JP2018518167A5 - - Google Patents
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- JP2018518167A5 JP2018518167A5 JP2017562026A JP2017562026A JP2018518167A5 JP 2018518167 A5 JP2018518167 A5 JP 2018518167A5 JP 2017562026 A JP2017562026 A JP 2017562026A JP 2017562026 A JP2017562026 A JP 2017562026A JP 2018518167 A5 JP2018518167 A5 JP 2018518167A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- sequence
- human type
- composition
- vii collagen
- 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.)
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- 150000001875 compounds Chemical class 0.000 claims description 61
- 102000004510 Collagen Type VII Human genes 0.000 claims description 56
- 108010017377 Collagen Type VII Proteins 0.000 claims description 56
- 230000000692 anti-sense effect Effects 0.000 claims description 56
- 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 group 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 51
- 230000008685 targeting Effects 0.000 claims description 48
- 125000003729 nucleotide group Chemical group 0.000 claims description 38
- 239000002773 nucleotide Substances 0.000 claims description 30
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical compound O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 claims description 28
- RWQNBRDOKXIBIV-UHFFFAOYSA-N thymine Chemical compound CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 claims description 28
- 238000000034 method Methods 0.000 claims description 25
- 108020004999 messenger RNA Proteins 0.000 claims description 24
- 239000000835 fiber Substances 0.000 claims description 22
- 238000009825 accumulation Methods 0.000 claims description 20
- 125000000217 alkyl group Chemical group 0.000 claims description 20
- -1 4-aminopiperidin-1-yl Chemical group 0.000 claims description 18
- 206010014989 Epidermolysis bullosa Diseases 0.000 claims description 16
- GLUUGHFHXGJENI-UHFFFAOYSA-N Piperazine Chemical compound C1CNCCN1 GLUUGHFHXGJENI-UHFFFAOYSA-N 0.000 claims description 16
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 16
- 208000035475 disorder Diseases 0.000 claims description 16
- 208000002720 Malnutrition Diseases 0.000 claims description 15
- 208000015380 nutritional deficiency disease Diseases 0.000 claims description 15
- 229940113082 thymine Drugs 0.000 claims description 14
- 229940035893 uracil Drugs 0.000 claims description 14
- 230000000295 complement effect Effects 0.000 claims description 12
- 229930010555 Inosine Natural products 0.000 claims description 10
- UGQMRVRMYYASKQ-KQYNXXCUSA-N Inosine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C2=NC=NC(O)=C2N=C1 UGQMRVRMYYASKQ-KQYNXXCUSA-N 0.000 claims description 10
- 229960003786 inosine Drugs 0.000 claims description 10
- 108091093037 Peptide nucleic acid Proteins 0.000 claims description 8
- 238000004873 anchoring Methods 0.000 claims description 8
- OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical compound NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 claims description 8
- 239000012634 fragment Substances 0.000 claims description 8
- UYTPUPDQBNUYGX-UHFFFAOYSA-N guanine Chemical compound O=C1NC(N)=NC2=C1N=CN2 UYTPUPDQBNUYGX-UHFFFAOYSA-N 0.000 claims description 8
- FDGQSTZJBFJUBT-UHFFFAOYSA-N hypoxanthine Chemical compound O=C1NC=NC2=C1NC=N2 FDGQSTZJBFJUBT-UHFFFAOYSA-N 0.000 claims description 8
- 108020004707 nucleic acids Proteins 0.000 claims description 8
- 102000039446 nucleic acids Human genes 0.000 claims description 8
- 150000007523 nucleic acids Chemical class 0.000 claims description 8
- 229960005141 piperazine Drugs 0.000 claims description 8
- ANCLJVISBRWUTR-UHFFFAOYSA-N diaminophosphinic acid Chemical compound NP(N)(O)=O ANCLJVISBRWUTR-UHFFFAOYSA-N 0.000 claims description 6
- 201000000744 recessive dystrophic epidermolysis bullosa Diseases 0.000 claims description 6
- 150000003839 salts Chemical class 0.000 claims description 6
- 208000010975 Dystrophic epidermolysis bullosa Diseases 0.000 claims description 5
- 208000004298 epidermolysis bullosa dystrophica Diseases 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
- MSSXOMSJDRHRMC-UHFFFAOYSA-N 9H-purine-2,6-diamine Chemical compound NC1=NC(N)=C2NC=NC2=N1 MSSXOMSJDRHRMC-UHFFFAOYSA-N 0.000 claims description 4
- 229930024421 Adenine Natural products 0.000 claims description 4
- GFFGJBXGBJISGV-UHFFFAOYSA-N Adenine Chemical compound NC1=NC=NC2=C1N=CN2 GFFGJBXGBJISGV-UHFFFAOYSA-N 0.000 claims description 4
- 239000005977 Ethylene Substances 0.000 claims description 4
- UGQMRVRMYYASKQ-UHFFFAOYSA-N Hypoxanthine nucleoside Natural products OC1C(O)C(CO)OC1N1C(NC=NC2=O)=C2N=C1 UGQMRVRMYYASKQ-UHFFFAOYSA-N 0.000 claims description 4
- 125000002252 acyl group Chemical group 0.000 claims description 4
- 229960000643 adenine Drugs 0.000 claims description 4
- 229940104302 cytosine Drugs 0.000 claims description 4
- 239000003814 drug Substances 0.000 claims description 4
- 125000001495 ethyl group Chemical group [H]C([H])([H])C([H])([H])* 0.000 claims description 4
- 125000005647 linker group Chemical group 0.000 claims description 4
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 claims description 4
- PTMHPRAIXMAOOB-UHFFFAOYSA-L phosphoramidate Chemical compound NP([O-])([O-])=O PTMHPRAIXMAOOB-UHFFFAOYSA-L 0.000 claims description 4
- 125000004437 phosphorous atom Chemical group 0.000 claims description 4
- 229910052698 phosphorus Inorganic materials 0.000 claims description 4
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims description 4
- 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 4
- 206010053177 Epidermolysis Diseases 0.000 claims description 3
- 230000001071 malnutrition Effects 0.000 claims description 3
- 235000000824 malnutrition Nutrition 0.000 claims description 3
- 239000004475 Arginine Substances 0.000 claims description 2
- 108010051109 Cell-Penetrating Peptides Proteins 0.000 claims description 2
- 102000020313 Cell-Penetrating Peptides Human genes 0.000 claims description 2
- ODKSFYDXXFIFQN-UHFFFAOYSA-N arginine Natural products OC(=O)C(N)CCCNC(N)=N ODKSFYDXXFIFQN-UHFFFAOYSA-N 0.000 claims description 2
- 125000003710 aryl alkyl group Chemical group 0.000 claims description 2
- 210000004899 c-terminal region Anatomy 0.000 claims description 2
- 210000004027 cell Anatomy 0.000 claims description 2
- 230000002401 inhibitory effect Effects 0.000 claims description 2
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 claims description 2
- 239000008194 pharmaceutical composition Substances 0.000 claims description 2
- 125000001644 phenoxazinyl group Chemical group C1(=CC=CC=2OC3=CC=CC=C3NC12)* 0.000 claims description 2
- 150000008299 phosphorodiamidates Chemical group 0.000 claims description 2
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 2
- 239000000203 mixture Substances 0.000 claims 26
- 0 *OP(*)(N(CC1)CCN1C(OCCOCCOCCO)=O)=O Chemical compound *OP(*)(N(CC1)CCN1C(OCCOCCOCCO)=O)=O 0.000 description 4
- QOJQSQLMUDYBFN-UHFFFAOYSA-N CN(C(CC1)CC[N]1(C1)C1=C)C(N)=N Chemical compound CN(C(CC1)CC[N]1(C1)C1=C)C(N)=N QOJQSQLMUDYBFN-UHFFFAOYSA-N 0.000 description 1
- FMFALOSHHNKGAA-UHFFFAOYSA-N CN(CC1)CCC1NC(N=N)=N Chemical compound CN(CC1)CCC1NC(N=N)=N FMFALOSHHNKGAA-UHFFFAOYSA-N 0.000 description 1
- PVOAHINGSUIXLS-UHFFFAOYSA-N CN1CCNCC1 Chemical compound CN1CCNCC1 PVOAHINGSUIXLS-UHFFFAOYSA-N 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021070962A JP2021130660A (ja) | 2015-06-01 | 2021-04-20 | Ｖｉｉ型コラーゲンにおけるアンチセンス誘導エクソン排除 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562169454P | 2015-06-01 | 2015-06-01 | |
| US62/169,454 | 2015-06-01 | ||
| PCT/US2016/035326 WO2016196670A1 (en) | 2015-06-01 | 2016-06-01 | Antisense-induced exon exclusion in type vii collagen |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021070962A Division JP2021130660A (ja) | 2015-06-01 | 2021-04-20 | Ｖｉｉ型コラーゲンにおけるアンチセンス誘導エクソン排除 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018518167A JP2018518167A (ja) | 2018-07-12 |
| JP2018518167A5 true JP2018518167A5 (OSRAM) | 2019-07-04 |
| JP6873052B2 JP6873052B2 (ja) | 2021-05-19 |
Family
ID=57441924
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017562026A Active JP6873052B2 (ja) | 2015-06-01 | 2016-06-01 | Ｖｉｉ型コラーゲンにおけるアンチセンス誘導エクソン排除 |
| JP2021070962A Pending JP2021130660A (ja) | 2015-06-01 | 2021-04-20 | Ｖｉｉ型コラーゲンにおけるアンチセンス誘導エクソン排除 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021070962A Pending JP2021130660A (ja) | 2015-06-01 | 2021-04-20 | Ｖｉｉ型コラーゲンにおけるアンチセンス誘導エクソン排除 |
Country Status (6)
| Country | Link |
|---|---|
| US (3) | US10849917B2 (OSRAM) |
| EP (2) | EP3302497A4 (OSRAM) |
| JP (2) | JP6873052B2 (OSRAM) |
| HK (1) | HK1253335A1 (OSRAM) |
| MA (1) | MA50829A (OSRAM) |
| WO (1) | WO2016196670A1 (OSRAM) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201504124D0 (en) | 2015-03-11 | 2015-04-22 | Proqr Therapeutics B V | Oligonucleotides |
| IL255180B2 (en) * | 2015-05-21 | 2023-11-01 | Proqr Therapeutics Ii Bv | Antisense oligonucleotides to treat dystrophic epidermolysis bullosa |
| HK1253335A1 (zh) | 2015-06-01 | 2019-06-14 | Sarepta Therapeutics, Inc. | 在vii型胶原蛋白中反义诱导外显子的排除 |
| KR102670349B1 (ko) * | 2016-12-30 | 2024-05-29 | 올리패스 주식회사 | 펩타이드 핵산 유도체들에 의한 엑손 스키핑 |
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| EA035030B1 (ru) | 2011-11-18 | 2020-04-20 | Сарепта Терапьютикс, Инк. | Модифицированные морфолиновые аналоги олигонуклеотидов |
| WO2013086444A2 (en) * | 2011-12-08 | 2013-06-13 | Sarepta Therapeutics, Inc. | Methods for treating progeroid laminopathies using oligonucleotide analogues targeting human lmna |
| CN111893117B (zh) | 2012-01-27 | 2024-06-04 | 比奥马林技术公司 | 治疗杜兴型肌营养不良症和贝克型肌营养不良症的具有改善特性的rna调节性寡核苷酸 |
| GB201202561D0 (en) * | 2012-02-15 | 2012-03-28 | Univ Dundee | Treatment of skin disorders |
| EP2754714A1 (en) | 2013-01-14 | 2014-07-16 | Sarepta Therapeutics, Inc. | Inhibitory oligonucleotides and their use in therapy |
| RS66199B1 (sr) | 2014-02-04 | 2024-12-31 | The United States Of America As Represented By The Secretary Department Of Health And Human Services | Metode za proizvodnju autolognih t ćelija koje su korisne za lečenje b ćelijskih maligniteta i drugih karcinoma i njihove kompozicije |
| GB201504124D0 (en) * | 2015-03-11 | 2015-04-22 | Proqr Therapeutics B V | Oligonucleotides |
| IL255180B2 (en) * | 2015-05-21 | 2023-11-01 | Proqr Therapeutics Ii Bv | Antisense oligonucleotides to treat dystrophic epidermolysis bullosa |
| HK1253335A1 (zh) | 2015-06-01 | 2019-06-14 | Sarepta Therapeutics, Inc. | 在vii型胶原蛋白中反义诱导外显子的排除 |
| JP7394753B2 (ja) | 2017-10-18 | 2023-12-08 | サレプタ セラピューティクス， インコーポレイテッド | アンチセンスオリゴマー化合物 |
| US10867398B2 (en) | 2017-11-21 | 2020-12-15 | Reliance Core Consulting LLC | Methods, systems, apparatuses and devices for facilitating motion analysis in an environment |
-
2016
- 2016-06-01 HK HK18112628.5A patent/HK1253335A1/zh unknown
- 2016-06-01 WO PCT/US2016/035326 patent/WO2016196670A1/en not_active Ceased
- 2016-06-01 EP EP16804357.8A patent/EP3302497A4/en not_active Withdrawn
- 2016-06-01 MA MA050829A patent/MA50829A/fr unknown
- 2016-06-01 US US15/578,612 patent/US10849917B2/en active Active
- 2016-06-01 JP JP2017562026A patent/JP6873052B2/ja active Active
- 2016-06-01 EP EP20210746.2A patent/EP3851531A1/en not_active Withdrawn
-
2020
- 2020-10-26 US US17/080,363 patent/US11911403B2/en active Active
-
2021
- 2021-04-20 JP JP2021070962A patent/JP2021130660A/ja active Pending
-
2024
- 2024-01-11 US US18/410,720 patent/US20240415857A1/en not_active Abandoned
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