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) | EP2785729B1 (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 |
|---|---|---|---|---|
| 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 |
| EP2614827B1 (en) | 2007-10-26 | 2017-06-28 | Academisch Ziekenhuis Leiden | Means and methods for counteracting muscle disorders |
| 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 |
| WO2011005761A1 (en) | 2009-07-06 | 2011-01-13 | Ontorii, Inc | Novel nucleic acid prodrugs and methods 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 |
| EP2734208B1 (en) | 2011-07-19 | 2017-03-01 | Wave Life Sciences Ltd. | Methods for the synthesis of functionalized nucleic acids |
| ES2727481T3 (es) | 2011-11-30 | 2019-10-16 | Sarepta Therapeutics Inc | Inclusión inducida de exón en atrofia muscular espinal |
| JP6317675B2 (ja) * | 2011-11-30 | 2018-04-25 | サレプタ セラピューティクス， インコーポレイテッド | 延長リピート病を処置するためのオリゴヌクレオチド |
| EP3228712A1 (en) | 2012-04-23 | 2017-10-11 | BioMarin Technologies B.V. | Rna modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders |
| AU2013288048A1 (en) | 2012-07-13 | 2015-01-22 | Wave Life Sciences Ltd. | Asymmetric auxiliary group |
| SG10201700751XA (en) | 2012-09-25 | 2017-03-30 | Genzyme Corp | Peptide-linked morpholino antisense oligonucleotides for treatment of myotonic dystrophy |
| WO2014062736A1 (en) | 2012-10-15 | 2014-04-24 | Isis Pharmaceuticals, Inc. | Methods for monitoring c9orf72 expression |
| BR112015008399A8 (pt) * | 2012-10-15 | 2017-10-03 | Ionis Pharmaceuticals Inc | Composto para modular a expressão de c90rf72, seu uso, oligonucleotídeo modificado, composto de fita dupla e composição |
| WO2014062686A1 (en) | 2012-10-15 | 2014-04-24 | Isis Pharmaceuticals, Inc. | Methods 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 |
| CN113278617A (zh) | 2014-01-16 | 2021-08-20 | 波涛生命科学有限公司 | 手性设计 |
| US10006027B2 (en) | 2014-03-19 | 2018-06-26 | Ionis Pharmaceuticals, Inc. | Methods for modulating Ataxin 2 expression |
| CA2942340A1 (en) | 2014-03-19 | 2015-09-24 | Ionis Pharmaceuticals, Inc. | Compositions 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 |
| WO2016094374A1 (en) * | 2014-12-09 | 2016-06-16 | The Board Of Regents Of The University Of Texas System | Compositions and mentods 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 |
| WO2016167780A1 (en) | 2015-04-16 | 2016-10-20 | Ionis Pharmaceuticals, Inc. | Compositions for modulating expression of c9orf72 antisense transcript |
| WO2016168592A2 (en) | 2015-04-16 | 2016-10-20 | Ionis Pharmaceuticals, Inc. | Compositions for modulating c9orf72 expression |
| US10675356B2 (en) | 2015-05-19 | 2020-06-09 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| PL3331891T3 (pl) | 2015-08-05 | 2022-03-28 | Eisai R&D Management Co., Ltd. | Sposób wytwarzania zasadniczo czystego diastereoizomerycznie oligomeru amidofosforanowego, oligomer amidofosforanowy wytworzony takim sposobem oraz kompozycja farmaceutyczna zawierająca taki oligomer amidofosforanowy |
| US10905709B2 (en) | 2015-08-28 | 2021-02-02 | Sarepta Therapeutics, Inc. | Modified antisense oligomers for exon inclusion in spinal muscular atrophy |
| WO2017079291A1 (en) * | 2015-11-02 | 2017-05-11 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating c90rf72 |
| SG10202004557QA (en) * | 2015-11-23 | 2020-06-29 | Univ California | Tracking and manipulating cellular rna via nuclear delivery of crispr/cas9 |
| EP3397288A4 (en) | 2015-12-31 | 2019-09-11 | Ionis Pharmaceuticals, Inc. | PROCESS 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 |
| IL263044B2 (en) | 2016-05-24 | 2024-06-01 | Sarepta Therapeutics Inc | Processes for preparing phosphorodiamidate morpholino oligomers |
| CN109152775B (zh) * | 2016-06-30 | 2022-04-26 | 萨勒普塔医疗公司 | 制备磷酸二酰胺吗啉代寡聚物的方法 |
| WO2018208998A1 (en) | 2017-05-10 | 2018-11-15 | The Regents Of The University Of California | Directed editing of cellular rna via nuclear delivery 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 |
| JP2021502882A (ja) * | 2017-11-15 | 2021-02-04 | アルキオーネ・ライフサイエンシズ・インコーポレイテッドＡｌｃｙｏｎｅ Ｌｉｆｅｓｃｉｅｎｃｅｓ， Ｉｎｃ． | 治療法別の事前プログラムされた自動注射デバイス |
| JP7394749B2 (ja) | 2018-05-10 | 2023-12-08 | 日本新薬株式会社 | オリゴ核酸化合物の製造方法 |
| EA202190299A1 (ru) | 2018-07-25 | 2021-06-11 | Ионис Фармасьютикалз, Инк. | Соединения и способы для снижения экспрессии atxn2 |
| EP3843845A4 (en) * | 2018-08-29 | 2022-05-11 | University Of Massachusetts | INHIBITION OF PROTEIN KINASE FOR THE TREATMENT OF FRIEDREICH'S ATAXIA |
| US11629347B2 (en) * | 2019-05-06 | 2023-04-18 | University Of Massachusetts | Anti-C9ORF72 oligonucleotides and related methods |
| AU2020368539A1 (en) | 2019-10-16 | 2022-04-28 | Massachusetts Institute Of Technology | Engineered muscle targeting compositions |
| JP7669280B2 (ja) | 2019-11-13 | 2025-04-28 | 日本新薬株式会社 | オリゴ核酸化合物の製造方法 |
| US20230018780A1 (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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| US5521063A (en) | 1985-03-15 | 1996-05-28 | Antivirals Inc. | Polynucleotide reagent containing chiral subunits and methods of use |
| 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 |
| CA1268404A (en) | 1985-03-15 | 1990-05-01 | Antivirals Inc. | Polynucleotide assay reagent and method |
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| US5185444A (en) | 1985-03-15 | 1993-02-09 | Anti-Gene Deveopment Group | Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages |
| AU659482B2 (en) | 1991-06-28 | 1995-05-18 | Massachusetts Institute Of Technology | Localized oligonucleotide therapy |
| PT1704878E (pt) | 1995-12-18 | 2013-07-17 | Angiodevice Internat Gmbh | Composições de polímeros reticulados e métodos para a sua utilização |
| US6245747B1 (en) | 1996-03-12 | 2001-06-12 | The Board Of Regents Of The University Of Nebraska | Targeted site specific antisense oligodeoxynucleotide delivery method |
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| PL2735568T3 (pl) | 2006-05-10 | 2018-02-28 | Sarepta Therapeutics, Inc. | Analogi oligonukleotydowe mające kationowe połączenia pomiędzy podjednostkami |
| 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 |
| US20100016215A1 (en) * | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
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| KR102095478B1 (ko) * | 2010-05-28 | 2020-04-01 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체 |
| CN103619356B (zh) | 2011-05-05 | 2017-09-12 | 萨勒普塔医疗公司 | 肽寡核苷酸缀合物 |
| US9161948B2 (en) * | 2011-05-05 | 2015-10-20 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| JP6317675B2 (ja) * | 2011-11-30 | 2018-04-25 | サレプタ セラピューティクス， インコーポレイテッド | 延長リピート病を処置するためのオリゴヌクレオチド |
| ES2727481T3 (es) | 2011-11-30 | 2019-10-16 | Sarepta Therapeutics Inc | Inclusión inducida de exón en atrofia muscular espinal |
-
2012
- 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
- 2012-11-30 ES ES12808975T patent/ES2832531T3/es active Active
- 2012-11-30 US US14/360,890 patent/US10066228B2/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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