JP2018538302A5 - - Google Patents
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- Publication number
- JP2018538302A5 JP2018538302A5 JP2018531210A JP2018531210A JP2018538302A5 JP 2018538302 A5 JP2018538302 A5 JP 2018538302A5 JP 2018531210 A JP2018531210 A JP 2018531210A JP 2018531210 A JP2018531210 A JP 2018531210A JP 2018538302 A5 JP2018538302 A5 JP 2018538302A5
- Authority
- JP
- Japan
- Prior art keywords
- alkyl
- oligomer
- peptide
- pharmaceutically acceptable
- acceptable salt
- 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.)
- Pending
Links
- 150000003839 salts Chemical class 0.000 claims description 94
- 125000000217 alkyl group Chemical group 0.000 claims description 61
- -1 monomethoxytrityl Chemical group 0.000 claims description 27
- PXBRQCKWGAHEHS-UHFFFAOYSA-N dichlorodifluoromethane Chemical compound FC(F)(Cl)Cl PXBRQCKWGAHEHS-UHFFFAOYSA-N 0.000 claims description 20
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 17
- 125000003236 benzoyl group Chemical group [H]C1=C([H])C([H])=C(C([H])=C1[H])C(*)=O 0.000 claims description 13
- 125000003696 stearoyl group Chemical group O=C([*])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])[H] 0.000 claims description 13
- KDCGOANMDULRCW-UHFFFAOYSA-N 7H-purine Chemical compound N1=CNC2=NC=NC2=C1 KDCGOANMDULRCW-UHFFFAOYSA-N 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
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical compound O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 claims description 10
- OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical compound NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 claims description 10
- UYTPUPDQBNUYGX-UHFFFAOYSA-N guanine Chemical compound O=C1NC(N)=NC2=C1N=CN2 UYTPUPDQBNUYGX-UHFFFAOYSA-N 0.000 claims description 10
- FDGQSTZJBFJUBT-UHFFFAOYSA-N hypoxanthine Chemical compound O=C1NC=NC2=C1NC=N2 FDGQSTZJBFJUBT-UHFFFAOYSA-N 0.000 claims description 10
- RWQNBRDOKXIBIV-UHFFFAOYSA-N thymine Chemical compound CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 claims description 10
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 6
- PEHVGBZKEYRQSX-UHFFFAOYSA-N 7-deaza-adenine Chemical compound NC1=NC=NC2=C1C=CN2 PEHVGBZKEYRQSX-UHFFFAOYSA-N 0.000 claims description 6
- JUJWROOIHBZHMG-UHFFFAOYSA-N Pyridine Chemical compound C1=CC=NC=C1 JUJWROOIHBZHMG-UHFFFAOYSA-N 0.000 claims description 6
- GFFGJBXGBJISGV-UHFFFAOYSA-N Adenine Chemical compound NC1=NC=NC2=C1N=CN2 GFFGJBXGBJISGV-UHFFFAOYSA-N 0.000 claims description 5
- 229930024421 Adenine Natural products 0.000 claims description 5
- UGQMRVRMYYASKQ-UHFFFAOYSA-N Hypoxanthine nucleoside Natural products OC1C(O)C(CO)OC1N1C(NC=NC2=O)=C2N=C1 UGQMRVRMYYASKQ-UHFFFAOYSA-N 0.000 claims description 5
- 229960000643 adenine Drugs 0.000 claims description 5
- 229940104302 cytosine Drugs 0.000 claims description 5
- 229940113082 thymine Drugs 0.000 claims description 5
- 229940035893 uracil Drugs 0.000 claims description 5
- LRSASMSXMSNRBT-UHFFFAOYSA-N 5-methylcytosine Chemical compound CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 claims description 4
- CZPWVGJYEJSRLH-UHFFFAOYSA-N Pyrimidine Chemical compound C1=CN=CN=C1 CZPWVGJYEJSRLH-UHFFFAOYSA-N 0.000 claims description 4
- 125000000623 heterocyclic group Chemical group 0.000 claims description 4
- LOSIULRWFAEMFL-UHFFFAOYSA-N 7-deazaguanine Chemical compound O=C1NC(N)=NC2=C1CC=N2 LOSIULRWFAEMFL-UHFFFAOYSA-N 0.000 claims description 3
- MSSXOMSJDRHRMC-UHFFFAOYSA-N 9H-purine-2,6-diamine Chemical compound NC1=NC(N)=C2NC=NC2=N1 MSSXOMSJDRHRMC-UHFFFAOYSA-N 0.000 claims description 3
- 125000002795 guanidino group Chemical group C(N)(=N)N* 0.000 claims description 3
- UMJSCPRVCHMLSP-UHFFFAOYSA-N pyridine Natural products COC1=CC=CN=C1 UMJSCPRVCHMLSP-UHFFFAOYSA-N 0.000 claims description 3
- 108091034117 Oligonucleotide Proteins 0.000 claims description 2
- 125000003118 aryl group Chemical group 0.000 claims description 2
- 108091092328 cellular RNA Proteins 0.000 claims description 2
- 125000004404 heteroalkyl group Chemical group 0.000 claims description 2
- 229910052760 oxygen Inorganic materials 0.000 claims description 2
- 125000006239 protecting group Chemical group 0.000 claims description 2
- 239000007787 solid Substances 0.000 claims description 2
- 239000000126 substance Substances 0.000 claims description 2
- 229910052717 sulfur Inorganic materials 0.000 claims description 2
- 208000035143 Bacterial infection Diseases 0.000 description 3
- 208000021642 Muscular disease Diseases 0.000 description 3
- 208000012902 Nervous system disease Diseases 0.000 description 3
- 208000036142 Viral infection Diseases 0.000 description 3
- 208000022362 bacterial infectious disease Diseases 0.000 description 3
- 208000015114 central nervous system disease Diseases 0.000 description 3
- 230000009385 viral infection Effects 0.000 description 3
- 102000004196 processed proteins & peptides Human genes 0.000 description 2
- OYWRDHBGMCXGFY-UHFFFAOYSA-N 1,2,3-triazinane Chemical compound C1CNNNC1 OYWRDHBGMCXGFY-UHFFFAOYSA-N 0.000 description 1
- HWPZZUQOWRWFDB-UHFFFAOYSA-N 1-methylcytosine Chemical compound CN1C=CC(N)=NC1=O HWPZZUQOWRWFDB-UHFFFAOYSA-N 0.000 description 1
- 241000725619 Dengue virus Species 0.000 description 1
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 description 1
- 241001115402 Ebolavirus Species 0.000 description 1
- 241001115401 Marburgvirus Species 0.000 description 1
- 241000187479 Mycobacterium tuberculosis Species 0.000 description 1
- 241000700605 Viruses Species 0.000 description 1
- 208000002320 spinal muscular atrophy Diseases 0.000 description 1
- 241000712461 unidentified influenza virus Species 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562267723P | 2015-12-15 | 2015-12-15 | |
| US62/267,723 | 2015-12-15 | ||
| PCT/US2016/066595 WO2017106304A1 (en) | 2015-12-15 | 2016-12-14 | Peptide oligonucleotide conjugates |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2018538302A JP2018538302A (ja) | 2018-12-27 |
| JP2018538302A5 true JP2018538302A5 (enExample) | 2020-01-30 |
Family
ID=59057789
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018531210A Pending JP2018538302A (ja) | 2015-12-15 | 2016-12-14 | ペプチドオリゴヌクレオチド複合体 |
Country Status (15)
| Country | Link |
|---|---|
| US (2) | US20190030176A1 (enExample) |
| EP (1) | EP3389719B1 (enExample) |
| JP (1) | JP2018538302A (enExample) |
| KR (1) | KR20180088874A (enExample) |
| CN (1) | CN108472504A (enExample) |
| AR (1) | AR107220A1 (enExample) |
| AU (1) | AU2016372026A1 (enExample) |
| BR (1) | BR112018012237A2 (enExample) |
| CA (1) | CA3006748A1 (enExample) |
| EA (1) | EA201891419A1 (enExample) |
| ES (1) | ES2901772T3 (enExample) |
| IL (2) | IL259587A (enExample) |
| MX (1) | MX2018007307A (enExample) |
| TW (1) | TWI740866B (enExample) |
| WO (1) | WO2017106304A1 (enExample) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2020368539A1 (en) | 2019-10-16 | 2022-04-28 | Massachusetts Institute Of Technology | Engineered muscle targeting compositions |
| KR102530865B1 (ko) * | 2020-09-09 | 2023-05-10 | 연세대학교 산학협력단 | 자기조립 고리형 펩티드-올리고뉴클레오티드 복합체 및 이의 제조방법 |
| US20250011777A1 (en) * | 2021-09-03 | 2025-01-09 | Sarepta Therapeutics, Inc. | Delivery of anitsense oligomers by mirror image peptides |
| JP2024539223A (ja) * | 2021-10-22 | 2024-10-28 | サレプタ セラピューティクス， インコーポレイテッド | 末梢ミエリンタンパク質２２関連疾患の処置のためのモルフォリノオリゴマー |
| CA3262097A1 (en) | 2022-07-14 | 2024-01-18 | The Broad Institute, Inc. | AAV capsids that allow gene distribution across the central nervous system via interactions with the transferrin receptor |
| WO2025155923A1 (en) | 2024-01-17 | 2025-07-24 | The Broad Institute, Inc. | Aav capsid modifications that enable improved cns-wide gene delivery through interactions with the transferrin receptor |
| WO2025217174A1 (en) | 2024-04-08 | 2025-10-16 | The Broad Institute, Inc. | Aav capsid modifications that enable improved cns-wide gene delivery through interactions with carbonic anhydrase iv |
| WO2025217163A2 (en) | 2024-04-08 | 2025-10-16 | The Broad Institute, Inc. | Novel aav capsids binding to human cd59 |
Family Cites Families (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5521063A (en) | 1985-03-15 | 1996-05-28 | Antivirals Inc. | Polynucleotide reagent containing chiral subunits and methods of use |
| US5217866A (en) | 1985-03-15 | 1993-06-08 | Anti-Gene Development Group | Polynucleotide assay reagent and method |
| 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 |
| US5185444A (en) | 1985-03-15 | 1993-02-09 | Anti-Gene Deveopment Group | Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages |
| AU5661386A (en) | 1985-03-15 | 1986-10-13 | Stirchak, E. | Stereoregular polynucleotide-binding polymers |
| US5166315A (en) | 1989-12-20 | 1992-11-24 | Anti-Gene Development Group | Sequence-specific binding polymers for duplex nucleic acids |
| US6660843B1 (en) * | 1998-10-23 | 2003-12-09 | Amgen Inc. | Modified peptides as therapeutic agents |
| JP2004500438A (ja) * | 2000-04-14 | 2004-01-08 | アルニス バイオサイエンシーズ， インコーポレイテッド | 高親和性のペプチド含有ナノ粒子 |
| WO2002042259A2 (en) * | 2000-11-27 | 2002-05-30 | University Of Maryland, Baltimore | Methods of synthesizing and using derivatives of [2-(2-aminoethoxy)ethoxy] acetic acid |
| US20070107072A1 (en) * | 2003-11-07 | 2007-05-10 | Peter Carmeliet | Transgenic amphibian models for lymphatic vessel development |
| US20050288246A1 (en) * | 2004-05-24 | 2005-12-29 | Iversen Patrick L | Peptide conjugated, inosine-substituted antisense oligomer compound and method |
| JP5066095B2 (ja) * | 2005-11-17 | 2012-11-07 | ボード・オブ・リージエンツ，ザ・ユニバーシテイ・オブ・テキサス・システム | 染色体ｄｎａに標的化されるオリゴマーによる遺伝子発現の調節 |
| US8658608B2 (en) * | 2005-11-23 | 2014-02-25 | Yale University | Modified triple-helix forming oligonucleotides for targeted mutagenesis |
| WO2007069068A2 (en) * | 2005-12-16 | 2007-06-21 | Diatos | Cell penetrating peptide conjugates for delivering nucleic acids into cells |
| US8299206B2 (en) | 2007-11-15 | 2012-10-30 | Avi Biopharma, Inc. | Method of synthesis of morpholino oligomers |
| CA2884340C (en) | 2007-11-15 | 2017-07-25 | Sarepta Therapeutics, Inc. | Method of synthesis of morpholino oligomers |
| US8076476B2 (en) | 2007-11-15 | 2011-12-13 | Avi Biopharma, Inc. | Synthesis of morpholino oligomers using doubly protected guanine morpholino subunits |
| CA2721982A1 (en) * | 2008-04-30 | 2009-11-05 | Novartis Ag | Assay for pathogenic conformers |
| PL2623507T3 (pl) * | 2010-09-30 | 2017-03-31 | Nippon Shinyaku Co., Ltd. | Pochodna kwasu morfolinonukleinowego |
| EP2681336A4 (en) * | 2011-03-02 | 2014-11-19 | Groove Biopharma Corp | ENHANCED BIODISTRIBUTION OF OLIGOMERS |
| BR112014011875B1 (pt) * | 2011-11-18 | 2022-01-04 | Sarepta Therapeutics, Inc | Oligonucleotídeos funcionalmente modificados e subunidades dos mesmos |
| MX369519B (es) * | 2012-09-25 | 2019-11-11 | Genzyme Corp | Oligonucleotidos antisentido morfolino unidos a peptido para el tratamiento de distrofia miotonica. |
| NZ732507A (en) * | 2013-03-15 | 2018-08-31 | Sarepta Therapeutics Inc | Improved compositions for treating muscular dystrophy |
| US20180216111A1 (en) * | 2015-02-27 | 2018-08-02 | Sarepta Therapeutics, Inc. | Antisense-induced exon2 inclusion in acid alpha-glucosidase |
-
2016
- 2016-12-14 MX MX2018007307A patent/MX2018007307A/es unknown
- 2016-12-14 CN CN201680074252.6A patent/CN108472504A/zh active Pending
- 2016-12-14 AU AU2016372026A patent/AU2016372026A1/en not_active Abandoned
- 2016-12-14 US US16/061,971 patent/US20190030176A1/en not_active Abandoned
- 2016-12-14 CA CA3006748A patent/CA3006748A1/en not_active Abandoned
- 2016-12-14 ES ES16876554T patent/ES2901772T3/es active Active
- 2016-12-14 KR KR1020187018470A patent/KR20180088874A/ko not_active Withdrawn
- 2016-12-14 EA EA201891419A patent/EA201891419A1/ru unknown
- 2016-12-14 WO PCT/US2016/066595 patent/WO2017106304A1/en not_active Ceased
- 2016-12-14 JP JP2018531210A patent/JP2018538302A/ja active Pending
- 2016-12-14 EP EP16876554.3A patent/EP3389719B1/en active Active
- 2016-12-14 BR BR112018012237A patent/BR112018012237A2/pt not_active IP Right Cessation
- 2016-12-15 AR ARP160103839A patent/AR107220A1/es not_active Application Discontinuation
- 2016-12-15 TW TW105141658A patent/TWI740866B/zh not_active IP Right Cessation
-
2018
- 2018-05-24 IL IL259587A patent/IL259587A/en unknown
-
2021
- 2021-07-11 IL IL284753A patent/IL284753A/en unknown
-
2024
- 2024-05-08 US US18/658,614 patent/US20250057965A1/en active Pending
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