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：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：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：US11739325B2

公开(公告)日：2023-08-29

申请号：US16636902

申请日：2018-08-07

Applicant: WAVE LIFE SCIENCES LTD.

Inventor： Chandra Vargeese ,  Zhong Zhong ,  Naoki Iwamoto ,  Jason Jingxin Zhang ,  Jean-Cosme Dodart ,  Yuanjing Liu ,  Pachamuthu Kandasamy ,  Sethumadhavan Divakaramenon ,  Genliang Lu ,  Subramanian Marappan

IPC: C12N15/113 ,  C12N15/11 ,  C12Q1/68

CPC classification number: C12N15/113 ,  C12N15/11 ,  C12N2310/11 ,  C12N2310/12 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/33 ,  C12N2310/341 ,  C12N2310/346

Abstract: Among other things, the present disclosure provides oligonucleotides, compositions, and methods thereof. 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., knockdown ability, stability, delivery, etc. In some embodiments, the oligonucleotides decrease the expression, activity and/or level of a C9orf72 gene, including but not limited to, one comprising a repeat expansion, or a gene product thereof. In some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions, for example, in treatment of C9orf72-related disorders.

公开(公告)号：US20250066775A1

公开(公告)日：2025-02-27

申请号：US18613034

申请日：2024-03-21

Applicant: WAVE LIFE SCIENCES LTD.

Inventor： Chandra Vargeese ,  Zhong Zhong ,  Naoki Iwamoto ,  Jason Jingxin Zhang ,  Jean-Cosme Dodart ,  Yuanjing Liu ,  Pachamuthu Kandasamy ,  Sethumadhavan Divakaramenon ,  Genliang Lu ,  Subramanian Marappan

IPC: C12N15/113 ,  C12N15/11

Abstract: Among other things, the present disclosure provides oligonucleotides, compositions, and methods thereof. 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., knockdown ability, stability, delivery, etc. In some embodiments, the oligonucleotides decrease the expression, activity and/or level of a C9orf72 gene, including but not limited to, one comprising a repeat expansion, or a gene product thereof. In some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions, for example, in treatment of C9orf72-related disorders.

公开(公告)号：US20210032620A1

公开(公告)日：2021-02-04

申请号：US16636902

申请日：2018-08-07

Applicant: WAVE LIFE SCIENCES LTD.

Inventor： Chandra Vargeese ,  Zhong Zhong ,  Naoki Iwamoto ,  Jason Jingxin Zhang ,  Jean-Cosme Dodart ,  Yuanjing Liu ,  Pachamuthu Kandasamy ,  Sethumadhavan Divakaramenon ,  Genliang Lu ,  Subramanian Marappan

IPC: C12N15/11

Abstract: Among other things, the present disclosure provides oligonucleotides, compositions, and methods thereof. 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., knockdown ability, stability, delivery, etc. In some embodiments, the oligonucleotides decrease the expression, activity and/or level of a C9orf72 gene, including but not limited to, one comprising a repeat expansion, or a gene product thereof. In some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions, for example, in treatment of C9orf72-related disorders.