-
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：US11634710B2
公开(公告)日：2023-04-25
申请号：US16782021
申请日：2020-02-04
Applicant: WAVE LIFE SCIENCES LTD.
Inventor： Maria David Frank-Kamenetsky , Hailin Yang , Aaron Jay Morris , Chandra Vargeese , Christopher J. Francis
IPC: C07H21/02 , C12N15/113 , C12N15/115 , A61P25/28
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：US20230089442A1
公开(公告)日：2023-03-23
申请号：US17439755
申请日：2020-03-19
Applicant: Pachamuthu KANDASAMY , Mamoru SHIMIZU , David Charles Donnell BUTLER , Jayakanthan KUMARASAMY , Gopal Reddy BOMMINENI , Mohammed Rowshon ALAM , Sethumadhavan DIVAKARAMENON , Bijay Tilak BHATTARAI , Chandra VARGEESE , Keith Andrew BOWMAN , Stephany Michelle STANDLEY , WAVE LIFE SCIENCES LTD.
Inventor： Pachamuthu Kandasamy , Mamoru Shimizu , David Charles Donnell Butler , Jayakanthan Kumarasamy , Gopal Reddy Bommineni , Mohammed Rowshon Alam , Sethumadhavan Divakaramenon , Bijay Tilak Bhattarai , Chandra Vargeese , Keith Andrew Bowman , Stephany Michelle Standley
Abstract: Among other things, the present disclosure provides technologies for oligonucleotide preparation, particularly chirally controlled oligonucleotide preparation, which technologies provide greatly improved crude purity and yield, and significantly reduce manufacturing costs.
-
公开(公告)号：US11608355B2
公开(公告)日：2023-03-21
申请号：US16648146
申请日：2018-09-17
Applicant: WAVE LIFE SCIENCES LTD.
Inventor： Keith Andrew Bowman , Chandra Vargeese , David Charles Donnell Butler , Pachamuthu Kandasamy , Mohammed Rowshon Alam , Mamoru Shimizu , Stephany Michelle Standley , Vincent Aduda , Gopal Reddy Bommineni , Snehlata Tripathi , Ilia Korboukh
Abstract: Among other things, the present disclosure provides technologies for oligonucleotide preparation, particularly chirally controlled oligonucleotide preparation, which technologies provide greatly improved crude purity and yield, and significantly reduce manufacturing costs.
-
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.
-
Patent Agency Ranking
公开(公告)号：US10724035B2
公开(公告)日：2020-07-28
申请号：US16098836
申请日：2017-05-03
Applicant: WAVE LIFE SCIENCES LTD.
Inventor： Chandra Vargeese , Meena , Nenad Svrzikapa , Susovan Mohapatra , Christopher J. Francis , Gregory L. Verdine , Anna Sokolovska
IPC: A61K31/712 , A61K31/7125 , C12N15/11 , C12N15/113
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：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
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：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：US20240150756A1
公开(公告)日：2024-05-09
申请号：US18305195
申请日：2023-04-21
Applicant: WAVE LIFE SCIENCES LTD.
Inventor： Maria David Frank-Kamenetsky , Hailin Yang , Aaron Jay Morris , Chandra Vargeese , Christopher J. Francis
IPC: C12N15/113 , A61P25/28 , C12N15/115
CPC classification number: C12N15/113 , A61P25/28 , C12N15/115 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/341 , C12N2310/346
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.
-
-
-
-
-
-
-
-
-
Search Results
56
records
(took 0.053 seconds)
