公开(公告)号：US20200080083A1

公开(公告)日：2020-03-12

申请号：US16551503

申请日：2019-08-26

Applicant: WAVE LIFE SCIENCES LTD.

Inventor： Chandra Vargeese ,  Meena . ,  Nenad Svrzikapa ,  Susovan Mohapatra ,  Christopher J. Francis ,  Gregory L. Verdine ,  Anna Sokolovska

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

Abstract: Among other things, the present disclosure 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. In some embodiments, a chirally controlled oligonucleotide composition has any sequence of bases, and/or pattern or base modifications, sugar modifications, backbone modifications and/or stereochemistry, or combination of these elements, described herein.

公开(公告)号：US20190209604A1

公开(公告)日：2019-07-11

申请号：US16305937

申请日：2017-06-02

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 ,  C12N15/117 ,  A61P21/00

CPC classification number: A61K31/7125 ,  A61K31/7088 ,  A61P21/00 ,  C12N15/117 ,  C12N2310/17 ,  C12N2310/31 ,  C12N2310/315 ,  C12N2310/351 ,  C12N2310/3515

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：US20250154190A1

公开(公告)日：2025-05-15

申请号：US18836993

申请日：2023-02-13

Applicant: WAVE LIFE SCIENCES LTD.

Inventor： Pachamuthu Kandasamy ,  Subramanian Marappan ,  Mamoru Shimizu ,  Chandra Vargeese ,  Jayakanthan Kumarasamy ,  Ik-Hyeon Paik ,  Marissa Sauter ,  Arindom Chatterjee

IPC: C07H19/067 ,  C07D207/08 ,  C07D207/09 ,  C07H19/16 ,  G01N33/53

Abstract: Among other things, the present disclosure provides technologies for stereoselective preparation chiral compounds. In some embodiments, prepared chiral compounds are useful for chirally controlled preparation of oligonucleotides. In some embodiments, such oligonucleotides target transcripts associate with various conditions, disorders or diseases.

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.

公开(公告)号：US20230392137A1

公开(公告)日：2023-12-07

申请号：US17953292

申请日：2022-09-26

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: C12N15/10 ,  C12N15/113

CPC classification number: C12N15/102 ,  C12N15/1135

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.

公开(公告)号：US20230203087A1

公开(公告)日：2023-06-29

申请号：US17926987

申请日：2021-05-24

Applicant: 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.

Inventor： 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: C07H21/04 ,  C07F9/165 ,  C07D295/02

CPC classification number: C07H21/04 ,  C07F9/165 ,  C07D295/02

Abstract: 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：US11603532B2

公开(公告)日：2023-03-14

申请号：US16618010

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 ,  Maria David Frank-Kamenetsky ,  Luciano Henrique Apponi ,  Young Jin Cho

IPC: C12N15/11 ,  C12N15/113 ,  A61K31/712

Abstract: 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：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.