JP2018525015A5 - - Google Patents
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- JP2018525015A5 JP2018525015A5 JP2018510718A JP2018510718A JP2018525015A5 JP 2018525015 A5 JP2018525015 A5 JP 2018525015A5 JP 2018510718 A JP2018510718 A JP 2018510718A JP 2018510718 A JP2018510718 A JP 2018510718A JP 2018525015 A5 JP2018525015 A5 JP 2018525015A5
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- JP
- Japan
- Prior art keywords
- alkyl
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- Prior art date
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- 150000001875 compounds Chemical class 0.000 claims 23
- 230000008685 targeting Effects 0.000 claims 22
- -1 4-aminopiperidin-1-yl moiety Chemical group 0.000 claims 10
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical compound O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 claims 8
- 125000002252 acyl group Chemical group 0.000 claims 8
- 230000000692 anti-sense effect Effects 0.000 claims 8
- OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical compound NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 claims 8
- RWQNBRDOKXIBIV-UHFFFAOYSA-N thymine Chemical compound CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 claims 8
- 125000005647 linker group Chemical group 0.000 claims 6
- 125000002221 trityl group Chemical group [H]C1=C([H])C([H])=C([H])C([H])=C1C([*])(C1=C(C(=C(C(=C1[H])[H])[H])[H])[H])C1=C([H])C([H])=C([H])C([H])=C1[H] 0.000 claims 6
- 125000003729 nucleotide group Chemical group 0.000 claims 5
- LRSASMSXMSNRBT-UHFFFAOYSA-N 5-methylcytosine Chemical compound CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 claims 4
- 229940104302 cytosine Drugs 0.000 claims 4
- 239000002773 nucleotide Substances 0.000 claims 4
- 150000003839 salts Chemical class 0.000 claims 4
- 229940113082 thymine Drugs 0.000 claims 4
- 229940035893 uracil Drugs 0.000 claims 4
- 108010051109 Cell-Penetrating Peptides Proteins 0.000 claims 3
- 102000020313 Cell-Penetrating Peptides Human genes 0.000 claims 3
- 125000000217 alkyl group Chemical group 0.000 claims 3
- 125000003710 aryl alkyl group Chemical group 0.000 claims 3
- 210000004899 c-terminal region Anatomy 0.000 claims 3
- 239000012634 fragment Substances 0.000 claims 3
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 claims 3
- 101000617738 Homo sapiens Survival motor neuron protein Proteins 0.000 claims 2
- 108091093037 Peptide nucleic acid Proteins 0.000 claims 2
- 102100021947 Survival motor neuron protein Human genes 0.000 claims 2
- ANCLJVISBRWUTR-UHFFFAOYSA-N diaminophosphinic acid Chemical compound NP(N)(O)=O ANCLJVISBRWUTR-UHFFFAOYSA-N 0.000 claims 2
- UYTPUPDQBNUYGX-UHFFFAOYSA-N guanine Chemical compound O=C1NC(N)=NC2=C1N=CN2 UYTPUPDQBNUYGX-UHFFFAOYSA-N 0.000 claims 2
- FDGQSTZJBFJUBT-UHFFFAOYSA-N hypoxanthine Chemical compound O=C1NC=NC2=C1NC=N2 FDGQSTZJBFJUBT-UHFFFAOYSA-N 0.000 claims 2
- 108020004999 messenger RNA Proteins 0.000 claims 2
- 239000000203 mixture Substances 0.000 claims 2
- 108020004707 nucleic acids Proteins 0.000 claims 2
- 150000007523 nucleic acids Chemical class 0.000 claims 2
- 102000039446 nucleic acids Human genes 0.000 claims 2
- 208000002320 spinal muscular atrophy Diseases 0.000 claims 2
- MSSXOMSJDRHRMC-UHFFFAOYSA-N 9H-purine-2,6-diamine Chemical compound NC1=NC(N)=C2NC=NC2=N1 MSSXOMSJDRHRMC-UHFFFAOYSA-N 0.000 claims 1
- 229930024421 Adenine Natural products 0.000 claims 1
- 239000004475 Arginine Substances 0.000 claims 1
- 239000005977 Ethylene Substances 0.000 claims 1
- UGQMRVRMYYASKQ-UHFFFAOYSA-N Hypoxanthine nucleoside Natural products OC1C(O)C(CO)OC1N1C(NC=NC2=O)=C2N=C1 UGQMRVRMYYASKQ-UHFFFAOYSA-N 0.000 claims 1
- 229930010555 Inosine Natural products 0.000 claims 1
- UGQMRVRMYYASKQ-KQYNXXCUSA-N Inosine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C2=NC=NC(O)=C2N=C1 UGQMRVRMYYASKQ-KQYNXXCUSA-N 0.000 claims 1
- 125000002777 acetyl group Chemical group [H]C([H])([H])C(*)=O 0.000 claims 1
- 229960000643 adenine Drugs 0.000 claims 1
- GFFGJBXGBJISGV-UHFFFAOYSA-N adenyl group Chemical group N1=CN=C2N=CNC2=C1N GFFGJBXGBJISGV-UHFFFAOYSA-N 0.000 claims 1
- ODKSFYDXXFIFQN-UHFFFAOYSA-N arginine Natural products OC(=O)C(N)CCCNC(N)=N ODKSFYDXXFIFQN-UHFFFAOYSA-N 0.000 claims 1
- 125000003236 benzoyl group Chemical group [H]C1=C([H])C([H])=C(C([H])=C1[H])C(*)=O 0.000 claims 1
- 210000004027 cell Anatomy 0.000 claims 1
- 230000000295 complement effect Effects 0.000 claims 1
- DMSZORWOGDLWGN-UHFFFAOYSA-N ctk1a3526 Chemical compound NP(N)(N)=O DMSZORWOGDLWGN-UHFFFAOYSA-N 0.000 claims 1
- 125000001495 ethyl group Chemical group [H]C([H])([H])C([H])([H])* 0.000 claims 1
- 229960003786 inosine Drugs 0.000 claims 1
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- PTMHPRAIXMAOOB-UHFFFAOYSA-L phosphoramidate Chemical compound NP([O-])([O-])=O PTMHPRAIXMAOOB-UHFFFAOYSA-L 0.000 claims 1
- 108090000765 processed proteins & peptides Proteins 0.000 claims 1
- 125000003696 stearoyl group Chemical group O=C([*])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])[H] 0.000 claims 1
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562211678P | 2015-08-28 | 2015-08-28 | |
| US62/211,678 | 2015-08-28 | ||
| US201662379696P | 2016-08-25 | 2016-08-25 | |
| US62/379,696 | 2016-08-25 | ||
| PCT/US2016/048965 WO2017040271A1 (en) | 2015-08-28 | 2016-08-26 | Modified antisense oligomers for exon inclusion in spinal muscular atrophy |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020216610A Division JP2021048877A (ja) | 2015-08-28 | 2020-12-25 | 脊髄性筋萎縮症におけるエクソン包含のための修飾アンチセンスオリゴマー |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018525015A JP2018525015A (ja) | 2018-09-06 |
| JP2018525015A5 true JP2018525015A5 (enExample) | 2019-10-03 |
| JP6987041B2 JP6987041B2 (ja) | 2021-12-22 |
Family
ID=56883867
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018510718A Active JP6987041B2 (ja) | 2015-08-28 | 2016-08-26 | 脊髄性筋萎縮症におけるエクソン包含のための修飾アンチセンスオリゴマー |
| JP2020216610A Withdrawn JP2021048877A (ja) | 2015-08-28 | 2020-12-25 | 脊髄性筋萎縮症におけるエクソン包含のための修飾アンチセンスオリゴマー |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020216610A Withdrawn JP2021048877A (ja) | 2015-08-28 | 2020-12-25 | 脊髄性筋萎縮症におけるエクソン包含のための修飾アンチセンスオリゴマー |
Country Status (8)
| Country | Link |
|---|---|
| US (3) | US10905709B2 (enExample) |
| EP (1) | EP3341480A1 (enExample) |
| JP (2) | JP6987041B2 (enExample) |
| AU (1) | AU2016317667A1 (enExample) |
| CA (1) | CA2996164A1 (enExample) |
| HK (1) | HK1257498A1 (enExample) |
| MA (1) | MA42695A (enExample) |
| WO (1) | WO2017040271A1 (enExample) |
Families Citing this family (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2016040748A1 (en) | 2014-09-12 | 2016-03-17 | Ionis Pharmaceuticals, Inc. | Compositions and methods for detection of smn protein in a subject and treatment of a subject |
| AU2016317667A1 (en) | 2015-08-28 | 2018-03-22 | Sarepta Therapeutics, Inc. | Modified antisense oligomers for exon inclusion in spinal muscular atrophy |
| WO2019079637A2 (en) * | 2017-10-18 | 2019-04-25 | Sarepta Therapeutics, Inc. | ANTISENSE OLIGOMERIC COMPOUNDS |
| US12215382B2 (en) | 2019-03-01 | 2025-02-04 | The General Hospital Corporation | Liver protective MARC variants and uses thereof |
| CA3153902A1 (en) | 2019-10-16 | 2021-04-22 | Pardis SABETI | Engineered muscle targeting compositions |
| PH12022552258A1 (en) | 2020-02-28 | 2023-11-20 | Ionis Pharmaceuticals Inc | Compounds and methods for modulating smn2 |
| US20230159949A1 (en) | 2020-07-22 | 2023-05-25 | The Broad Institute, Inc. | Engineered muscle targeting compositions |
Family Cites Families (29)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| FR2802206B1 (fr) | 1999-12-14 | 2005-04-22 | Sod Conseils Rech Applic | Derives de 4-aminopiperidine et leur utilisation en tant que medicament |
| WO2001083740A2 (en) | 2000-05-04 | 2001-11-08 | Avi Biopharma, Inc. | Splice-region antisense composition and method |
| ES2397113T3 (es) * | 2005-06-23 | 2013-03-04 | Isis Pharmaceuticals, Inc. | Composiciones y procedimientos para modular el corte y empalme de SMN2 |
| US8067571B2 (en) | 2005-07-13 | 2011-11-29 | Avi Biopharma, Inc. | Antibacterial antisense oligonucleotide and method |
| ES2645410T3 (es) * | 2006-05-10 | 2017-12-05 | Sarepta Therapeutics, Inc. | Análogos de oligonucleótidos que tienen enlaces intersubunitarios catiónicos |
| JP6047270B2 (ja) | 2006-08-11 | 2016-12-21 | バイオマリン テクノロジーズ ベー．フェー． | Ｄｎａリピートの不安定性に関連した遺伝的障害を治療するための方法及び手段 |
| US20100016215A1 (en) | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| US20120149757A1 (en) | 2009-04-13 | 2012-06-14 | Krainer Adrian R | Compositions and methods for modulation of smn2 splicing |
| CN115227709A (zh) * | 2009-06-17 | 2022-10-25 | 冷泉港实验室 | 用于在对象中调节smn2剪接的组合物和方法 |
| US8802642B2 (en) | 2010-04-28 | 2014-08-12 | Iowa State University Research Foundation, Inc. | Spinal muscular atrophy treatment via targeting SMN2 catalytic core |
| JP5585822B2 (ja) | 2010-05-11 | 2014-09-10 | 東レ・ファインケミカル株式会社 | 光学活性ニペコチン酸誘導体の製造方法 |
| KR102182663B1 (ko) * | 2010-05-28 | 2020-11-25 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체 |
| EP2582397A4 (en) | 2010-06-15 | 2014-10-29 | Isis Pharmaceuticals Inc | COMPOUNDS AND METHOD FOR MODULATING INTERACTION BETWEEN PROTEINS AND TARGET NUCLEIC ACIDS |
| US9161948B2 (en) | 2011-05-05 | 2015-10-20 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| IL273838B (en) * | 2011-05-05 | 2022-09-01 | Sarepta Therapeutics Inc | Peptide and oligonucleotide conjugates, preparations containing them and their uses |
| US20140298496A1 (en) | 2011-06-23 | 2014-10-02 | Cold Spring Harbor Laboratory | Phenocopy model of disease |
| AU2012345638C1 (en) * | 2011-11-30 | 2018-10-18 | Sarepta Therapeutics, Inc. | Induced exon inclusion in spinal muscle atrophy |
| WO2013082548A1 (en) | 2011-11-30 | 2013-06-06 | Sarepta Therapeutics, Inc. | Oligonucleotides for treating expanded repeat diseases |
| EP2788087A4 (en) * | 2011-12-06 | 2015-08-26 | Ohio State Innovation Foundation | Non-ionic, low-osmolar contrast agents for the administration of antisense oligonucleotides and the treatment of diseases |
| JP6542662B2 (ja) * | 2012-03-20 | 2019-07-10 | サレプタ セラピューティクス， インコーポレイテッド | オリゴヌクレオチドアナログのボロン酸結合体 |
| KR20150030205A (ko) * | 2012-05-16 | 2015-03-19 | 라나 테라퓨틱스, 인크. | Smn 유전자 패밀리 발현을 조절하기 위한 조성물 및 방법 |
| WO2013173789A2 (en) * | 2012-05-17 | 2013-11-21 | Isis Pharmaceuticals, Inc. | Antisense oligonucleotide compositions |
| WO2014110291A1 (en) | 2013-01-09 | 2014-07-17 | Isis Pharmaceuticals, Inc. | Compositions and methods for modulation of smn2 splicing in a subject |
| WO2014113540A1 (en) * | 2013-01-16 | 2014-07-24 | Iowa State University Research Foundation, Inc. | A deep intronic target for splicing correction on spinal muscular atrophy gene |
| WO2014169243A2 (en) * | 2013-04-12 | 2014-10-16 | The Curators Of The University Of Missouri | Smn2 element 1 antisense compositions and methods and uses thereof |
| CA2955698A1 (en) * | 2013-07-29 | 2015-02-05 | Universitat Zu Koln | Neurocalcin delta inhibitors and therapeutic and non-therapeutic uses thereof |
| JP2016530887A (ja) * | 2013-09-13 | 2016-10-06 | ザ ユニバーシティ オブ ウェスタン オーストラリア | Ｓｍｎ関連病理を処置するためのアンチセンスオリゴマーおよび方法 |
| US9845469B2 (en) * | 2014-02-10 | 2017-12-19 | Ohio State Innovation Foundation | Antisense oligonucleotides for treatment of spinal muscular atrophy |
| AU2016317667A1 (en) | 2015-08-28 | 2018-03-22 | Sarepta Therapeutics, Inc. | Modified antisense oligomers for exon inclusion in spinal muscular atrophy |
-
2016
- 2016-08-26 AU AU2016317667A patent/AU2016317667A1/en not_active Abandoned
- 2016-08-26 JP JP2018510718A patent/JP6987041B2/ja active Active
- 2016-08-26 WO PCT/US2016/048965 patent/WO2017040271A1/en not_active Ceased
- 2016-08-26 HK HK18116288.7A patent/HK1257498A1/zh unknown
- 2016-08-26 EP EP16762935.1A patent/EP3341480A1/en not_active Withdrawn
- 2016-08-26 CA CA2996164A patent/CA2996164A1/en not_active Abandoned
- 2016-08-26 MA MA042695A patent/MA42695A/fr unknown
- 2016-08-26 US US15/754,782 patent/US10905709B2/en active Active
-
2020
- 2020-11-23 US US16/949,980 patent/US12121532B2/en active Active
- 2020-12-25 JP JP2020216610A patent/JP2021048877A/ja not_active Withdrawn
-
2024
- 2024-08-23 US US18/813,696 patent/US20250241940A1/en active Pending