JP2018530560A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2018530560A5 JP2018530560A5 JP2018517603A JP2018517603A JP2018530560A5 JP 2018530560 A5 JP2018530560 A5 JP 2018530560A5 JP 2018517603 A JP2018517603 A JP 2018517603A JP 2018517603 A JP2018517603 A JP 2018517603A JP 2018530560 A5 JP2018530560 A5 JP 2018530560A5
- Authority
- JP
- Japan
- Prior art keywords
- alkyl
- formula
- composition
- item
- myostatin
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Withdrawn
Links
- 125000003729 nucleotide group Chemical group 0.000 claims description 81
- 230000000692 anti-sense effect Effects 0.000 claims description 80
- 239000002773 nucleotide Substances 0.000 claims description 59
- 230000008685 targeting Effects 0.000 claims description 58
- 108010069091 Dystrophin Proteins 0.000 claims description 55
- 125000000217 alkyl group Chemical group 0.000 claims description 54
- 108020004999 messenger RNA Proteins 0.000 claims description 54
- 102000004472 Myostatin Human genes 0.000 claims description 51
- 108010056852 Myostatin Proteins 0.000 claims description 51
- 102000001039 Dystrophin Human genes 0.000 claims description 44
- 230000000295 complement effect Effects 0.000 claims description 42
- 239000000203 mixture Substances 0.000 claims description 37
- 239000003814 drug Substances 0.000 claims description 29
- 101000886562 Homo sapiens Growth/differentiation factor 8 Proteins 0.000 claims description 27
- 102000054677 human MSTN Human genes 0.000 claims description 27
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims description 26
- 229940124597 therapeutic agent Drugs 0.000 claims description 26
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 23
- 208000035475 disorder Diseases 0.000 claims description 23
- 108700024394 Exon Proteins 0.000 claims description 20
- 230000001939 inductive effect Effects 0.000 claims description 15
- 230000035772 mutation Effects 0.000 claims description 15
- -1 methoxytrityl Chemical group 0.000 claims description 14
- 150000001875 compounds Chemical class 0.000 claims description 13
- 230000002401 inhibitory effect Effects 0.000 claims description 13
- 101001053946 Homo sapiens Dystrophin Proteins 0.000 claims description 12
- 108020005067 RNA Splice Sites Proteins 0.000 claims description 12
- 125000002252 acyl group Chemical group 0.000 claims description 12
- 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 description 12
- 210000000663 muscle cell Anatomy 0.000 claims description 9
- 108090000623 proteins and genes Proteins 0.000 claims description 8
- 102000004169 proteins and genes Human genes 0.000 claims description 8
- 230000000694 effects Effects 0.000 claims description 7
- 238000013518 transcription Methods 0.000 claims description 7
- 230000035897 transcription Effects 0.000 claims description 7
- 239000004475 Arginine Substances 0.000 claims description 6
- ODKSFYDXXFIFQN-UHFFFAOYSA-N arginine Natural products OC(=O)C(N)CCCNC(N)=N ODKSFYDXXFIFQN-UHFFFAOYSA-N 0.000 claims description 6
- 125000003710 aryl alkyl group Chemical group 0.000 claims description 6
- 125000005647 linker group Chemical group 0.000 claims description 6
- 108020004707 nucleic acids Proteins 0.000 claims description 6
- 102000039446 nucleic acids Human genes 0.000 claims description 6
- 150000007523 nucleic acids Chemical group 0.000 claims description 6
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 6
- 150000003839 salts Chemical class 0.000 claims description 6
- 239000012634 fragment Substances 0.000 claims description 5
- 102000020313 Cell-Penetrating Peptides Human genes 0.000 claims description 4
- 108010051109 Cell-Penetrating Peptides Proteins 0.000 claims description 4
- 210000004369 blood Anatomy 0.000 claims description 4
- 239000008280 blood Substances 0.000 claims description 4
- 125000004169 (C1-C6) alkyl group Chemical group 0.000 claims description 3
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims description 3
- 210000004899 c-terminal region Anatomy 0.000 claims description 3
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 claims description 3
- 210000001519 tissue Anatomy 0.000 claims description 3
- 102100021886 Activin receptor type-2A Human genes 0.000 claims description 2
- 101000970954 Homo sapiens Activin receptor type-2A Proteins 0.000 claims description 2
- 108020004459 Small interfering RNA Proteins 0.000 claims description 2
- 230000007423 decrease Effects 0.000 claims description 2
- 230000007812 deficiency Effects 0.000 claims description 2
- 210000002027 skeletal muscle Anatomy 0.000 claims description 2
- 238000000034 method Methods 0.000 description 111
- 230000001225 therapeutic effect Effects 0.000 description 6
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical compound O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 description 4
- OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical compound NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 description 4
- UYTPUPDQBNUYGX-UHFFFAOYSA-N guanine Chemical compound O=C1NC(N)=NC2=C1N=CN2 UYTPUPDQBNUYGX-UHFFFAOYSA-N 0.000 description 4
- FDGQSTZJBFJUBT-UHFFFAOYSA-N hypoxanthine Chemical compound O=C1NC=NC2=C1NC=N2 FDGQSTZJBFJUBT-UHFFFAOYSA-N 0.000 description 4
- 239000008194 pharmaceutical composition Substances 0.000 description 4
- RWQNBRDOKXIBIV-UHFFFAOYSA-N thymine Chemical compound CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 description 4
- 0 *N1CCCCC1 Chemical compound *N1CCCCC1 0.000 description 3
- 102000011856 Utrophin Human genes 0.000 description 3
- 108010075653 Utrophin Proteins 0.000 description 3
- 238000009825 accumulation Methods 0.000 description 3
- LRSASMSXMSNRBT-UHFFFAOYSA-N 5-methylcytosine Chemical compound CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 description 2
- MSSXOMSJDRHRMC-UHFFFAOYSA-N 9H-purine-2,6-diamine Chemical compound NC1=NC(N)=C2NC=NC2=N1 MSSXOMSJDRHRMC-UHFFFAOYSA-N 0.000 description 2
- 229930024421 Adenine Natural products 0.000 description 2
- GFFGJBXGBJISGV-UHFFFAOYSA-N Adenine Chemical compound NC1=NC=NC2=C1N=CN2 GFFGJBXGBJISGV-UHFFFAOYSA-N 0.000 description 2
- UGQMRVRMYYASKQ-UHFFFAOYSA-N Hypoxanthine nucleoside Natural products OC1C(O)C(CO)OC1N1C(NC=NC2=O)=C2N=C1 UGQMRVRMYYASKQ-UHFFFAOYSA-N 0.000 description 2
- 229930010555 Inosine Natural products 0.000 description 2
- 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 description 2
- 108091028043 Nucleic acid sequence Proteins 0.000 description 2
- 229960000643 adenine Drugs 0.000 description 2
- 229940104302 cytosine Drugs 0.000 description 2
- 239000003937 drug carrier Substances 0.000 description 2
- 229960003786 inosine Drugs 0.000 description 2
- 229940113082 thymine Drugs 0.000 description 2
- 229940035893 uracil Drugs 0.000 description 2
- 108091034117 Oligonucleotide Proteins 0.000 description 1
- 239000000074 antisense oligonucleotide Substances 0.000 description 1
- 238000012230 antisense oligonucleotides Methods 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021185544A JP2022017594A (ja) | 2015-10-09 | 2021-11-15 | デュシェンヌ型筋ジストロフィーおよび関連障害の処置のための組成物および方法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562239812P | 2015-10-09 | 2015-10-09 | |
| US62/239,812 | 2015-10-09 | ||
| PCT/US2016/056093 WO2017062835A2 (en) | 2015-10-09 | 2016-10-07 | Compositions and methods for treating duchenne muscular dystrophy and related disorders |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021185544A Division JP2022017594A (ja) | 2015-10-09 | 2021-11-15 | デュシェンヌ型筋ジストロフィーおよび関連障害の処置のための組成物および方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2018530560A JP2018530560A (ja) | 2018-10-18 |
| JP2018530560A5 true JP2018530560A5 (enExample) | 2019-10-17 |
Family
ID=58488705
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018517603A Withdrawn JP2018530560A (ja) | 2015-10-09 | 2016-10-07 | デュシェンヌ型筋ジストロフィーおよび関連障害の処置のための組成物および方法 |
| JP2021185544A Pending JP2022017594A (ja) | 2015-10-09 | 2021-11-15 | デュシェンヌ型筋ジストロフィーおよび関連障害の処置のための組成物および方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021185544A Pending JP2022017594A (ja) | 2015-10-09 | 2021-11-15 | デュシェンヌ型筋ジストロフィーおよび関連障害の処置のための組成物および方法 |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20190177723A1 (enExample) |
| EP (2) | EP3858993A1 (enExample) |
| JP (2) | JP2018530560A (enExample) |
| CN (1) | CN108699555A (enExample) |
| BR (1) | BR112018007066A2 (enExample) |
| EA (1) | EA201890908A1 (enExample) |
| IL (2) | IL258069A (enExample) |
| MA (1) | MA45819A (enExample) |
| WO (1) | WO2017062835A2 (enExample) |
Families Citing this family (52)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2852549T3 (es) | 2005-02-09 | 2021-09-13 | Sarepta Therapeutics Inc | Composición antisentido para tratamiento de la atrofia muscular |
| TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| US20130085139A1 (en) | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
| MA41795A (fr) | 2015-03-18 | 2018-01-23 | Sarepta Therapeutics Inc | Exclusion d'un exon induite par des composés antisens dans la myostatine |
| TW201811807A (zh) * | 2016-06-30 | 2018-04-01 | 美商薩羅塔治療公司 | 用於肌肉萎縮症之外顯子跳躍寡聚物 |
| MX2019006882A (es) * | 2016-12-19 | 2019-08-16 | Sarepta Therapeutics Inc | Conjugados de oligomeros de omision de exon para distrofia muscular. |
| CN110337308B (zh) | 2016-12-19 | 2023-09-01 | 萨勒普塔医疗公司 | 用于肌肉萎缩症的外显子跳跃寡聚体缀合物 |
| SMT202200403T1 (it) * | 2016-12-19 | 2022-11-18 | Sarepta Therapeutics Inc | Coniugati di oligomeri per salto di esone per distrofia muscolare |
| CN111417388A (zh) * | 2017-08-31 | 2020-07-14 | 萨勒普塔医疗公司 | 治疗肌营养不良症的方法 |
| EA201991450A1 (ru) * | 2017-09-22 | 2019-12-30 | Сарепта Терапьютикс, Инк. | Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии |
| EP3672601B1 (en) * | 2017-09-25 | 2023-09-13 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers via fast-flow synthesis |
| EP3717008A1 (en) | 2017-12-01 | 2020-10-07 | Merus N.V. | Use of bispecific antibody and il-15 for combination therapy |
| AU2019216321A1 (en) * | 2018-01-31 | 2020-07-30 | The Board Of Regents Of The University Of Texas System | Compositions and methods for correcting dystrophin mutations in human cardiomyocytes |
| WO2019209764A2 (en) * | 2018-04-26 | 2019-10-31 | Sarepta Therapeutics, Inc. | Exon skipping oligomers and oligomer conjugates for muscular dystrophy |
| CA3098624A1 (en) * | 2018-05-11 | 2019-11-14 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| US10758629B2 (en) * | 2018-05-29 | 2020-09-01 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| EP4219717A3 (en) * | 2018-06-13 | 2023-12-20 | Sarepta Therapeutics, Inc. | Exon skipping oligomers for muscular dystrophy |
| WO2019241385A2 (en) * | 2018-06-13 | 2019-12-19 | Sarepta Therapeutics, Inc. | Exon skipping oligomers for muscular dystropy |
| JP7579147B2 (ja) * | 2018-06-14 | 2024-11-07 | サレプタ セラピューティクス， インコーポレイテッド | 筋ジストロフィーに対するエクソンスキッピングオリゴマーおよびオリゴマーコンジュゲート |
| TW202449155A (zh) * | 2018-07-27 | 2024-12-16 | 美商薩羅塔治療公司 | 用於肌肉萎縮症之外顯子跳躍寡聚物 |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US12018087B2 (en) | 2018-08-02 | 2024-06-25 | Dyne Therapeutics, Inc. | Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject |
| SG11202100934PA (en) | 2018-08-02 | 2021-02-25 | Dyne Therapeutics Inc | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| KR20210081324A (ko) | 2018-08-02 | 2021-07-01 | 다인 세라퓨틱스, 인크. | 근육 표적화 복합체 및 안면견갑상완 근육 이영양증을 치료하기 위한 그의 용도 |
| GB201812972D0 (en) | 2018-08-09 | 2018-09-26 | Univ Oxford Innovation Ltd | Cell-penetrating peptides |
| GB201812980D0 (en) | 2018-08-09 | 2018-09-26 | Univ Oxford Innovation Ltd | Cell-penetrating peptides |
| CN113286887A (zh) | 2018-11-02 | 2021-08-20 | 比奥马林技术公司 | 用于肌营养不良蛋白外显子跳跃的双特异性反义低聚核苷酸 |
| WO2020118246A1 (en) * | 2018-12-06 | 2020-06-11 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| WO2020115494A1 (en) | 2018-12-07 | 2020-06-11 | Oxford University Innovation Limited | Linkers |
| CA3122281A1 (en) * | 2018-12-13 | 2020-06-18 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| GB201821269D0 (en) * | 2018-12-28 | 2019-02-13 | Nippon Shinyaku Co Ltd | Myostatin signal inhibitor |
| US12215382B2 (en) | 2019-03-01 | 2025-02-04 | The General Hospital Corporation | Liver protective MARC variants and uses thereof |
| JP7687682B2 (ja) * | 2019-04-08 | 2025-06-03 | 国立大学法人東京科学大学 | 筋疾患治療用医薬組成物 |
| AU2020263487A1 (en) | 2019-04-25 | 2021-12-16 | Avidity Biosciences, Inc. | Nucleic acid compositions and methods of multi-exon skipping |
| CN114555095A (zh) * | 2019-05-06 | 2022-05-27 | 反义治疗有限公司 | 使用针对CD49d的抑制性寡核苷酸治疗肌营养不良症的方法 |
| EP4007633A4 (en) * | 2019-08-02 | 2024-05-08 | Research Institute at Nationwide Children's Hospital | EXON 44-TARGETED NUCLEIC ACIDS AND RECOMBINANT ADENOA-ASSOCIATED VIRUS FOR THE TREATMENT OF DYSTROPHIN-BASED MYOPATHIES |
| EP4045637A4 (en) | 2019-10-16 | 2023-11-22 | The Broad Institute, Inc. | TECHNICAL COMPOSITIONS FOR MUSCLE BUILDING |
| US20230108783A1 (en) * | 2019-11-27 | 2023-04-06 | Dtx Pharma, Inc. | Compounds and methods for the treatment of duchenne muscular dystrophy |
| MY209476A (en) * | 2019-12-26 | 2025-07-11 | Nippon Shinyaku Co Ltd | Antisense nucleic acid that induces skipping of exon 50 |
| IL301781A (en) * | 2020-09-30 | 2023-05-01 | Biomarin Tech Bv | Antisense oligonucleotides targeting the exon 51 of dystrophin gene |
| CA3211057A1 (en) * | 2021-02-26 | 2022-09-01 | Knc Laboratories Co., Ltd. | Nucleic acid medicine expressing splicing variant of myostatin |
| JP2024511955A (ja) * | 2021-03-12 | 2024-03-18 | ペプゲン インコーポレイテッド． | ペプチド－オリゴヌクレオチド複合体を用いるデュシェンヌ型筋ジストロフィーの治療法 |
| AU2022309028A1 (en) * | 2021-07-09 | 2024-01-25 | Dyne Therapeutics, Inc. | Muscle targeting complexes and formulations for treating dystrophinopathies |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| CA3252177A1 (en) * | 2022-03-17 | 2023-09-21 | Sarepta Therapeutics, Inc. | MORPHOLINO PHOSPHORODIAMIDATE OLIGOMER CONJUGATE |
| US12071621B2 (en) | 2022-04-05 | 2024-08-27 | Avidity Biosciences, Inc. | Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping |
| IL316143A (en) | 2022-04-15 | 2024-12-01 | Dyne Therapeutics Inc | Muscle targeting complexes and formulations for the treatment of myotonic dystrophy |
| WO2023212193A1 (en) * | 2022-04-27 | 2023-11-02 | The Curators Of The University Of Missouri | Micro-dystrophin for heart protection |
| WO2025072246A1 (en) * | 2023-09-26 | 2025-04-03 | Entrada Therapeutics, Inc. | Compounds and methods for skipping exon 50 in duchenne muscular dystrophy |
| CN120648680A (zh) * | 2024-03-15 | 2025-09-16 | 迦进生物医药(上海)有限公司 | 治疗杜兴型肌营养不良症和贝克型肌营养不良症的药物 |
Family Cites Families (118)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4426330A (en) | 1981-07-20 | 1984-01-17 | Lipid Specialties, Inc. | Synthetic phospholipid compounds |
| US4534899A (en) | 1981-07-20 | 1985-08-13 | Lipid Specialties, Inc. | Synthetic phospholipid compounds |
| US5217866A (en) | 1985-03-15 | 1993-06-08 | Anti-Gene Development Group | Polynucleotide assay reagent and method |
| US5034506A (en) | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
| US5521063A (en) | 1985-03-15 | 1996-05-28 | Antivirals Inc. | Polynucleotide reagent containing chiral subunits and methods of use |
| US5185444A (en) | 1985-03-15 | 1993-02-09 | Anti-Gene Deveopment Group | Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages |
| JP2528107B2 (ja) | 1985-03-15 | 1996-08-28 | サマ−トン，ジエ−ムス | ポリヌクレオチド測定試薬と方法 |
| US5506337A (en) | 1985-03-15 | 1996-04-09 | Antivirals Inc. | Morpholino-subunit combinatorial library and method |
| US5166315A (en) | 1989-12-20 | 1992-11-24 | Anti-Gene Development Group | Sequence-specific binding polymers for duplex nucleic acids |
| US5354844A (en) | 1989-03-16 | 1994-10-11 | Boehringer Ingelheim International Gmbh | Protein-polycation conjugates |
| US5108921A (en) | 1989-04-03 | 1992-04-28 | Purdue Research Foundation | Method for enhanced transmembrane transport of exogenous molecules |
| US5227170A (en) | 1989-06-22 | 1993-07-13 | Vestar, Inc. | Encapsulation process |
| US5356633A (en) | 1989-10-20 | 1994-10-18 | Liposome Technology, Inc. | Method of treatment of inflamed tissues |
| US5013556A (en) | 1989-10-20 | 1991-05-07 | Liposome Technology, Inc. | Liposomes with enhanced circulation time |
| US5527528A (en) | 1989-10-20 | 1996-06-18 | Sequus Pharmaceuticals, Inc. | Solid-tumor treatment method |
| US5580575A (en) | 1989-12-22 | 1996-12-03 | Imarx Pharmaceutical Corp. | Therapeutic drug delivery systems |
| US5469854A (en) | 1989-12-22 | 1995-11-28 | Imarx Pharmaceutical Corp. | Methods of preparing gas-filled liposomes |
| US5264618A (en) | 1990-04-19 | 1993-11-23 | Vical, Inc. | Cationic lipids for intracellular delivery of biologically active molecules |
| JP3220180B2 (ja) | 1991-05-23 | 2001-10-22 | 三菱化学株式会社 | 薬剤含有タンパク質結合リポソーム |
| US5719262A (en) | 1993-11-22 | 1998-02-17 | Buchardt, Deceased; Ole | Peptide nucleic acids having amino acid side chains |
| US5539082A (en) | 1993-04-26 | 1996-07-23 | Nielsen; Peter E. | Peptide nucleic acids |
| US5714331A (en) | 1991-05-24 | 1998-02-03 | Buchardt, Deceased; Ole | Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility |
| WO1993001286A2 (en) | 1991-06-28 | 1993-01-21 | Massachusetts Institute Of Technology | Localized oligonucleotide therapy |
| US5521291A (en) | 1991-09-30 | 1996-05-28 | Boehringer Ingelheim International, Gmbh | Conjugates for introducing nucleic acid into higher eucaryotic cells |
| NZ244306A (en) | 1991-09-30 | 1995-07-26 | Boehringer Ingelheim Int | Composition for introducing nucleic acid complexes into eucaryotic cells, complex containing nucleic acid and endosomolytic agent, peptide with endosomolytic domain and nucleic acid binding domain and preparation |
| FR2692265B1 (fr) | 1992-05-25 | 1996-11-08 | Centre Nat Rech Scient | Composes biologiquement actifs de type phosphotriesters. |
| ATE427968T1 (de) | 1992-08-21 | 2009-04-15 | Univ Bruxelles | Immunoglobuline ohne leichtkette |
| US6005079A (en) | 1992-08-21 | 1999-12-21 | Vrije Universiteit Brussels | Immunoglobulins devoid of light chains |
| US5583020A (en) | 1992-11-24 | 1996-12-10 | Ribozyme Pharmaceuticals, Inc. | Permeability enhancers for negatively charged polynucleotides |
| JP3351476B2 (ja) | 1993-01-22 | 2002-11-25 | 三菱化学株式会社 | リン脂質誘導体及びそれを含有するリポソーム |
| US5395619A (en) | 1993-03-03 | 1995-03-07 | Liposome Technology, Inc. | Lipid-polymer conjugates and liposomes |
| US5994618A (en) | 1997-02-05 | 1999-11-30 | Johns Hopkins University School Of Medicine | Growth differentiation factor-8 transgenic mice |
| WO1994021681A1 (en) | 1993-03-19 | 1994-09-29 | Johns Hopkins University School Of Medicine | Growth differentiation factor-8 |
| US5462854A (en) | 1993-04-19 | 1995-10-31 | Beckman Instruments, Inc. | Inverse linkage oligonucleotides for chemical and enzymatic processes |
| ES2162863T3 (es) | 1993-04-29 | 2002-01-16 | Unilever Nv | Produccion de anticuerpos o fragmentos (funcionalizados) de los mismos derivados de inmunoglobulinas de cadena pesada de camelidae. |
| FR2705099B1 (fr) | 1993-05-12 | 1995-08-04 | Centre Nat Rech Scient | Oligonucléotides phosphorothioates triesters et procédé de préparation. |
| US5534259A (en) | 1993-07-08 | 1996-07-09 | Liposome Technology, Inc. | Polymer compound and coated particle composition |
| US5543158A (en) | 1993-07-23 | 1996-08-06 | Massachusetts Institute Of Technology | Biodegradable injectable nanoparticles |
| US5417978A (en) | 1993-07-29 | 1995-05-23 | Board Of Regents, The University Of Texas System | Liposomal antisense methyl phosphonate oligonucleotides and methods for their preparation and use |
| US5595756A (en) | 1993-12-22 | 1997-01-21 | Inex Pharmaceuticals Corporation | Liposomal compositions for enhanced retention of bioactive agents |
| US5543152A (en) | 1994-06-20 | 1996-08-06 | Inex Pharmaceuticals Corporation | Sphingosomes for enhanced drug delivery |
| US5591721A (en) | 1994-10-25 | 1997-01-07 | Hybridon, Inc. | Method of down-regulating gene expression |
| US5512295A (en) | 1994-11-10 | 1996-04-30 | The Board Of Trustees Of The Leland Stanford Junior University | Synthetic liposomes for enhanced uptake and delivery |
| US5707618A (en) | 1995-03-24 | 1998-01-13 | Genzyme Corporation | Adenovirus vectors for gene therapy |
| PT876165E (pt) | 1995-12-18 | 2006-10-31 | Angiotech Biomaterials Corp | Composicoes de polimeros reticulados e processos para a sua utilizacao |
| US6245747B1 (en) | 1996-03-12 | 2001-06-12 | The Board Of Regents Of The University Of Nebraska | Targeted site specific antisense oligodeoxynucleotide delivery method |
| PT2045322E (pt) * | 1997-07-14 | 2015-10-16 | Université de Liège | Musculatura dupla em mamíferos |
| US6794499B2 (en) | 1997-09-12 | 2004-09-21 | Exiqon A/S | Oligonucleotide analogues |
| US7572582B2 (en) | 1997-09-12 | 2009-08-11 | Exiqon A/S | Oligonucleotide analogues |
| US7084125B2 (en) | 1999-03-18 | 2006-08-01 | Exiqon A/S | Xylo-LNA analogues |
| JP2002543214A (ja) | 1999-05-04 | 2002-12-17 | エクシコン エ／エス | Ｌ−リボ−ｌｎａ類縁体 |
| US6284882B1 (en) | 1999-06-10 | 2001-09-04 | Abbott Laboratories | Myostatin gene promoter and inhibition of activation thereof |
| US6287860B1 (en) | 2000-01-20 | 2001-09-11 | Isis Pharmaceuticals, Inc. | Antisense inhibition of MEKK2 expression |
| EP1191097A1 (en) | 2000-09-21 | 2002-03-27 | Leids Universitair Medisch Centrum | Induction of exon skipping in eukaryotic cells |
| US20050124566A1 (en) | 2001-05-18 | 2005-06-09 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of myostatin gene expression using short interfering nucleic acid (siNA) |
| AU2002334307A1 (en) | 2001-09-04 | 2003-03-18 | Exiqon A/S | Novel lna compositions and uses thereof |
| US6965025B2 (en) | 2001-12-10 | 2005-11-15 | Isis Pharmaceuticals, Inc. | Antisense modulation of connective tissue growth factor expression |
| KR100464261B1 (ko) | 2002-01-24 | 2005-01-03 | 주식회사 파나진 | Ｐｎａ 올리고머를 합성하기 위한 신규한 단량체 및 그의제조방법 |
| KR20030084444A (ko) | 2002-04-26 | 2003-11-01 | 주식회사 파나진 | Ｐｎａ 올리고머를 합성하기 위한 신규한 단량체 및 그의제조방법 |
| US7569575B2 (en) | 2002-05-08 | 2009-08-04 | Santaris Pharma A/S | Synthesis of locked nucleic acid derivatives |
| WO2004043977A2 (en) | 2002-11-05 | 2004-05-27 | Isis Pharmaceuticals, Inc. | 2’-fluoro substituted oligomeric compounds and compositions for use in gene modulations |
| AU2003225410A1 (en) | 2003-03-21 | 2004-10-11 | Academisch Ziekenhuis Leiden | Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure |
| EP1629105B1 (en) * | 2003-04-29 | 2010-09-01 | AVI BioPharma, Inc. | Compositions for enhancing transport and antisense efficacy of nucleic acid analog into cells |
| US7211668B2 (en) | 2003-07-28 | 2007-05-01 | Panagene, Inc. | PNA monomer and precursor |
| EP1766010B1 (en) | 2004-06-28 | 2011-02-16 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| US7838657B2 (en) | 2004-12-03 | 2010-11-23 | University Of Massachusetts | Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences |
| NZ538097A (en) | 2005-02-07 | 2006-07-28 | Ovita Ltd | Method and compositions for improving wound healing |
| ES2852549T3 (es) * | 2005-02-09 | 2021-09-13 | Sarepta Therapeutics Inc | Composición antisentido para tratamiento de la atrofia muscular |
| CA2605512A1 (en) | 2005-04-22 | 2006-10-26 | Academisch Ziekenhuis Leiden | Modulation of exon recognition in pre-mrna by interfering with the binding of sr proteins and by interfering with secondary rna structure. |
| AP2007004243A0 (en) | 2005-04-25 | 2007-12-31 | Pfizer | Antibodies to myostatin |
| CA2538208A1 (en) | 2005-05-04 | 2006-11-04 | Universite Laval | Modulation of myostatin and use thereof in cell transplantation-based treatment of muscle disease |
| DE202005008426U1 (de) | 2005-05-31 | 2005-09-15 | Ricon Sieb Und Foerdertechnik | Fördervorrichtung mit Fördertuch |
| WO2007002390A2 (en) | 2005-06-23 | 2007-01-04 | Isis Pharmaceuticals, Inc. | Compositions and methods for modulation of smn2 splicing |
| WO2007003216A1 (en) | 2005-07-06 | 2007-01-11 | Universidad Autónoma de Madrid | Anti-ccr7 receptor antibodies for the treatment of cancer |
| HUE035799T2 (en) | 2006-05-10 | 2018-05-28 | Sarepta Therapeutics Inc | Cationic oligonucleotide analogues containing subunits |
| EP1857548A1 (en) | 2006-05-19 | 2007-11-21 | Academisch Ziekenhuis Leiden | Means and method for inducing exon-skipping |
| US8097596B2 (en) * | 2006-06-30 | 2012-01-17 | Lakewood-Amedex, Inc. | Compositions and methods for the treatment of muscle wasting |
| US7892824B2 (en) | 2007-01-18 | 2011-02-22 | University Of Missouri-Columbia | Synthetic mini/micro-dystrophin genes to restore nNOS to the sarcolemma |
| TWI782836B (zh) | 2007-02-02 | 2022-11-01 | 美商艾瑟勒朗法瑪公司 | 衍生自ActRIIB的變體與其用途 |
| US20100016215A1 (en) | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| WO2009005793A2 (en) * | 2007-06-29 | 2009-01-08 | Avi Biopharma, Inc. | Tissue specific peptide conjugates and methods |
| WO2009008725A2 (en) | 2007-07-12 | 2009-01-15 | Prosensa Technologies B.V. | Molecules for targeting compounds to various selected organs, tissues or tumor cells |
| US7935816B2 (en) | 2007-10-25 | 2011-05-03 | Gene Tools, Llc | Molecular transporter compositions comprising dendrimeric oligoguanidine with a triazine core that facilitate delivery into cells in vivo |
| PL2203173T3 (pl) | 2007-10-26 | 2016-06-30 | Academisch Ziekenhuis Leiden | Środki i sposoby przeciwdziałania zaburzeniom mięśni |
| MX2010004955A (es) | 2007-11-15 | 2010-06-30 | Avi Biopharma Inc | Metodo de sintesis de oligomeros de morfolina. |
| US8076476B2 (en) | 2007-11-15 | 2011-12-13 | Avi Biopharma, Inc. | Synthesis of morpholino oligomers using doubly protected guanine morpholino subunits |
| WO2009088895A2 (en) | 2008-01-04 | 2009-07-16 | Cue Acoustics, Inc. | Audio device with integrated switching power supply |
| EP2119783A1 (en) | 2008-05-14 | 2009-11-18 | Prosensa Technologies B.V. | Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means |
| US8236557B2 (en) | 2008-05-28 | 2012-08-07 | University Of Missouri-Columbia | Hybrid-AAV vectors to deliver large gene expression cassette |
| HRP20191109T1 (hr) | 2008-08-14 | 2019-09-20 | Acceleron Pharma Inc. | Gdf klopke za uporabu u liječenju anemije |
| US8084601B2 (en) | 2008-09-11 | 2011-12-27 | Royal Holloway And Bedford New College Royal Holloway, University Of London | Oligomers |
| KR20180118828A (ko) | 2008-10-24 | 2018-10-31 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | Dmd를 위한 다중 엑손 스키핑 조성물 |
| CN102625840A (zh) | 2009-04-10 | 2012-08-01 | 肌肉学研究协会 | 用于治疗疾病的三环dna反义寡核苷酸、组合物和方法 |
| WO2010120820A1 (en) | 2009-04-13 | 2010-10-21 | Isis Pharmaceuticals, Inc. | Compositions and methods for modulation of smn2 splicing |
| WO2010123369A1 (en) | 2009-04-24 | 2010-10-28 | Prosensa Technologies B.V. | Oligonucleotide comprising an inosine for treating dmd |
| SG174273A1 (en) | 2009-04-27 | 2011-10-28 | Novartis Ag | Compositions and methods for increasing muscle growth |
| WO2010129406A2 (en) * | 2009-05-04 | 2010-11-11 | The Johns Hopkins University | Methods of promoting muscle growth |
| KR20200047790A (ko) | 2009-06-17 | 2020-05-07 | 바이오젠 엠에이 인코포레이티드 | 대상에게서 ｓｍｎ２ 스플라이싱을 조정하기 위한 조성물 및 방법 |
| EP3431603A1 (en) | 2009-11-12 | 2019-01-23 | The University Of Western Australia | Antisense molecules and methods for treating pathologies |
| US8802642B2 (en) | 2010-04-28 | 2014-08-12 | Iowa State University Research Foundation, Inc. | Spinal muscular atrophy treatment via targeting SMN2 catalytic core |
| CN107353317A (zh) | 2010-05-28 | 2017-11-17 | 萨勒普塔医疗公司 | 具有修饰的亚基间键和/或端基的寡核苷酸类似物 |
| TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| KR101764462B1 (ko) | 2010-09-30 | 2017-08-02 | 니뽄 신야쿠 가부시키가이샤 | 모르폴리노 핵산 유도체 |
| EP2672977A1 (en) | 2011-02-08 | 2013-12-18 | The Charlotte-Mecklenburg Hospital Authority d/b/a Carolina Healthcare System | Antisense oligonucleotides |
| WO2012150960A1 (en) | 2011-05-05 | 2012-11-08 | Avi Biopharma, Inc. | Peptide oligonucleotide conjugates |
| US20130085139A1 (en) * | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
| EP2581448B1 (en) | 2011-10-13 | 2015-01-28 | Association Institut de Myologie | Tricyclo-phosphorothioate DNA |
| JP2015500204A (ja) | 2011-11-18 | 2015-01-05 | サレプタ セラピューティクス， インコーポレイテッド | 機能的に修飾されたオリゴヌクレオチドおよびそのサブユニット |
| WO2013078316A1 (en) | 2011-11-23 | 2013-05-30 | Nationwide Children's Hospital, Inc. | Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides |
| CN104024414A (zh) | 2011-12-28 | 2014-09-03 | 日本新药株式会社 | 反义核酸 |
| JP2015509922A (ja) | 2012-01-27 | 2015-04-02 | プロセンサ テクノロジーズ ビー．ブイ．Ｐｒｏｓｅｎｓａ Ｔｅｃｈｎｏｌｏｇｉｅｓ Ｂ．Ｖ． | デュシェンヌ型及びベッカー型筋ジストロフィーの治療のための改善された特徴を有するｒｎａ調節オリゴヌクレオチド |
| CN110257379B (zh) * | 2012-07-03 | 2023-08-11 | 马林生物科技有限公司 | 用于治疗肌肉萎缩症患者的寡核苷酸 |
| EA201591178A1 (ru) | 2012-12-20 | 2015-11-30 | Сарепта Терапьютикс, Инк. | Улучшенные композиции для пропускания экзона для лечения мышечной дистрофии |
| KR20230116945A (ko) | 2013-03-14 | 2023-08-04 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이영양증의 치료를 위한 엑손 스키핑 조성물 |
| BR112015022998A2 (pt) | 2013-03-15 | 2017-11-14 | Sarepta Therapeutics Inc | composições melhoradas para o tratamento de distrofia muscular |
| WO2014172448A2 (en) | 2013-04-17 | 2014-10-23 | Anaptysbio, Inc. | Antibodies directed against activin receptor type ii (actrii) |
| IL300444A (en) * | 2013-09-05 | 2023-04-01 | Sarepta Therapeutics Inc | Inclusion of exon 2 is induced by antisense in acid alpha-glucosidase |
| US10867398B2 (en) | 2017-11-21 | 2020-12-15 | Reliance Core Consulting LLC | Methods, systems, apparatuses and devices for facilitating motion analysis in an environment |
-
2016
- 2016-10-07 WO PCT/US2016/056093 patent/WO2017062835A2/en not_active Ceased
- 2016-10-07 JP JP2018517603A patent/JP2018530560A/ja not_active Withdrawn
- 2016-10-07 MA MA045819A patent/MA45819A/fr unknown
- 2016-10-07 EP EP21151113.4A patent/EP3858993A1/en not_active Withdrawn
- 2016-10-07 EP EP16854468.2A patent/EP3359668A4/en not_active Withdrawn
- 2016-10-07 EA EA201890908A patent/EA201890908A1/ru unknown
- 2016-10-07 US US15/765,466 patent/US20190177723A1/en not_active Abandoned
- 2016-10-07 BR BR112018007066A patent/BR112018007066A2/pt not_active Application Discontinuation
- 2016-10-07 CN CN201680072146.4A patent/CN108699555A/zh active Pending
-
2018
- 2018-03-13 IL IL258069A patent/IL258069A/en unknown
-
2021
- 2021-11-15 JP JP2021185544A patent/JP2022017594A/ja active Pending
-
2022
- 2022-01-27 IL IL290160A patent/IL290160A/en unknown
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2018530560A5 (enExample) | ||
| JP6954843B2 (ja) | ペプチドオリゴヌクレオチドコンジュゲート | |
| AU2013306006B2 (en) | Polynucleotides having bioreversible groups | |
| JPWO2023026994A5 (enExample) | ||
| JP2019516719A5 (enExample) | ||
| TW201840339A (zh) | 用於肌肉萎縮症之外顯子跳躍寡聚物結合物 | |
| US10758558B2 (en) | Hybrid oligonucleotides and uses thereof | |
| TW202332470A (zh) | 用於肌肉萎縮症之外顯子跳躍寡聚物結合物 | |
| KR20130118227A (ko) | 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체 | |
| TW201828996A (zh) | 用於肌肉萎縮症之外顯子跳躍寡聚物結合物 | |
| TW201018471A (en) | Multiple exon skipping compositions for DMD | |
| JP2009515823A5 (enExample) | ||
| TWI740866B (zh) | 肽寡核苷酸結合物 | |
| JP2016513976A5 (enExample) | ||
| US20220251551A1 (en) | Exon skipping oligomers for muscular dystrophy | |
| JP2021532784A (ja) | アンチセンス送達のための三量体ペプチド | |
| CN107074911B (zh) | 肽核酸单体和低聚体 | |
| JP2018518148A (ja) | ミオスタチンにおけるアンチセンスに誘導されるエクソンの排除 | |
| AU2016317667A1 (en) | Modified antisense oligomers for exon inclusion in spinal muscular atrophy | |
| JP2018525015A5 (enExample) | ||
| US20250179487A1 (en) | Exon skipping oligomer conjugates for muscular dystrophy | |
| JP2018518167A5 (enExample) | ||
| US20230073008A1 (en) | Antisense nucleic acid that induces skipping of exon 50 | |
| JP2020521491A5 (enExample) | ||
| WO2018056871A1 (ru) | Аналоги олигонуклеотидов как средства коррекции сплайсинга для лечения мышечной дистрофии дюшена |