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：US20240175018A1

公开(公告)日：2024-05-30

申请号：US18178470

申请日：2023-03-03

Applicant: WAVE LIFE SCIENCES LTD.

Inventor： 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: C12N15/113 ,  A61K45/06 ,  A61K47/54 ,  A61K47/55

CPC classification number: C12N15/113 ,  A61K45/06 ,  A61K47/54 ,  A61K47/545 ,  A61K47/548 ,  A61K47/549 ,  A61K47/55 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/32 ,  C12N2310/33 ,  C12N2310/352

Abstract: 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：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：US20220145300A1

公开(公告)日：2022-05-12

申请号：US17609330

申请日：2020-05-08

Applicant: WAVE LIFE SCIENCES LTD.

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

IPC: C12N15/113 ,  A61K31/713 ,  A61P25/28

Abstract: 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

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：US20160347784A1

公开(公告)日：2016-12-01

申请号：US15167669

申请日：2016-05-27

Applicant: WAVE LIFE SCIENCES LTD.

Inventor： Gregory L. Verdine ,  Meena ,  Naoki Iwamoto

IPC: C07H21/00

CPC classification number: A61K31/70 ,  C07F9/65515 ,  C07H21/00

Abstract: 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.

Abstract translation: 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。

公开(公告)号：US12180472B2

公开(公告)日：2024-12-31

申请号：US17016313

申请日：2020-09-09

Applicant: WAVE LIFE SCIENCES LTD.

Inventor： Meena Meena ,  David Butler ,  Naoki Iwamoto ,  Nenad Svrzikapa ,  Gregory L. Verdine ,  Ivan Zlatev

IPC: C12N15/113

Abstract: The present invention 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.

公开(公告)号：US20240175016A1

公开(公告)日：2024-05-30

申请号：US17766680

申请日：2020-10-05

Applicant: WAVE LIFE SCIENCES LTD.

Inventor： Yuanjing Liu ,  Naoki Iwamoto ,  Chandra Vargeese ,  Zhong Zhong ,  Amy Jada Andreucci ,  Susovan Mohapatra

IPC: C12N15/113

CPC classification number: C12N15/113 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3341

Abstract: 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：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：US20240109931A1

公开(公告)日：2024-04-04

申请号：US17960090

申请日：2022-10-04

Applicant: WAVE LIFE SCIENCES LTD.

Inventor： Chandra Vargeese ,  Naoki Iwamoto ,  David Charles Donnell Butler ,  Subramanian Marappan ,  Genliang Lu ,  Jason Jingxin Zhang ,  Vinod Vathipadiekal ,  Maria David Frank-Kamenetsky ,  Luciano Henrique Apponi ,  Hanna Maria Wisniewska-wrona ,  Xiayun Cheng ,  Young Jin Cho

IPC: C07H17/04 ,  C07H19/04 ,  C12N15/113

CPC classification number: C07H17/04 ,  C07H19/04 ,  C12N15/113 ,  C12N2310/14 ,  C12N2310/315

Abstract: Among other things, the present disclosure provides designed APOC3 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.