公开(公告)号：US20230295619A1

公开(公告)日：2023-09-21

申请号：US17907895

申请日：2021-02-26

Applicant: Abbie Madeline MAGUIRE ,  Priyanka Shiva PRAKASHA ,  Naoki IWAMOTO ,  Kenneth Allan LONGO ,  Chandra VARGEESE ,  Kevin KIM ,  Elena DALE ,  Pachamuthu KANDASAMY ,  Mamoru SHIMIZU ,  WAVE LIFE SCIENCES LTD.

Inventor： Abbie Madeline Maguire ,  Priyanka Shiva Prakasha ,  Naoki Iwamoto ,  Kenneth Allan Longo ,  Chandra Vargeese ,  Kevin Kim ,  Elena Dale ,  Pachamuthu Kandasamy ,  Mamoru Shimizu

IPC: C12N15/113 ,  A61P25/28

CPC classification number: C12N15/113 ,  A61P25/28 ,  C12N2310/14 ,  C12N2310/314 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322

Abstract: Among other things, the present disclosure provides oligonucleotides, compositions, and methods for preventing and/ or treating various conditions, disorders or diseases. In some embodiments, provided technologies comprise nucleobase modifications, sugar modifications, intemucleotidic linkage modifications and/or patterns thereof, and have improved properties, activities and/or selectivities. In some embodiments, provided technologies target MAPT. In some embodiments, the present disclosure provides MAPT oligonucleotides, compositions and methods for preventing and/or treating MAPT-associated conditions, disorders or diseases, such as Alzheimer’s Disease (AD) or Frontotemporal Dementia (FTD).

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：US20230243854A1

公开(公告)日：2023-08-03

申请号：US18174127

申请日：2023-02-24

Applicant: WAVE LIFE SCIENCES LTD.

Inventor： Ramakrishna Bharadwaj Boyanapalli ,  Jaya Goyal

IPC: G01N33/68

CPC classification number: G01N33/6896 ,  G01N2800/2835 ,  G01N2800/52

Abstract: The present disclosure relates to immunoassay methods and immunoassay kits for determining measuring the amount of wild type huntingtin protein in a sample, e.g., a cerebral spinal fluid sample from a Huntington's disease patient, as well as methods of treating Huntington's disease patients employing such immunoassays.

公开(公告)号：US20230220384A1

公开(公告)日：2023-07-13

申请号：US17766677

申请日：2020-10-06

Applicant: Prashant MONIAN ,  Chikdu Shakti SHIVAILA ,  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 ,  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

IPC: C12N15/113

CPC classification number: C12N15/113 ,  C12N2310/14 ,  C12N2310/351 ,  C12N2310/314 ,  C12N2310/322 ,  C12N2310/321 ,  C12N2310/315

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：US20230203484A1

公开(公告)日：2023-06-29

申请号：US17926967

申请日：2021-05-24

Applicant: WAVE LIFE SCIENCES LTD.

Inventor： Chandra VARGEESE ,  Naoki IWAMOTO ,  Luciano H. APPONI ,  David Charles Donnell BUTLER ,  Pachamuthu KANDASAMY ,  Subramanian MARAPPAN ,  Snehlata TRIPATHI ,  Wei LIU ,  Mugdha BEDEKAR ,  Vinod VATHIPADIEKAL

IPC: C12N15/113

CPC classification number: C12N15/113 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/351

Abstract: The present disclosure provides double stranded oligonucleotides, compositions, and methods relating thereto. The present disclosure encompasses the recognition that structural elements of double stranded oligonucleotides, such as base sequence, chemical modifications (e.g, modifications of sugar, base, and/or internucleotidic linkages) or patterns thereof, 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. The present disclosure also provides methods for treatment of diseases using provided double stranded oligonucleotide compositions, for example, in RNA interference.

公开(公告)号：US20220307019A1

公开(公告)日：2022-09-29

申请号：US17442663

申请日：2020-03-25

Applicant: National University Corporation Tokyo Medical and Dental University ,  WAVE LIFE SCIENCES LTD.

Inventor： Takanori YOKOTA ,  Takeshi WADA ,  Mamoru SHIMIZU

IPC: C12N15/113

Abstract: A double-stranded nucleic acid complex is a double-stranded nucleic acid complex including a first nucleic acid strand and a second nucleic acid strand bonded to each other, the second nucleic acid strand including a complementary region having a base sequence complementary to the first nucleic acid strand; the first nucleic acid strand including natural nucleosides and non-natural nucleosides; some of the nucleosides in at least one nucleic acid strand selected from the group consisting of the first nucleic acid strand and the second nucleic acid strand being bonded together by bonds including asymmetric phosphorus atoms; and absolute configurations of the asymmetric phosphorus atoms being regulated.

公开(公告)号：US11407775B2

公开(公告)日：2022-08-09

申请号：US16084556

申请日：2017-03-13

Applicant: WAVE LIFE SCIENCES LTD.

Inventor： David Charles Donnell Butler ,  Subramanian Marappan ,  Ik-Hyeon Paik

IPC: C07F9/6584 ,  C07H1/06 ,  C07H19/06 ,  C07H19/16

Abstract: The present disclosure, among other things, provides technologies for preparing and purifying phosphoramidites for oligonucleotide synthesis.

公开(公告)号：US20220186217A1

公开(公告)日：2022-06-16

申请号：US17311285

申请日：2019-12-06

Applicant: 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 ,  Khoa Ngoc Dang Luu

IPC: C12N15/113 ,  C12N15/11

Abstract: Among other things, the present disclosure provides designed DMD oligonucleotides, compositions, and methods of use thereof. In some embodiments, the present disclosure provides technologies useful for repairing mutant DMD transcripts by skipping exon 51, so that the transcript can be translated into an internally truncated but at least partially functional Dystrophin protein variant. In some embodiments, the present disclosure provides technologies useful for modulating DMD transcript splicing. In some embodiments, provided technologies can alter splicing of a dystrophin (DMD) DMD transcript. In some embodiments, the present disclosure provides methods for treating diseases, such as muscular dystrophy, including but not limited to Duchenne muscular dystrophy, Becker's muscular dystrophy, etc.

公开(公告)号：US20220127301A1

公开(公告)日：2022-04-28

申请号：US17465238

申请日：2021-09-02

Applicant: WAVE LIFE SCIENCES LTD.

Inventor： Mamoru Shimizu ,  Takeshi Wada

IPC: C07H23/00 ,  C07D405/04 ,  C07D473/18 ,  C07D473/34 ,  C07B53/00 ,  C07H19/11 ,  C07H19/213 ,  C07H21/04 ,  C07D207/08 ,  C07F7/08 ,  C07H1/00 ,  C07H19/10 ,  C07H19/207

Abstract: To provide a chiral reagent or a salt thereof.
The chiral reagent has following chemical formula (I). In the formula (I), G1 and G2 are independently a hydrogen atom, a nitro group (—NO2), a halogen atom, a cyano group (—CN), a group of formula (II) or (III), or both G1 and G2 taken together to form a group of formula (IV).

公开(公告)号：US20220098585A1

公开(公告)日：2022-03-31

申请号：US17426511

申请日：2020-01-30

Applicant: WAVE LIFE SCIENCES LTD.

Inventor： Jeffrey Matthew Brown ,  Shaunna Syu-Mei Berkovitch ,  Naoki Iwamoto ,  Chandra Vargeese ,  Kidist M. Aklilu ,  Maria David Frank-Kamenetsky ,  Duncan Parley Brown

IPC: C12N15/113

Abstract: Among other things, the present disclosure provides oligonucleotides, compositions, and methods for preventing and/or treating various conditions, disorders or diseases. 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 oligonucleotides, compositions and methods for HTT-related conditions, disorders or diseases, such as Huntington's disease.