ,  C12N15/113 ,  C12N15/115 ,  A61P25/28

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：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：US20230329201A1

公开(公告)日：2023-10-19

申请号：US18022509

申请日：2021-08-23

Applicant: WAVE LIFE SCIENCES LTD.

Inventor： Hailin Yang ,  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 ,  Jack David Godfrey ,  Naoki Iwanmoto

IPC: A01K67/027 ,  C12N15/85 ,  C12N9/78 ,  A61K49/00

CPC classification number: A01K67/0275 ,  A61K49/0008 ,  C12N9/78 ,  C12N15/8509 ,  A01K2217/072 ,  A01K2227/105 ,  A01K2267/01 ,  C12N2015/8518

Abstract: Among other things, the present disclosure provides cells and non-human animals engineered to express an ADAR1 polypeptide or a characteristic portion thereof. In some embodiments, the present disclosure provides cells and non-human animals engineered to express a human ADAR1 polypeptide or a characteristic portion thereof. In some embodiments, non-human animals are genetically modified rodents such as mice, rat, etc. In some embodiments, non-human animals are mice. In some embodiments, the present disclosure provides technologies for assessing an agent comprising administering the agent to a cell or non-human animal engineered to express an ADAR1 polypeptide or a characteristic portion thereof. In some embodiments, such a cell or non-human animal is engineered to express a human ADAR1 polypeptide or a characteristic portion thereof. In some embodiments, an agent is a pharmaceutical agent. In some embodiments, an agent is or comprises an oligonucleotide.