公开(公告)号：US20230145795A1

公开(公告)日：2023-05-11

申请号：US17605997

申请日：2020-04-24

Applicant: WAVE LIFE SCIENCES LTD.

Inventor： Michael John Byrne ,  Vinod Vathipadiekal ,  Naoki Iwamoto ,  Chandra Vargeese ,  Lankai Guo ,  Andrew Guzior Hoss

IPC: C12N15/113 ,  A61P27/00

CPC classification number: C12N15/1138 ,  A61P27/00 ,  C12N2310/315 ,  C12N2310/351

Abstract: Among other things, the present disclosure provides RHO oligonucleotides, compositions, and methods. In some embodiments, provided oligonucleotides comprise nucleobase modifications, sugar modifications, internucleotidic linkage modifications and/or patterns thereof, and have improved properties, activities and/or selectivities. In some embodiments, the present disclosure provides RHO oligonucleotides, compositions and methods for preventing and/or treating RHO-related conditions, disorders or diseases, such as retinopathy (e.g, retinal degeneration, retinal degenerative disease, retinal degenerative disorder, inherited retinal degenerative disorder, retinitis pigmentosa, autosomal dominant retinitis pigmentosa, etc.).

公开(公告)号：US11643657B2

公开(公告)日：2023-05-09

申请号：US16717986

申请日：2019-12-17

Applicant: WAVE LIFE SCIENCES LTD.

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

IPC: C12N15/113 ,  C07H21/00 ,  C07C317/28 ,  C07D295/088 ,  C12N15/11 ,  C12Q1/6876

CPC classification number: C12N15/113 ,  C07C317/28 ,  C07D295/088 ,  C07H21/00 ,  C12N15/11 ,  C12Q1/6876 ,  C12N15/111 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/31 ,  C12N2310/315 ,  C12N2330/30

Abstract: The present invention relates to chirally controlled oligonucleotides, chirally controlled oligonucleotide compositions, and the method of making and using the same. The invention specifically encompasses the identification of the source of certain problems with prior methodologies for preparing chiral oligonucleotides, including problems that prohibit preparation of fully chirally controlled compositions, particularly compositions comprising a plurality of oligonucleotide types. In some embodiments, the present invention provides chirally controlled oligonucleotide compositions. In some embodiments, the present invention provides methods of making chirally controlled oligonucleotides and chirally controlled oligonucleotide compositions.

公开(公告)号：US11597927B2

公开(公告)日：2023-03-07

申请号：US16618001

申请日：2018-06-01

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 ,  A61K47/54 ,  A61K47/55 ,  A61K45/06

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 N 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：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：US09982257B2

公开(公告)日：2018-05-29

申请号：US14414614

申请日：2013-07-12

Applicant: WAVE LIFE SCIENCES LTD.

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

IPC: C12N15/113 ,  C07C317/28 ,  C07D295/088 ,  C07H21/00 ,  C12N15/11 ,  C12Q1/68

CPC classification number: C12N15/113 ,  C07C317/28 ,  C07D295/088 ,  C07H21/00 ,  C12N15/11 ,  C12N15/111 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/31 ,  C12N2310/315 ,  C12N2330/30 ,  C12Q1/6876

Abstract: The present invention relates to chirally controlled oligonucleotides, chirally controlled oligonucleotide compositions, and the method of making and using the same. The invention specifically encompasses the identification of the source of certain problems with prior methodologies for preparing chiral oligonucleotides, including problems that prohibit preparation of fully chirally controlled compositions, particularly compositions comprising a plurality of oligonucleotide types. In some embodiments, the present invention provides chirally controlled oligonucleotide compositions. In some embodiments, the present invention provides methods of making chirally controlled oligonucleotides and chirally controlled oligonucleotide compositions.

公开(公告)号：US20170037399A1

公开(公告)日：2017-02-09

申请号：US15112146

申请日：2015-01-16

Applicant: WAVE LIFE SCIENCES LTD.

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

IPC: C12N15/113

CPC classification number: C12N15/113 ,  C12N2310/31 ,  C12N2310/315 ,  C12N2320/30

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.

Abstract translation: 本发明涉及选择性设计的手性控制寡核苷酸，手性控制寡核苷酸组合物及其制备和使用方法。 在一些实施方案中，提供的手性控制的寡核苷酸组合物提供核酸聚合物与参考寡核苷酸组合物不同的切割模式。 在一些实施方案中，提供的手性控制寡核苷酸组合物在核酸聚合物的互补序列内提供单位切割。

公开(公告)号：US20250051778A1

公开(公告)日：2025-02-13

申请号：US17605998

申请日：2020-04-24

Applicant: WAVE LIFE SCIENCES LTD.

Inventor： Michael John Byrne ,  Vinod Vathipadiekal ,  Naoki Iwamoto ,  Chandra Vargeese ,  Lankai Guo

IPC: C12N15/113

Abstract: Among other things, the present disclosure provides USH2A oligonucleotides, and compositions and methods of use thereof, for preventing and/or treating various conditions, disorders or diseases. In some embodiments, provided USH2A oligonucleotides comprise nucleobase modifications, sugar modifications, internucleotidic linkage modifications and/or patterns thereof, and have improved properties, activities and/or selectivities. In some embodiments, the present disclosure provides USH2A oligonucleotides, compositions and methods for preventing and/or treating USH2A-related conditions, disorders or diseases, such as Usher Syndrome (e.g., Usher Syndrome Type 2A), atypical Usher syndrome, or nonsyndromic retinitis pigmentosa.

公开(公告)号：US20240384273A1

公开(公告)日：2024-11-21

申请号：US18667824

申请日：2024-05-17

Applicant: Wave Life Sciences Ltd.

Inventor： Chandra Vargeese ,  Naoki Iwamoto ,  Wei Liu ,  Mugdha Bedekar ,  Brett Schrand ,  Priyank Shiva Prakasha ,  Anthony Lamattina ,  Luciano H. Apponi

IPC: C12N15/113

Abstract: The present disclosure provides HSD17B13-related double stranded oligonucleotides, compositions, and methods of using such double stranded oligonucleotides and compositions for preventing and/or treating various conditions, disorders, or diseases associated with expression of HSD17B13. In some embodiments, the provided double stranded oligonucleotides and compositions comprise nucleobase modifications, sugar modifications, internucleotidic linkage modifications and/or patterns thereof, and have improved properties, activities and/or selectivities. In some embodiments, the provided double stranded oligonucleotides and compositions target HSD17B13.

公开(公告)号：US20240026358A1

公开(公告)日：2024-01-25

申请号：US18252029

申请日：2022-03-11

Applicant: WAVE LIFE SCIENCES LTD.

Inventor： 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 ,  Tom Liantang Pu

IPC: C12N15/113

CPC classification number: C12N15/113 ,  C12N2310/322 ,  C12N2310/321 ,  C12N2310/315 ,  C12N2320/34

Abstract: 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.