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 (cg-RX-API-DMAC7.html) | 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 (cg-RX-API-DMAC7.html) |
| EP (2) | EP2785729B1 (cg-RX-API-DMAC7.html) |
| JP (4) | JP6317675B2 (cg-RX-API-DMAC7.html) |
| ES (1) | ES2832531T3 (cg-RX-API-DMAC7.html) |
| WO (1) | WO2013082548A1 (cg-RX-API-DMAC7.html) |
Families Citing this family (50)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| ES2564563T3 (es) | 2007-10-26 | 2016-03-23 | Academisch Ziekenhuis Leiden | Medios y métodos para contrarrestar trastornos del músculo |
| CN102282155B (zh) | 2008-12-02 | 2017-06-09 | 日本波涛生命科学公司 | 磷原子修饰的核酸的合成方法 |
| BR112012000828A8 (pt) | 2009-07-06 | 2017-10-10 | Ontorii Inc | Novas pró-drogas de ácido nucleico e métodos de uso das mesmas |
| EP2620428B1 (en) | 2010-09-24 | 2019-05-22 | Wave Life Sciences Ltd. | Asymmetric auxiliary group |
| WO2012138289A1 (en) * | 2011-04-08 | 2012-10-11 | Zain-Luqman Rula | Diagnosis and treatment of friedreich's ataxia |
| ES2626488T3 (es) | 2011-07-19 | 2017-07-25 | Wave Life Sciences Pte. Ltd. | Procedimientos para la síntesis de ácidos nucleicos funcionalizados |
| US9944926B2 (en) | 2011-11-30 | 2018-04-17 | 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 |
| EP4400169A3 (en) | 2012-04-23 | 2024-12-25 | Vico Therapeutics B.V. | Rna modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders |
| ES2862073T3 (es) | 2012-07-13 | 2021-10-06 | Wave Life Sciences Ltd | Grupo auxiliar asimétrico |
| MX369519B (es) | 2012-09-25 | 2019-11-11 | Genzyme Corp | Oligonucleotidos antisentido morfolino unidos a peptido para el tratamiento de distrofia miotonica. |
| ES2762326T5 (es) * | 2012-10-15 | 2023-04-27 | Ionis Pharmaceuticals Inc | Métodos para modular la expresión de C9ORF72 |
| AU2013331434B2 (en) | 2012-10-15 | 2019-08-08 | Ionis Pharmaceuticals, Inc. | Compositions for modulating C90RF72 expression |
| EP2906697A4 (en) * | 2012-10-15 | 2016-06-22 | Ionis Pharmaceuticals Inc | METHOD FOR MONITORING THE C9ORF72 EXPRESSION |
| MX2016004651A (es) * | 2013-10-11 | 2016-08-05 | Ionis Pharmaceuticals Inc | Composiciones para modular la expresion de c9orf72. |
| US11162096B2 (en) | 2013-10-14 | 2021-11-02 | Ionis Pharmaceuticals, Inc | Methods for modulating expression of C9ORF72 antisense transcript |
| CN113278617A (zh) | 2014-01-16 | 2021-08-20 | 波涛生命科学有限公司 | 手性设计 |
| RU2702838C2 (ru) | 2014-03-19 | 2019-10-11 | Ионис Фармасьютикалз, Инк. | Композиции для модуляции экспрессии атаксина 2 |
| US10006027B2 (en) | 2014-03-19 | 2018-06-26 | Ionis 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 |
| US10793855B2 (en) | 2015-01-06 | 2020-10-06 | Ionis Pharmaceuticals, Inc. | Compositions for modulating expression of C9ORF72 antisense transcript |
| SG11201708468YA (en) * | 2015-04-16 | 2017-11-29 | 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 |
| CN107921092B (zh) | 2015-05-19 | 2022-04-08 | 萨勒普塔医疗公司 | 肽寡核苷酸缀合物 |
| MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| EP4039690B1 (en) | 2015-08-05 | 2024-07-17 | Eisai R&D Management Co., Ltd. | A substantially diastereomerically pure phosphoramidochloridate, a method and a pharmaceutical composition |
| 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 |
| WO2017079291A1 (en) | 2015-11-02 | 2017-05-11 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating c90rf72 |
| JP2019500899A (ja) * | 2015-11-23 | 2019-01-17 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | ＣＲＩＳＰＲ／Ｃａｓ９の核送達を通じた細胞ＲＮＡの追跡と操作 |
| AU2016381174A1 (en) | 2015-12-31 | 2018-05-31 | 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 |
| US11453891B2 (en) | 2017-05-10 | 2022-09-27 | The Regents Of The University Of California | Directed editing of cellular RNA via nuclear delivery of CRISPR/CAS9 |
| JP7246729B2 (ja) * | 2017-05-18 | 2023-03-28 | 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 |
| CA3082568A1 (en) * | 2017-11-15 | 2019-05-23 | Alcyone Lifesciences, Inc. | Therapy specific, pre-programmed auto injection device |
| WO2019216433A1 (ja) | 2018-05-10 | 2019-11-14 | 日本新薬株式会社 | オリゴ核酸化合物の製造方法 |
| MX2021000922A (es) | 2018-07-25 | 2021-03-31 | Ionis Pharmaceuticals Inc | Compuestos y metodos para reducir la expresion de la atxn2. |
| EP3843845A4 (en) * | 2018-08-29 | 2022-05-11 | University Of Massachusetts | INHIBITION OF PROTEIN KINASE FOR THE TREATMENT OF FRIEDREICH'S ATAXIA |
| WO2020227395A2 (en) * | 2019-05-06 | 2020-11-12 | University Of Massachusetts | Anti-c9orf72 oligonucleotides and related methods |
| CN115175991A (zh) | 2019-10-16 | 2022-10-11 | 博德研究所 | 工程化肌肉靶向组合物 |
| EP4059944A4 (en) | 2019-11-13 | 2024-04-10 | Nippon Shinyaku Co., Ltd. | METHOD FOR PREPARING AN OLIGONUCLICE ACID COMPOUND |
| US20230014840A1 (en) | 2019-11-13 | 2023-01-19 | Nippon Shinyaku Co., Ltd. | Method for producing oligonucleic 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 |
| US5034506A (en) | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
| US5506337A (en) | 1985-03-15 | 1996-04-09 | Antivirals Inc. | Morpholino-subunit combinatorial library and method |
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| JPH07501204A (ja) | 1991-06-28 | 1995-02-09 | マサチューセッツ インスティテュート オブ テクノロジー | 局所的オリゴヌクレオチド療法 |
| 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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| KR102182663B1 (ko) | 2010-05-28 | 2020-11-25 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체 |
| CA2834128A1 (en) | 2011-05-05 | 2012-11-08 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| US9161948B2 (en) * | 2011-05-05 | 2015-10-20 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| US10066228B2 (en) * | 2011-11-30 | 2018-09-04 | Sarepta Therapeutics, Inc. | Oligonucleotides for treating expanded repeat diseases |
| US9944926B2 (en) | 2011-11-30 | 2018-04-17 | Sarepta Therapeutics, Inc. | Induced exon inclusion in spinal muscle atrophy |
-
2012
- 2012-11-30 US US14/360,890 patent/US10066228B2/en active Active
- 2012-11-30 ES ES12808975T patent/ES2832531T3/es active Active
- 2012-11-30 JP JP2014544959A patent/JP6317675B2/ja active Active
- 2012-11-30 EP EP12808975.2A patent/EP2785729B1/en active Active
- 2012-11-30 WO PCT/US2012/067470 patent/WO2013082548A1/en not_active Ceased
- 2012-11-30 EP EP20196300.6A patent/EP3858847A1/en active Pending
-
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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