JP2015506669A5 - - Google Patents
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- Publication number
- JP2015506669A5 JP2015506669A5 JP2014544959A JP2014544959A JP2015506669A5 JP 2015506669 A5 JP2015506669 A5 JP 2015506669A5 JP 2014544959 A JP2014544959 A JP 2014544959A JP 2014544959 A JP2014544959 A JP 2014544959A JP 2015506669 A5 JP2015506669 A5 JP 2015506669A5
- Authority
- JP
- Japan
- Prior art keywords
- antisense oligonucleotide
- group
- pharmaceutically acceptable
- acceptable salt
- alkyl
- 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.)
- Granted
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- 108091034117 Oligonucleotide Proteins 0.000 claims description 36
- 239000000074 antisense oligonucleotide Substances 0.000 claims description 32
- 238000012230 antisense oligonucleotides Methods 0.000 claims description 32
- 239000002773 nucleotide Substances 0.000 claims description 22
- 125000003729 nucleotide group Chemical group 0.000 claims description 21
- 229910052739 hydrogen Inorganic materials 0.000 claims description 16
- 239000001257 hydrogen Substances 0.000 claims description 16
- 108020004999 messenger RNA Proteins 0.000 claims description 16
- 125000000217 alkyl group Chemical group 0.000 claims description 13
- 150000002431 hydrogen Chemical class 0.000 claims description 12
- 150000003839 salts Chemical class 0.000 claims description 12
- RWQNBRDOKXIBIV-UHFFFAOYSA-N thymine Chemical compound CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 claims description 12
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 10
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical compound O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 claims description 8
- 201000010099 disease Diseases 0.000 claims description 8
- 208000023105 Huntington disease Diseases 0.000 claims description 6
- 125000002252 acyl group Chemical group 0.000 claims description 6
- 125000003710 aryl alkyl group Chemical group 0.000 claims description 6
- OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical compound NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 claims description 6
- UYTPUPDQBNUYGX-UHFFFAOYSA-N guanine Chemical compound O=C1NC(N)=NC2=C1N=CN2 UYTPUPDQBNUYGX-UHFFFAOYSA-N 0.000 claims description 6
- FDGQSTZJBFJUBT-UHFFFAOYSA-N hypoxanthine Chemical compound O=C1NC=NC2=C1NC=N2 FDGQSTZJBFJUBT-UHFFFAOYSA-N 0.000 claims description 6
- 238000000034 method Methods 0.000 claims description 6
- 239000008194 pharmaceutical composition Substances 0.000 claims description 6
- 229940113082 thymine Drugs 0.000 claims description 6
- UFHFLCQGNIYNRP-UHFFFAOYSA-N Hydrogen Chemical compound [H][H] UFHFLCQGNIYNRP-UHFFFAOYSA-N 0.000 claims description 4
- 206010002026 amyotrophic lateral sclerosis Diseases 0.000 claims description 4
- 239000003937 drug carrier Substances 0.000 claims description 4
- 239000012528 membrane Substances 0.000 claims description 4
- 239000000203 mixture Substances 0.000 claims description 4
- 239000000523 sample Substances 0.000 claims description 4
- 229940035893 uracil Drugs 0.000 claims description 4
- GFFGJBXGBJISGV-UHFFFAOYSA-N Adenine Chemical compound NC1=NC=NC2=C1N=CN2 GFFGJBXGBJISGV-UHFFFAOYSA-N 0.000 claims description 3
- 229930024421 Adenine Natural products 0.000 claims description 3
- 108010051109 Cell-Penetrating Peptides Proteins 0.000 claims description 3
- 102000020313 Cell-Penetrating Peptides Human genes 0.000 claims description 3
- UGQMRVRMYYASKQ-UHFFFAOYSA-N Hypoxanthine nucleoside Natural products OC1C(O)C(CO)OC1N1C(NC=NC2=O)=C2N=C1 UGQMRVRMYYASKQ-UHFFFAOYSA-N 0.000 claims description 3
- 229960000643 adenine Drugs 0.000 claims description 3
- 150000001413 amino acids Chemical class 0.000 claims description 3
- 229940104302 cytosine Drugs 0.000 claims description 3
- 229910052736 halogen Inorganic materials 0.000 claims description 3
- 150000002367 halogens Chemical class 0.000 claims description 3
- 125000001997 phenyl group Chemical group [H]C1=C([H])C([H])=C(*)C([H])=C1[H] 0.000 claims description 3
- 125000004193 piperazinyl group Chemical group 0.000 claims description 3
- 108090000765 processed proteins & peptides Chemical group 0.000 claims description 3
- 125000001424 substituent group Chemical group 0.000 claims description 3
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 2
- 108020000948 Antisense Oligonucleotides Proteins 0.000 claims description 2
- 108020004414 DNA Proteins 0.000 claims description 2
- 206010068871 Myotonic dystrophy Diseases 0.000 claims description 2
- 238000000636 Northern blotting Methods 0.000 claims description 2
- 108091081062 Repeated sequence (DNA) Proteins 0.000 claims description 2
- 238000001502 gel electrophoresis Methods 0.000 claims description 2
- 230000003100 immobilizing effect Effects 0.000 claims description 2
- 230000002035 prolonged effect Effects 0.000 claims description 2
- OAICVXFJPJFONN-UHFFFAOYSA-N Phosphorus Chemical compound [P] OAICVXFJPJFONN-UHFFFAOYSA-N 0.000 claims 2
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 claims 2
- 229910052760 oxygen Inorganic materials 0.000 claims 2
- 229910052698 phosphorus Inorganic materials 0.000 claims 2
- 239000011574 phosphorus Substances 0.000 claims 2
- 125000003341 7 membered heterocyclic group Chemical group 0.000 claims 1
- -1 C 1 -C 6 alkyl Substances 0.000 claims 1
- 239000001301 oxygen Substances 0.000 claims 1
- 229910052717 sulfur Inorganic materials 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 description 9
- 102000004169 proteins and genes Human genes 0.000 description 7
- 108091033319 polynucleotide Proteins 0.000 description 6
- 239000002157 polynucleotide Substances 0.000 description 6
- 102000040430 polynucleotide Human genes 0.000 description 6
- 230000001717 pathogenic effect Effects 0.000 description 4
- 230000000692 anti-sense effect Effects 0.000 description 3
- QVGXLLKOCUKJST-UHFFFAOYSA-N atomic oxygen Chemical group [O] QVGXLLKOCUKJST-UHFFFAOYSA-N 0.000 description 2
- 125000000623 heterocyclic group Chemical group 0.000 description 2
- 238000002560 therapeutic procedure Methods 0.000 description 2
- 208000026350 Inborn Genetic disease Diseases 0.000 description 1
- 230000003321 amplification Effects 0.000 description 1
- 238000012258 culturing Methods 0.000 description 1
- 238000000605 extraction Methods 0.000 description 1
- 208000016361 genetic disease Diseases 0.000 description 1
- 238000003199 nucleic acid amplification method Methods 0.000 description 1
- 230000004043 responsiveness Effects 0.000 description 1
- 238000010839 reverse transcription Methods 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201161565475P | 2011-11-30 | 2011-11-30 | |
| US61/565,475 | 2011-11-30 | ||
| PCT/US2012/067470 WO2013082548A1 (en) | 2011-11-30 | 2012-11-30 | Oligonucleotides for treating expanded repeat diseases |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018008762A Division JP6718474B2 (ja) | 2011-11-30 | 2018-01-23 | 延長リピート病を処置するためのオリゴヌクレオチド |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2015506669A JP2015506669A (ja) | 2015-03-05 |
| JP2015506669A5 true JP2015506669A5 (enExample) | 2016-01-28 |
| JP6317675B2 JP6317675B2 (ja) | 2018-04-25 |
Family
ID=47470150
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2014544959A Active JP6317675B2 (ja) | 2011-11-30 | 2012-11-30 | 延長リピート病を処置するためのオリゴヌクレオチド |
| JP2018008762A Active JP6718474B2 (ja) | 2011-11-30 | 2018-01-23 | 延長リピート病を処置するためのオリゴヌクレオチド |
| JP2020033113A Active JP7057385B2 (ja) | 2011-11-30 | 2020-02-28 | 延長リピート病を処置するためのオリゴヌクレオチド |
| JP2022010846A Pending JP2022048248A (ja) | 2011-11-30 | 2022-01-27 | 延長リピート病を処置するためのオリゴヌクレオチド |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018008762A Active JP6718474B2 (ja) | 2011-11-30 | 2018-01-23 | 延長リピート病を処置するためのオリゴヌクレオチド |
| JP2020033113A Active JP7057385B2 (ja) | 2011-11-30 | 2020-02-28 | 延長リピート病を処置するためのオリゴヌクレオチド |
| JP2022010846A Pending JP2022048248A (ja) | 2011-11-30 | 2022-01-27 | 延長リピート病を処置するためのオリゴヌクレオチド |
Country Status (5)
| Country | Link |
|---|---|
| US (3) | US10066228B2 (enExample) |
| EP (2) | EP3858847A1 (enExample) |
| JP (4) | JP6317675B2 (enExample) |
| ES (1) | ES2832531T3 (enExample) |
| WO (1) | WO2013082548A1 (enExample) |
Families Citing this family (50)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PT2049664E (pt) | 2006-08-11 | 2012-01-03 | Prosensa Technologies Bv | Oligonucleotídeos de cadeia simples complementares dos elementos repetitivos para tratar perturbações genéticas associadas à instabilidade das repetições do adn |
| PL2203173T3 (pl) | 2007-10-26 | 2016-06-30 | Academisch Ziekenhuis Leiden | Środki i sposoby przeciwdziałania zaburzeniom mięśni |
| CA2744987C (en) | 2008-12-02 | 2018-01-16 | Chiralgen, Ltd. | Method for the synthesis of phosphorus atom modified nucleic acids |
| CA2767253A1 (en) | 2009-07-06 | 2011-01-13 | Ontorii, Inc. | Novel nucleic acid prodrugs and methods of use thereof |
| WO2012039448A1 (ja) | 2010-09-24 | 2012-03-29 | 株式会社キラルジェン | 不斉補助基 |
| WO2012138289A1 (en) * | 2011-04-08 | 2012-10-11 | Zain-Luqman Rula | Diagnosis and treatment of friedreich's ataxia |
| WO2013012758A1 (en) | 2011-07-19 | 2013-01-24 | Ontorii, Inc. | Methods for the synthesis of functionalized nucleic acids |
| US10066228B2 (en) * | 2011-11-30 | 2018-09-04 | Sarepta Therapeutics, Inc. | Oligonucleotides for treating expanded repeat diseases |
| AU2012345638C1 (en) | 2011-11-30 | 2018-10-18 | Sarepta Therapeutics, Inc. | Induced exon inclusion in spinal muscle atrophy |
| EP3228712A1 (en) | 2012-04-23 | 2017-10-11 | BioMarin Technologies B.V. | Rna modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders |
| JP6268157B2 (ja) | 2012-07-13 | 2018-01-24 | 株式会社Ｗａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｊａｐａｎ | 不斉補助基 |
| US10111962B2 (en) | 2012-09-25 | 2018-10-30 | Genzyme Corporation | Peptide-linked morpholino antisense oligonucleotides for treatment of myotonic dystrophy |
| DK2906696T4 (da) * | 2012-10-15 | 2023-02-27 | Ionis Pharmaceuticals Inc | Fremgangsmåder til modulering af c9orf72-ekspression |
| EP2906697A4 (en) * | 2012-10-15 | 2016-06-22 | Ionis Pharmaceuticals Inc | METHOD FOR MONITORING THE C9ORF72 EXPRESSION |
| WO2014062691A2 (en) | 2012-10-15 | 2014-04-24 | Isis Pharmaceuticals, Inc. | Compositions for modulating c9orf72 expression |
| EP3055414A4 (en) * | 2013-10-11 | 2017-07-19 | Ionis Pharmaceuticals, Inc. | Compositions for modulating c9orf72 expression |
| US11162096B2 (en) | 2013-10-14 | 2021-11-02 | Ionis Pharmaceuticals, Inc | Methods for modulating expression of C9ORF72 antisense transcript |
| EP4137572A1 (en) | 2014-01-16 | 2023-02-22 | Wave Life Sciences Ltd. | Chiral design |
| WO2015143246A1 (en) | 2014-03-19 | 2015-09-24 | Isis Pharmaceuticals, Inc. | Compositions for modulating ataxin 2 expression |
| WO2015143245A1 (en) | 2014-03-19 | 2015-09-24 | Isis Pharmaceuticals, Inc. | Methods for modulating ataxin 2 expression |
| US20160017327A1 (en) * | 2014-07-11 | 2016-01-21 | The Johns Hopkins University | Phosphorodiamidate morpholino oligomers (pmos) and their use in suppression of mutant huntingtin expression and attenuation of neurotoxicity |
| US20190002876A1 (en) * | 2014-12-09 | 2019-01-03 | The Board Of Regents Of The University Of Texas System | Compositions and methods for treatment of friedreich's ataxia |
| WO2016112132A1 (en) | 2015-01-06 | 2016-07-14 | Ionis Pharmaceuticals, Inc. | Compositions for modulating expression of c9orf72 antisense transcript |
| MY181815A (en) * | 2015-04-16 | 2021-01-08 | Ionis Pharmaceuticals Inc | Compositions for modulating c9orf72 expression |
| WO2016167780A1 (en) | 2015-04-16 | 2016-10-20 | Ionis Pharmaceuticals, Inc. | Compositions for modulating expression of c9orf72 antisense transcript |
| EP3297649B1 (en) * | 2015-05-19 | 2023-10-11 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| CN113461733A (zh) | 2015-08-05 | 2021-10-01 | 卫材R&D管理有限公司 | 用于制备均一低聚物的手性试剂 |
| JP6987041B2 (ja) | 2015-08-28 | 2021-12-22 | サレプタ セラピューティクス，インコーポレイテッド | 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 |
| US11260073B2 (en) | 2015-11-02 | 2022-03-01 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating C90RF72 |
| ES2930643T3 (es) * | 2015-11-23 | 2022-12-20 | Univ California | Seguimiento y manipulación de ARN celular a través de la administración nuclear de CRISPR/CAS9 |
| CA3006015A1 (en) | 2015-12-31 | 2017-07-06 | Ionis Pharmaceuticals, Inc. | Methods for reducing ataxin-2 expression |
| MA45270A (fr) | 2016-05-04 | 2017-11-09 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| EA039716B1 (ru) * | 2016-05-17 | 2022-03-03 | Сарепта Терапьютикс, Инк. | Пептид-олигонуклеотидные конъюгаты |
| SG10202101836TA (en) | 2016-05-24 | 2021-03-30 | Sarepta Therapeutics Inc | Processes for preparing phosphorodiamidate morpholino oligomers |
| KR102523527B1 (ko) * | 2016-06-30 | 2023-04-20 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 포스포로디아미데이트 모르폴리노 올리고머의 제조 방법 |
| EP3622062A4 (en) | 2017-05-10 | 2020-10-14 | The Regents of the University of California | DIRECTED EDITING OF CELLULAR RNA BY NUCLEAR ADMINISTRATION OF CRISPR / CAS9 |
| WO2018212271A1 (ja) * | 2017-05-18 | 2018-11-22 | 国立大学法人京都大学 | 脊髄小脳変性症３６型の予防又は治療用組成物 |
| US10668247B2 (en) * | 2017-11-15 | 2020-06-02 | Alcyone Lifesciences, Inc. | Therapy specific, pre-programmed auto injection device |
| CA3099778A1 (en) | 2018-05-10 | 2019-11-14 | Nippon Shinyaku Co., Ltd. | Method for preparing oligonucleic acid compound |
| KR20210038589A (ko) | 2018-07-25 | 2021-04-07 | 아이오니스 파마수티컬즈, 인코포레이티드 | Atxn2 발현 감소용 화합물 및 방법 |
| CA3110661A1 (en) * | 2018-08-29 | 2020-03-05 | University Of Massachusetts | Inhibition of protein kinases to treat friedreich ataxia |
| US11629347B2 (en) * | 2019-05-06 | 2023-04-18 | University Of Massachusetts | Anti-C9ORF72 oligonucleotides and related methods |
| EP4045637A4 (en) | 2019-10-16 | 2023-11-22 | The Broad Institute, Inc. | TECHNICAL COMPOSITIONS FOR MUSCLE BUILDING |
| KR20220097986A (ko) | 2019-11-13 | 2022-07-08 | 니뽄 신야쿠 가부시키가이샤 | 올리고 핵산 화합물의 제조 방법 |
| EP4059944A4 (en) | 2019-11-13 | 2024-04-10 | Nippon Shinyaku Co., Ltd. | METHOD FOR PREPARING AN OLIGONUCLICE ACID COMPOUND |
| US12378267B2 (en) | 2021-06-17 | 2025-08-05 | Entrada Therapeutics, Inc. | Synthesis of FMOC-protected morpholino monomers and oligomers |
| WO2023225506A1 (en) * | 2022-05-16 | 2023-11-23 | The University Of North Carolina At Chapel Hill | Compositions and methods comprising synthetic rna molecules for treatment of intragenic nucleotide repeat disorders |
| WO2025137623A1 (en) * | 2023-12-22 | 2025-06-26 | Iris Medicine, Inc. | Methods and compositions for treating ctg repeat expansion diseases |
| WO2025151707A1 (en) * | 2024-01-11 | 2025-07-17 | Entrada Therapeutics, Inc. | Method for preparing multimers of phosphorodiamidate morpholino oligomers |
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| US5166315A (en) | 1989-12-20 | 1992-11-24 | Anti-Gene Development Group | Sequence-specific binding polymers for duplex nucleic acids |
| 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 |
| 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 |
| US5506337A (en) | 1985-03-15 | 1996-04-09 | Antivirals Inc. | Morpholino-subunit combinatorial library and method |
| 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 |
| WO1993001286A2 (en) | 1991-06-28 | 1993-01-21 | Massachusetts Institute Of Technology | Localized oligonucleotide therapy |
| PT876165E (pt) | 1995-12-18 | 2006-10-31 | Angiotech Biomaterials Corp | Composicoes de polimeros reticulados e processos para a sua utilizacao |
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| FR2802206B1 (fr) | 1999-12-14 | 2005-04-22 | Sod Conseils Rech Applic | Derives de 4-aminopiperidine et leur utilisation en tant que medicament |
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| US20100016215A1 (en) * | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| MX2010004955A (es) | 2007-11-15 | 2010-06-30 | Avi Biopharma Inc | Metodo de sintesis de oligomeros de morfolina. |
| DE102008000410A1 (de) | 2008-02-26 | 2009-08-27 | Wacker Chemie Ag | Verfahren zur Herstellung von Alkylchlorsilanen aus den Rückständen der Direktsynthese von Alkylchlorsilanen |
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| 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 |
| WO2012150960A1 (en) | 2011-05-05 | 2012-11-08 | Avi Biopharma, Inc. | Peptide oligonucleotide conjugates |
| US9161948B2 (en) * | 2011-05-05 | 2015-10-20 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| AU2012345638C1 (en) | 2011-11-30 | 2018-10-18 | Sarepta Therapeutics, Inc. | Induced exon inclusion in spinal muscle atrophy |
| US10066228B2 (en) * | 2011-11-30 | 2018-09-04 | Sarepta Therapeutics, Inc. | Oligonucleotides for treating expanded repeat diseases |
-
2012
- 2012-11-30 US US14/360,890 patent/US10066228B2/en active Active
- 2012-11-30 EP EP20196300.6A patent/EP3858847A1/en active Pending
- 2012-11-30 JP JP2014544959A patent/JP6317675B2/ja active Active
- 2012-11-30 WO PCT/US2012/067470 patent/WO2013082548A1/en not_active Ceased
- 2012-11-30 ES ES12808975T patent/ES2832531T3/es active Active
- 2012-11-30 EP EP12808975.2A patent/EP2785729B1/en active Active
-
2018
- 2018-01-23 JP JP2018008762A patent/JP6718474B2/ja active Active
- 2018-07-18 US US16/038,580 patent/US11674139B2/en active Active
-
2020
- 2020-02-28 JP JP2020033113A patent/JP7057385B2/ja active Active
-
2022
- 2022-01-27 JP JP2022010846A patent/JP2022048248A/ja active Pending
-
2023
- 2023-05-02 US US18/310,922 patent/US20240209363A1/en active Pending
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