JP2024056842A5 - - Google Patents
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- JP2024056842A5 JP2024056842A5 JP2024019301A JP2024019301A JP2024056842A5 JP 2024056842 A5 JP2024056842 A5 JP 2024056842A5 JP 2024019301 A JP2024019301 A JP 2024019301A JP 2024019301 A JP2024019301 A JP 2024019301A JP 2024056842 A5 JP2024056842 A5 JP 2024056842A5
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- Prior art keywords
- compound
- compound according
- wing segment
- modified
- modified oligonucleotide
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- 150000001875 compounds Chemical class 0.000 claims description 192
- 239000002777 nucleoside Substances 0.000 claims description 112
- 125000003835 nucleoside group Chemical group 0.000 claims description 79
- 108091034117 Oligonucleotide Proteins 0.000 claims description 76
- 235000000346 sugar Nutrition 0.000 claims description 67
- 108020004707 nucleic acids Proteins 0.000 claims description 60
- 102000039446 nucleic acids Human genes 0.000 claims description 60
- 150000007523 nucleic acids Chemical class 0.000 claims description 60
- 108010052185 Myotonin-Protein Kinase Proteins 0.000 claims description 35
- 150000003833 nucleoside derivatives Chemical class 0.000 claims description 33
- 230000000295 complement effect Effects 0.000 claims description 28
- 125000002619 bicyclic group Chemical group 0.000 claims description 27
- 239000000203 mixture Substances 0.000 claims description 19
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims description 11
- 201000009340 myotonic dystrophy type 1 Diseases 0.000 claims description 10
- 230000004048 modification Effects 0.000 claims description 9
- 238000012986 modification Methods 0.000 claims description 9
- 150000008163 sugars Chemical class 0.000 claims description 8
- 208000037140 Steinert myotonic dystrophy Diseases 0.000 claims description 6
- 102100022437 Myotonin-protein kinase Human genes 0.000 claims description 5
- 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 4
- 239000003085 diluting agent Substances 0.000 claims description 2
- 239000003814 drug Substances 0.000 claims description 2
- 239000003937 drug carrier Substances 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 238000000034 method Methods 0.000 description 35
- 102000018658 Myotonin-Protein Kinase Human genes 0.000 description 34
- 241001465754 Metazoa Species 0.000 description 25
- 108091032973 (ribonucleotides)n+m Proteins 0.000 description 12
- LRSASMSXMSNRBT-UHFFFAOYSA-N 5-methylcytosine Chemical group CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 description 11
- OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical compound NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 description 11
- ABEXEQSGABRUHS-UHFFFAOYSA-N 16-methylheptadecyl 16-methylheptadecanoate Chemical compound CC(C)CCCCCCCCCCCCCCCOC(=O)CCCCCCCCCCCCCCC(C)C ABEXEQSGABRUHS-UHFFFAOYSA-N 0.000 description 9
- 241000764238 Isis Species 0.000 description 9
- 238000005417 image-selected in vivo spectroscopy Methods 0.000 description 9
- 238000012739 integrated shape imaging system Methods 0.000 description 9
- 108020004999 messenger RNA Proteins 0.000 description 7
- 239000008194 pharmaceutical composition Substances 0.000 description 7
- 206010061533 Myotonia Diseases 0.000 description 4
- 101150086792 CLCN1 gene Proteins 0.000 description 3
- 101100107064 Drosophila melanogaster Zasp52 gene Proteins 0.000 description 3
- 206010068871 Myotonic dystrophy Diseases 0.000 description 3
- 102000006382 Ribonucleases Human genes 0.000 description 2
- 108010083644 Ribonucleases Proteins 0.000 description 2
- 230000001419 dependent effect Effects 0.000 description 2
- 238000007910 systemic administration Methods 0.000 description 2
- ZIEXYIQTFZVRBI-UHFFFAOYSA-N 2-[(4-bromophenyl)methoxy]acetic acid Chemical compound OC(=O)COCC1=CC=C(Br)C=C1 ZIEXYIQTFZVRBI-UHFFFAOYSA-N 0.000 description 1
- 239000000074 antisense oligonucleotide Substances 0.000 description 1
- 238000012230 antisense oligonucleotides Methods 0.000 description 1
- 229940104302 cytosine Drugs 0.000 description 1
- 238000007918 intramuscular administration Methods 0.000 description 1
- 238000007913 intrathecal administration Methods 0.000 description 1
- 238000001990 intravenous administration Methods 0.000 description 1
- 238000007914 intraventricular administration Methods 0.000 description 1
- 210000003205 muscle Anatomy 0.000 description 1
- 238000007911 parenteral administration Methods 0.000 description 1
- 239000002243 precursor Substances 0.000 description 1
- 230000000717 retained effect Effects 0.000 description 1
- 238000007920 subcutaneous administration Methods 0.000 description 1
- 208000024891 symptom Diseases 0.000 description 1
Applications Claiming Priority (6)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361864439P | 2013-08-09 | 2013-08-09 | |
| US61/864,439 | 2013-08-09 | ||
| US201361889337P | 2013-10-10 | 2013-10-10 | |
| US61/889,337 | 2013-10-10 | ||
| JP2019212072A JP7059242B2 (ja) | 2013-08-09 | 2019-11-25 | 筋強直性ジストロフィープロテインキナーゼ（ｄｍｐｋ）の発現を調節するための化合物及び方法 |
| JP2022029819A JP2022071053A (ja) | 2013-08-09 | 2022-02-28 | 筋強直性ジストロフィープロテインキナーゼ（ｄｍｐｋ）の発現を調節するための化合物及び方法 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022029819A Division JP2022071053A (ja) | 2013-08-09 | 2022-02-28 | 筋強直性ジストロフィープロテインキナーゼ（ｄｍｐｋ）の発現を調節するための化合物及び方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2024056842A JP2024056842A (ja) | 2024-04-23 |
| JP2024056842A5 true JP2024056842A5 (enExample) | 2024-05-16 |
Family
ID=52462058
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016533487A Withdrawn JP2016530882A (ja) | 2013-08-09 | 2014-08-11 | 筋強直性ジストロフィープロテインキナーゼ（ｄｍｐｋ）の発現を調節するための化合物及び方法 |
| JP2019212072A Active JP7059242B2 (ja) | 2013-08-09 | 2019-11-25 | 筋強直性ジストロフィープロテインキナーゼ（ｄｍｐｋ）の発現を調節するための化合物及び方法 |
| JP2022029819A Pending JP2022071053A (ja) | 2013-08-09 | 2022-02-28 | 筋強直性ジストロフィープロテインキナーゼ（ｄｍｐｋ）の発現を調節するための化合物及び方法 |
| JP2024019301A Pending JP2024056842A (ja) | 2013-08-09 | 2024-02-13 | 筋強直性ジストロフィープロテインキナーゼ（ｄｍｐｋ）の発現を調節するための化合物及び方法 |
Family Applications Before (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016533487A Withdrawn JP2016530882A (ja) | 2013-08-09 | 2014-08-11 | 筋強直性ジストロフィープロテインキナーゼ（ｄｍｐｋ）の発現を調節するための化合物及び方法 |
| JP2019212072A Active JP7059242B2 (ja) | 2013-08-09 | 2019-11-25 | 筋強直性ジストロフィープロテインキナーゼ（ｄｍｐｋ）の発現を調節するための化合物及び方法 |
| JP2022029819A Pending JP2022071053A (ja) | 2013-08-09 | 2022-02-28 | 筋強直性ジストロフィープロテインキナーゼ（ｄｍｐｋ）の発現を調節するための化合物及び方法 |
Country Status (27)
| Country | Link |
|---|---|
| US (4) | US20160304877A1 (enExample) |
| EP (2) | EP3030658B1 (enExample) |
| JP (4) | JP2016530882A (enExample) |
| KR (1) | KR102589140B1 (enExample) |
| CN (1) | CN105452464A (enExample) |
| AU (2) | AU2014306284B2 (enExample) |
| BR (1) | BR112016002399A2 (enExample) |
| CA (1) | CA2920776A1 (enExample) |
| CL (1) | CL2016000301A1 (enExample) |
| CY (1) | CY1124522T1 (enExample) |
| DK (1) | DK3030658T3 (enExample) |
| ES (1) | ES2895099T3 (enExample) |
| HR (1) | HRP20211365T1 (enExample) |
| HU (1) | HUE055867T2 (enExample) |
| IL (3) | IL243917B (enExample) |
| LT (1) | LT3030658T (enExample) |
| MX (2) | MX380778B (enExample) |
| PH (1) | PH12016500263A1 (enExample) |
| PL (1) | PL3030658T3 (enExample) |
| PT (1) | PT3030658T (enExample) |
| RS (1) | RS62403B1 (enExample) |
| RU (1) | RU2690333C2 (enExample) |
| SG (1) | SG11201600941YA (enExample) |
| SI (1) | SI3030658T1 (enExample) |
| SM (1) | SMT202100554T1 (enExample) |
| TW (1) | TW201536329A (enExample) |
| WO (1) | WO2015021457A2 (enExample) |
Families Citing this family (47)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2011282217B2 (en) | 2010-07-19 | 2015-12-03 | Ionis Pharmaceuticals, Inc. | Modulation of dystrophia myotonica-protein kinase (DMPK) expression |
| US9828582B2 (en) | 2013-03-19 | 2017-11-28 | Duke University | Compositions and methods for the induction and tuning of gene expression |
| TW201536329A (zh) | 2013-08-09 | 2015-10-01 | Isis Pharmaceuticals Inc | 用於調節失養性肌強直蛋白質激酶(dmpk)表現之化合物及方法 |
| WO2016130600A2 (en) | 2015-02-09 | 2016-08-18 | Duke University | Compositions and methods for epigenome editing |
| HK1249140A1 (zh) | 2015-04-03 | 2018-10-26 | Ionis Pharmaceuticals, Inc. | 用於调节tmprss6表达的化合物和方法 |
| WO2017053999A1 (en) | 2015-09-25 | 2017-03-30 | Ionis Pharmaceuticals, Inc. | Conjugated antisense compounds and their use |
| HK1257955A1 (zh) | 2015-11-16 | 2019-11-01 | The Research Institute At Nationwide Children's Hospital | 用於治疗肌联蛋白类肌病和其他肌联蛋白病变的材料和方法 |
| AU2016362282B2 (en) | 2015-11-30 | 2023-03-16 | Duke University | Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use |
| MA45328A (fr) | 2016-04-01 | 2019-02-06 | Avidity Biosciences Llc | Compositions acide nucléique-polypeptide et utilisations de celles-ci |
| US20190127713A1 (en) | 2016-04-13 | 2019-05-02 | Duke University | Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use |
| US11427838B2 (en) | 2016-06-29 | 2022-08-30 | Vertex Pharmaceuticals Incorporated | Materials and methods for treatment of myotonic dystrophy type 1 (DM1) and other related disorders |
| KR102824513B1 (ko) * | 2016-07-05 | 2025-06-25 | 바이오마린 테크놀로지스 비.브이. | 유전적 장애 치료를 위한, 특징이 개선된, 비사이클릭 스캐폴드 모이어티를 포함하는 프리-mrna 스플라이스 스위칭 또는 조정 올리고뉴클레오티드 |
| EP4275747A3 (en) | 2016-07-19 | 2024-01-24 | Duke University | Therapeutic applications of cpf1-based genome editing |
| AU2017368050A1 (en) | 2016-11-29 | 2019-06-20 | Puretech Lyt, Inc. | Exosomes for delivery of therapeutic agents |
| MX2019008199A (es) | 2017-01-06 | 2019-11-25 | Avidity Biosciences Llc | Composiciones de acido nucleico polipeptido y metodos de induccion de la omision de exon. |
| EP3599844A4 (en) * | 2017-06-07 | 2021-01-13 | University of Massachusetts | ANTI-ADAM33 OLIGONUCLEOTIDES AND RELATED PROCESSES |
| GB201711809D0 (en) | 2017-07-21 | 2017-09-06 | Governors Of The Univ Of Alberta | Antisense oligonucleotide |
| EP3662060A2 (en) | 2017-08-03 | 2020-06-10 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| KR20250148679A (ko) | 2017-12-06 | 2025-10-14 | 어비디티 바이오사이언시스 인크. | 근위축증 및 근긴장성 이영양증을 치료하는 조성물 및 방법 |
| KR102824021B1 (ko) | 2018-01-31 | 2025-06-24 | 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 | 2c형 지대근이영양증에 대한 유전자 치료 |
| AU2019218987B2 (en) | 2018-02-12 | 2025-04-24 | Ionis Pharmaceuticals, Inc. | Modified compounds and uses thereof |
| MX2020014119A (es) | 2018-06-29 | 2021-06-18 | Res Inst Nationwide Childrens Hospital | Productos y métodos de virus adenoasociado recombinante para el tratamiento de la distrofia muscular de cinturas 2a. |
| US12370264B1 (en) | 2018-08-02 | 2025-07-29 | Dyne Therapeutics, Inc. | Complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and method of delivering oligonucleotide to a subject |
| 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 |
| US20220193250A1 (en) | 2018-08-02 | 2022-06-23 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US12097263B2 (en) | 2018-08-02 | 2024-09-24 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| EP3829596A4 (en) * | 2018-08-02 | 2022-05-11 | Dyne Therapeutics, Inc. | MUSCLE TARGETED COMPLEXES AND THEIR USES IN THE TREATMENT OF MYOTONIC DYSTROPHY |
| US11911484B2 (en) | 2018-08-02 | 2024-02-27 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| CA3110466A1 (en) | 2018-08-22 | 2020-02-27 | Research Institute At Nationwide Children's Hospital | Recombinant virus products and methods for inhibiting expression of dystrophia myotonica protein kinase and/or interfering with a trinucleotide repeat expansion in the 3' untranslated region of the dmpk gene |
| IL319265A (en) | 2018-12-21 | 2025-04-01 | Avidity Biosciences Inc | Anti-transferrin receptor antibodies and their uses |
| MX2021010356A (es) | 2019-02-26 | 2022-03-04 | Res Inst Nationwide Childrens Hospital | SUMINISTRO DE ß-SARCOGLICANO MEDIANTE VECTORES DE VIRUS ADENOASOCIADO Y EL TRATAMIENTO DE LA DISTROFIA MUSCULAR. |
| EP3959319A4 (en) | 2019-04-25 | 2023-06-07 | Avidity Biosciences, Inc. | NUCLEIC ACID COMPOSITIONS AND METHODS FOR MULTI-EXON SKIPPING |
| CN120400170A (zh) | 2019-08-21 | 2025-08-01 | 全国儿童医院研究所 | Α-肌聚糖的腺相关病毒载体递送和肌营养不良症的治疗 |
| IL296387B2 (en) | 2020-03-19 | 2024-08-01 | Avidity Biosciences Inc | Preparations and methods for the treatment of facial, back and arm muscle atrophy |
| MX2022011880A (es) | 2020-03-27 | 2022-10-20 | Avidity Biosciences Inc | Composiciones y metodos para tratar distrofia muscular. |
| JP2023130528A (ja) * | 2020-06-24 | 2023-09-21 | 田辺三菱製薬株式会社 | Ｐｌｐ１発現を調節するための化合物、方法及び医薬組成物 |
| IL304048A (en) | 2020-12-31 | 2023-08-01 | Dyne Therapeutics Inc | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| US11969475B2 (en) * | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US11633498B2 (en) * | 2021-07-09 | 2023-04-25 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| EP4396351A4 (en) * | 2021-09-01 | 2025-12-24 | Ionis Pharmaceuticals Inc | COMPOUNDS AND METHODS FOR REDUCING DMPK EXPRESSION |
| US11833221B2 (en) | 2021-09-01 | 2023-12-05 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds for reducing DMPK expression |
| KR20240055874A (ko) | 2021-09-16 | 2024-04-29 | 어비디티 바이오사이언시스 인크. | 안면견갑상완 근이영양증을 치료하는 조성물 및 방법 |
| EP4219726A1 (en) | 2021-10-15 | 2023-08-02 | Research Institute at Nationwide Children's Hospital | Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy |
| US12071621B2 (en) | 2022-04-05 | 2024-08-27 | Avidity Biosciences, Inc. | Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping |
| AU2023254846A1 (en) | 2022-04-15 | 2024-10-10 | Dyne Therapeutics, Inc. | Muscle targeting complexes and formulations for treating myotonic dystrophy |
| WO2025206092A1 (ja) * | 2024-03-27 | 2025-10-02 | 田辺三菱製薬株式会社 | Ｍｙｏｒｅｇｕｌｉｎ発現を調節するための化合物及び医薬組成物 |
Family Cites Families (130)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US3687808A (en) | 1969-08-14 | 1972-08-29 | Univ Leland Stanford Junior | Synthetic polynucleotides |
| US5118800A (en) | 1983-12-20 | 1992-06-02 | California Institute Of Technology | Oligonucleotides possessing a primary amino group in the terminal nucleotide |
| FR2567892B1 (fr) | 1984-07-19 | 1989-02-17 | Centre Nat Rech Scient | Nouveaux oligonucleotides, leur procede de preparation et leurs applications comme mediateurs dans le developpement des effets des interferons |
| US5367066A (en) | 1984-10-16 | 1994-11-22 | Chiron Corporation | Oligonucleotides with selectably cleavable and/or abasic sites |
| FR2575751B1 (fr) | 1985-01-08 | 1987-04-03 | Pasteur Institut | Nouveaux nucleosides de derives de l'adenosine, leur preparation et leurs applications biologiques |
| US5506337A (en) | 1985-03-15 | 1996-04-09 | Antivirals Inc. | Morpholino-subunit combinatorial library and method |
| US5034506A (en) | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
| US5166315A (en) | 1989-12-20 | 1992-11-24 | Anti-Gene Development Group | Sequence-specific binding polymers for duplex nucleic acids |
| US5185444A (en) | 1985-03-15 | 1993-02-09 | Anti-Gene Deveopment Group | Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages |
| JP2828642B2 (ja) | 1987-06-24 | 1998-11-25 | ハワード フローレイ インスティテュト オブ イクスペリメンタル フィジオロジー アンド メディシン | ヌクレオシド誘導体 |
| US5175273A (en) | 1988-07-01 | 1992-12-29 | Genentech, Inc. | Nucleic acid intercalating agents |
| US5134066A (en) | 1989-08-29 | 1992-07-28 | Monsanto Company | Improved probes using nucleosides containing 3-dezauracil analogs |
| US5591722A (en) | 1989-09-15 | 1997-01-07 | Southern Research Institute | 2'-deoxy-4'-thioribonucleosides and their antiviral activity |
| WO1991004753A1 (en) | 1989-10-02 | 1991-04-18 | Cetus Corporation | Conjugates of antisense oligonucleotides and therapeutic uses thereof |
| DE69034150T2 (de) | 1989-10-24 | 2005-08-25 | Isis Pharmaceuticals, Inc., Carlsbad | 2'-Modifizierte Oligonukleotide |
| US5130302A (en) | 1989-12-20 | 1992-07-14 | Boron Bilogicals, Inc. | Boronated nucleoside, nucleotide and oligonucleotide compounds, compositions and methods for using same |
| US5681941A (en) | 1990-01-11 | 1997-10-28 | Isis Pharmaceuticals, Inc. | Substituted purines and oligonucleotide cross-linking |
| US5670633A (en) | 1990-01-11 | 1997-09-23 | Isis Pharmaceuticals, Inc. | Sugar modified oligonucleotides that detect and modulate gene expression |
| US5587470A (en) | 1990-01-11 | 1996-12-24 | Isis Pharmaceuticals, Inc. | 3-deazapurines |
| US5459255A (en) | 1990-01-11 | 1995-10-17 | Isis Pharmaceuticals, Inc. | N-2 substituted purines |
| US5646265A (en) | 1990-01-11 | 1997-07-08 | Isis Pharmceuticals, Inc. | Process for the preparation of 2'-O-alkyl purine phosphoramidites |
| GB9009980D0 (en) | 1990-05-03 | 1990-06-27 | Amersham Int Plc | Phosphoramidite derivatives,their preparation and the use thereof in the incorporation of reporter groups on synthetic oligonucleotides |
| DK0455905T3 (da) | 1990-05-11 | 1998-12-07 | Microprobe Corp | Dipsticks til nukleinsyrehybridiseringsassays og fremgangsmåde til kovalent immobilisering af oligonukleotider |
| JPH0874B2 (ja) | 1990-07-27 | 1996-01-10 | アイシス・ファーマシューティカルス・インコーポレーテッド | 遺伝子発現を検出および変調するヌクレアーゼ耐性、ピリミジン修飾オリゴヌクレオチド |
| US5432272A (en) | 1990-10-09 | 1995-07-11 | Benner; Steven A. | Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases |
| US6582908B2 (en) | 1990-12-06 | 2003-06-24 | Affymetrix, Inc. | Oligonucleotides |
| EP0538194B1 (de) | 1991-10-17 | 1997-06-04 | Novartis AG | Bicyclische Nukleoside, Oligonukleotide, Verfahren zu deren Herstellung und Zwischenprodukte |
| US5594121A (en) | 1991-11-07 | 1997-01-14 | Gilead Sciences, Inc. | Enhanced triple-helix and double-helix formation with oligomers containing modified purines |
| TW393513B (en) | 1991-11-26 | 2000-06-11 | Isis Pharmaceuticals Inc | Enhanced triple-helix and double-helix formation with oligomers containing modified pyrimidines |
| DE637965T1 (de) | 1991-11-26 | 1995-12-14 | Gilead Sciences Inc | Gesteigerte bildung von triple- und doppelhelices aus oligomeren mit modifizierten pyrimidinen. |
| US5484908A (en) | 1991-11-26 | 1996-01-16 | Gilead Sciences, Inc. | Oligonucleotides containing 5-propynyl pyrimidines |
| US5359044A (en) | 1991-12-13 | 1994-10-25 | Isis Pharmaceuticals | Cyclobutyl oligonucleotide surrogates |
| FR2687679B1 (fr) | 1992-02-05 | 1994-10-28 | Centre Nat Rech Scient | Oligothionucleotides. |
| US5955265A (en) | 1992-02-06 | 1999-09-21 | Massachusetts Institute Of Technology | DNA sequence encoding the myotonic dystrophy gene and uses thereof |
| US5434257A (en) | 1992-06-01 | 1995-07-18 | Gilead Sciences, Inc. | Binding compentent oligomers containing unsaturated 3',5' and 2',5' linkages |
| EP0577558A2 (de) | 1992-07-01 | 1994-01-05 | Ciba-Geigy Ag | Carbocyclische Nukleoside mit bicyclischen Ringen, Oligonukleotide daraus, Verfahren zu deren Herstellung, deren Verwendung und Zwischenproduckte |
| JPH08504559A (ja) | 1992-12-14 | 1996-05-14 | ハネウエル・インコーポレーテッド | 個別に制御される冗長巻線を有するモータシステム |
| US5552282A (en) * | 1993-02-19 | 1996-09-03 | Baylor College Of Medicine | Diagnosis of myotonic muscular dystrophy |
| ES2107205T3 (es) | 1993-03-30 | 1997-11-16 | Sanofi Sa | Analogos de nucleosidos aciclicos y secuencias oligonucleotidas que los contienen. |
| US5502177A (en) | 1993-09-17 | 1996-03-26 | Gilead Sciences, Inc. | Pyrimidine derivatives for labeled binding partners |
| US5801154A (en) | 1993-10-18 | 1998-09-01 | Isis Pharmaceuticals, Inc. | Antisense oligonucleotide modulation of multidrug resistance-associated protein |
| US5457187A (en) | 1993-12-08 | 1995-10-10 | Board Of Regents University Of Nebraska | Oligonucleotides containing 5-fluorouracil |
| US5446137B1 (en) | 1993-12-09 | 1998-10-06 | Behringwerke Ag | Oligonucleotides containing 4'-substituted nucleotides |
| US5519134A (en) | 1994-01-11 | 1996-05-21 | Isis Pharmaceuticals, Inc. | Pyrrolidine-containing monomers and oligomers |
| US5596091A (en) | 1994-03-18 | 1997-01-21 | The Regents Of The University Of California | Antisense oligonucleotides comprising 5-aminoalkyl pyrimidine nucleotides |
| US5627053A (en) | 1994-03-29 | 1997-05-06 | Ribozyme Pharmaceuticals, Inc. | 2'deoxy-2'-alkylnucleotide containing nucleic acid |
| US5646269A (en) | 1994-04-28 | 1997-07-08 | Gilead Sciences, Inc. | Method for oligonucleotide analog synthesis |
| US5525711A (en) | 1994-05-18 | 1996-06-11 | The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services | Pteridine nucleotide analogs as fluorescent DNA probes |
| US5597909A (en) | 1994-08-25 | 1997-01-28 | Chiron Corporation | Polynucleotide reagents containing modified deoxyribose moieties, and associated methods of synthesis and use |
| US9096636B2 (en) * | 1996-06-06 | 2015-08-04 | Isis Pharmaceuticals, Inc. | Chimeric oligomeric compounds and their use in gene modulation |
| USRE44779E1 (en) | 1997-03-07 | 2014-02-25 | Santaris Pharma A/S | Bicyclonucleoside and oligonucleotide analogues |
| JP3756313B2 (ja) | 1997-03-07 | 2006-03-15 | 武 今西 | 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体 |
| US6770748B2 (en) | 1997-03-07 | 2004-08-03 | Takeshi Imanishi | Bicyclonucleoside and oligonucleotide analogue |
| US6329501B1 (en) | 1997-05-29 | 2001-12-11 | Auburn University | Methods and compositions for targeting compounds to muscle |
| US6794499B2 (en) | 1997-09-12 | 2004-09-21 | Exiqon A/S | Oligonucleotide analogues |
| AU9063398A (en) | 1997-09-12 | 1999-04-05 | Exiqon A/S | Oligonucleotide analogues |
| US6028183A (en) | 1997-11-07 | 2000-02-22 | Gilead Sciences, Inc. | Pyrimidine derivatives and oligonucleotides containing same |
| US6007992A (en) | 1997-11-10 | 1999-12-28 | Gilead Sciences, Inc. | Pyrimidine derivatives for labeled binding partners |
| US20030228597A1 (en) | 1998-04-13 | 2003-12-11 | Cowsert Lex M. | Identification of genetic targets for modulation by oligonucleotides and generation of oligonucleotides for gene modulation |
| US6043352A (en) | 1998-08-07 | 2000-03-28 | Isis Pharmaceuticals, Inc. | 2'-O-Dimethylaminoethyloxyethyl-modified oligonucleotides |
| US6107092A (en) | 1999-03-29 | 2000-08-22 | Isis Pharmaceuticals Inc. | Antisense modulation of SRA expression |
| AU776362B2 (en) | 1999-05-04 | 2004-09-09 | Roche Innovation Center Copenhagen A/S | L-ribo-LNA analogues |
| US6525191B1 (en) | 1999-05-11 | 2003-02-25 | Kanda S. Ramasamy | Conformationally constrained L-nucleosides |
| JP2003509030A (ja) | 1999-09-17 | 2003-03-11 | アイシス・ファーマシューティカルス・インコーポレーテッド | トランスフォーミング成長因子−β発現のアンチセンスオリゴヌクレオチド調節 |
| AU2698301A (en) | 1999-12-30 | 2001-07-16 | K.U. Leuven Research And Development | Cyclohexene nucleic acids |
| WO2001077384A2 (de) | 2000-04-07 | 2001-10-18 | Epigenomics Ag | DETEKTION VON SNPs UND CYTOSIN-METHYLIERUNGEN |
| WO2002001953A1 (en) | 2000-06-30 | 2002-01-10 | Brown University Research Foundation | Methods to screen for antibiotic agents and their use in treatment of opportunistic infections |
| US20030207804A1 (en) | 2001-05-25 | 2003-11-06 | Muthiah Manoharan | Modified peptide nucleic acids |
| EP1499627A2 (en) | 2001-07-03 | 2005-01-26 | ISIS Pharmaceuticals, Inc. | Nuclease resistant chimeric oligonucleotides |
| US20030158403A1 (en) | 2001-07-03 | 2003-08-21 | Isis Pharmaceuticals, Inc. | Nuclease resistant chimeric oligonucleotides |
| US7407943B2 (en) | 2001-08-01 | 2008-08-05 | Isis Pharmaceuticals, Inc. | Antisense modulation of apolipoprotein B expression |
| AU2002326589B2 (en) | 2001-08-07 | 2008-06-05 | University Of Delaware | Compositions and methods for the prevention and treatment of Huntington's disease |
| US20060009409A1 (en) * | 2002-02-01 | 2006-01-12 | Woolf Tod M | Double-stranded oligonucleotides |
| JP2006500066A (ja) | 2002-09-25 | 2006-01-05 | ファルマシア・コーポレーション | ミクロソームのプロスタグランジンｅ２シンターゼ発現のアンチセンス調節 |
| AU2003291753B2 (en) | 2002-11-05 | 2010-07-08 | Isis Pharmaceuticals, Inc. | Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation |
| EP2957568B1 (en) | 2002-11-05 | 2016-12-21 | Ionis Pharmaceuticals, Inc. | Compositions comprising alternating 2'-modified nucleosides for use in gene modulation |
| EP2213738B1 (en) | 2002-11-14 | 2012-10-10 | Dharmacon, Inc. | siRNA molecules targeting Bcl-2 |
| WO2006006948A2 (en) | 2002-11-14 | 2006-01-19 | Dharmacon, Inc. | METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY |
| US20050019746A1 (en) | 2003-01-23 | 2005-01-27 | Eirx Therapeutics Limited | Apoptosis-related kinase/GPCRs |
| US7598227B2 (en) | 2003-04-16 | 2009-10-06 | Isis Pharmaceuticals Inc. | Modulation of apolipoprotein C-III expression |
| US8158354B2 (en) | 2003-05-13 | 2012-04-17 | Ibis Biosciences, Inc. | Methods for rapid purification of nucleic acids for subsequent analysis by mass spectrometry by solution capture |
| US7589189B2 (en) | 2003-05-14 | 2009-09-15 | Japan Science And Technology Agency | Inhibition of the expression of huntingtin gene |
| WO2004106356A1 (en) | 2003-05-27 | 2004-12-09 | Syddansk Universitet | Functionalized nucleotide derivatives |
| CN100577680C (zh) * | 2003-07-03 | 2010-01-06 | 宾夕法尼亚大学理事会 | 对Syk激酶表达的抑制 |
| ATE555118T1 (de) | 2003-08-28 | 2012-05-15 | Takeshi Imanishi | Neue synthetische nukleidsäuren vom typ mit quervernetzter n-o-bindung |
| JP5379347B2 (ja) | 2003-09-18 | 2013-12-25 | アイシス ファーマシューティカルズ， インコーポレーテッド | ４’−チオヌクレオシドおよびオリゴマー化合物 |
| US7208174B2 (en) | 2003-12-18 | 2007-04-24 | Hoffmann-La Roche Inc. | Liposome compositions |
| AU2003300239A1 (en) * | 2003-12-29 | 2005-07-21 | Galapagos Genomics N.V. | Modulators of bone homeostasis identified in a high-throughput screen |
| US7374927B2 (en) | 2004-05-03 | 2008-05-20 | Affymetrix, Inc. | Methods of analysis of degraded nucleic acid samples |
| CA2566286A1 (en) | 2004-05-11 | 2005-12-08 | Rnai Co., Ltd. | Polynucleotide causing rna interfere and method of regulating gene expression with the use of the same |
| EP1766052A4 (en) | 2004-06-03 | 2009-12-16 | Isis Pharmaceuticals Inc | OLIGOMERIC CHIMERIC COMPOSITIONS WITH BRECH |
| CA2569419A1 (en) | 2004-06-03 | 2005-12-22 | Isis Pharmaceuticals, Inc. | Double strand compositions comprising differentially modified strands for use in gene modulation |
| EP1799859B1 (en) | 2004-09-17 | 2014-07-02 | Isis Pharmaceuticals, Inc. | Enhanced antisense oligonucleotides |
| WO2006047842A2 (en) | 2004-11-08 | 2006-05-11 | K.U. Leuven Research And Development | Modified nucleosides for rna interference |
| WO2007089611A2 (en) | 2006-01-26 | 2007-08-09 | Isis Pharmaceuticals Inc. | Compositions and their uses directed to huntingtin |
| EP1984381B1 (en) | 2006-01-27 | 2010-09-29 | Isis Pharmaceuticals, Inc. | 6-modified bicyclic nucleic acid analogs |
| US8158364B2 (en) | 2006-04-11 | 2012-04-17 | The Board Of Regents Of The University Of Texas System | Methods and compositions involving nucleotide repeat disorders |
| ES2389737T3 (es) | 2006-05-11 | 2012-10-31 | Isis Pharmaceuticals, Inc. | Análogos de ácidos nucleicos bicíclicos modificados en 5' |
| DK2049664T3 (da) | 2006-08-11 | 2012-01-02 | Prosensa Technologies Bv | Enkeltstrengede oligonukleotider, som er komplementære til repetitive elementer, til behandling af med DNA-repetitiv-ustabilitet associerede lidelser |
| WO2008036406A2 (en) | 2006-09-21 | 2008-03-27 | University Of Rochester | Compositions and methods related to protein displacement therapy for myotonic distrophy |
| EP2410053B2 (en) | 2006-10-18 | 2020-07-15 | Ionis Pharmaceuticals, Inc. | Antisense compounds |
| BRPI0720689A2 (pt) | 2006-12-20 | 2014-02-25 | Novarx | Vacina de célula tumoral universal para utilização profilática e terapêutica anticâncer |
| EP2125852B1 (en) | 2007-02-15 | 2016-04-06 | Ionis Pharmaceuticals, Inc. | 5'-substituted-2'-f modified nucleosides and oligomeric compounds prepared therefrom |
| US8278425B2 (en) | 2007-05-30 | 2012-10-02 | Isis Pharmaceuticals, Inc. | N-substituted-aminomethylene bridged bicyclic nucleic acid analogs |
| DK2173760T4 (en) | 2007-06-08 | 2016-02-08 | Isis Pharmaceuticals Inc | Carbocyclic bicyclic nukleinsyreanaloge |
| US20100016215A1 (en) * | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| US8278283B2 (en) | 2007-07-05 | 2012-10-02 | Isis Pharmaceuticals, Inc. | 6-disubstituted or unsaturated bicyclic nucleic acid analogs |
| US7973019B1 (en) | 2007-10-03 | 2011-07-05 | Alcon Research, Ltd. | Transferrin/transferrin receptor-mediated siRNA delivery |
| US8546556B2 (en) | 2007-11-21 | 2013-10-01 | Isis Pharmaceuticals, Inc | Carbocyclic alpha-L-bicyclic nucleic acid analogs |
| EP2265627A2 (en) | 2008-02-07 | 2010-12-29 | Isis Pharmaceuticals, Inc. | Bicyclic cyclohexitol nucleic acid analogs |
| JP2011510678A (ja) | 2008-02-08 | 2011-04-07 | プロセンサ ホールディング ビーブイ | Ｄｎａ反復不安定性関連遺伝性障害を治療するための方法及び手段 |
| EP2317847B1 (en) | 2008-07-29 | 2019-04-17 | The Board of Regents of The University of Texas System | Selective inhibition of polyglutamine protein expression |
| GB0816778D0 (en) | 2008-09-12 | 2008-10-22 | Isis Innovation | Gene silencing |
| WO2010036698A1 (en) | 2008-09-24 | 2010-04-01 | Isis Pharmaceuticals, Inc. | Substituted alpha-l-bicyclic nucleosides |
| EP2361256B1 (en) | 2008-09-24 | 2013-04-10 | Isis Pharmaceuticals, Inc. | Cyclohexenyl nucleic acid analogs |
| CN102307997B (zh) * | 2008-12-04 | 2018-03-30 | 库尔纳公司 | 通过抑制针对沉默调节蛋白1的天然反义转录物来治疗沉默调节蛋白1(sirt1)相关的疾病 |
| PL2417257T3 (pl) | 2009-04-10 | 2016-11-30 | Antysensowne oligonukleotydy tricyklo-dna, kompozycje i sposoby leczenia choroby | |
| EP2462153B1 (en) | 2009-08-06 | 2015-07-29 | Isis Pharmaceuticals, Inc. | Bicyclic cyclohexose nucleic acid analogs |
| US9574191B2 (en) | 2010-02-03 | 2017-02-21 | The Board Of Regents Of The University Of Texas System | Selective inhibition of polyglutamine protein expression |
| JP2013518603A (ja) * | 2010-02-08 | 2013-05-23 | アイシス ファーマシューティカルズ， インコーポレーテッド | 反復伸張に関連する疾患または病態の治療に有用な方法および組成物 |
| EP2547768B1 (en) * | 2010-03-17 | 2015-12-30 | Association Institut de Myologie | Modified u7 snrnas for treatment of neuromuscular diseases |
| AU2011282217B2 (en) | 2010-07-19 | 2015-12-03 | Ionis Pharmaceuticals, Inc. | Modulation of dystrophia myotonica-protein kinase (DMPK) expression |
| EP2623600A4 (en) * | 2010-09-30 | 2014-11-26 | Lsip Llc | EXPRESSION INHIBITOR OF A DOMINANTLY MUTANT GENE |
| KR20160074368A (ko) * | 2012-05-16 | 2016-06-28 | 라나 테라퓨틱스, 인크. | Utrn 발현을 조절하기 위한 조성물 및 방법 |
| JP2016522674A (ja) | 2012-05-16 | 2016-08-04 | ラナ セラピューティクス インコーポレイテッド | 遺伝子発現を調節するための組成物及び方法 |
| DK2951191T3 (en) | 2013-01-31 | 2019-01-14 | Ionis Pharmaceuticals Inc | PROCEDURE FOR MANUFACTURING OLIGOMERIC COMPOUNDS USING MODIFIED CLUTCH PROTOCOLS |
| TW201536329A (zh) | 2013-08-09 | 2015-10-01 | Isis Pharmaceuticals Inc | 用於調節失養性肌強直蛋白質激酶(dmpk)表現之化合物及方法 |
| US20210052631A1 (en) | 2015-09-25 | 2021-02-25 | Ionis Pharmaceuticals, Inc. | Conjugated antisense compounds and their use |
| US11833221B2 (en) | 2021-09-01 | 2023-12-05 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds for reducing DMPK expression |
| EP4396351A4 (en) | 2021-09-01 | 2025-12-24 | Ionis Pharmaceuticals Inc | COMPOUNDS AND METHODS FOR REDUCING DMPK EXPRESSION |
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