JP2016533761A5 - - Google Patents
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- JP2016533761A5 JP2016533761A5 JP2016540437A JP2016540437A JP2016533761A5 JP 2016533761 A5 JP2016533761 A5 JP 2016533761A5 JP 2016540437 A JP2016540437 A JP 2016540437A JP 2016540437 A JP2016540437 A JP 2016540437A JP 2016533761 A5 JP2016533761 A5 JP 2016533761A5
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- Prior art keywords
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- aralkyl
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- 125000000217 alkyl group Chemical group 0.000 claims description 90
- 229910052739 hydrogen Inorganic materials 0.000 claims description 90
- 239000001257 hydrogen Substances 0.000 claims description 90
- 150000002431 hydrogen Chemical class 0.000 claims description 66
- 125000003710 aryl alkyl group Chemical group 0.000 claims description 60
- RWQNBRDOKXIBIV-UHFFFAOYSA-N Thymine Chemical compound CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 claims description 40
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical group O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 claims description 40
- 150000001875 compounds Chemical class 0.000 claims description 33
- 229920002459 Intron Polymers 0.000 claims description 28
- 102000019679 Cell-Penetrating Peptides Human genes 0.000 claims description 27
- 108010051109 Cell-Penetrating Peptides Proteins 0.000 claims description 27
- 125000003729 nucleotide group Chemical group 0.000 claims description 26
- UFHFLCQGNIYNRP-UHFFFAOYSA-N hydrogen Chemical compound [H][H] UFHFLCQGNIYNRP-UHFFFAOYSA-N 0.000 claims description 24
- 229940113082 Thymine Drugs 0.000 claims description 20
- 229940035893 Uracil Drugs 0.000 claims description 20
- 239000002773 nucleotide Substances 0.000 claims description 20
- 239000000203 mixture Substances 0.000 claims description 19
- 125000002252 acyl group Chemical group 0.000 claims description 18
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 claims description 18
- 230000000692 anti-sense Effects 0.000 claims description 16
- 101000525742 GAA Proteins 0.000 claims description 14
- 108020004999 Messenger RNA Proteins 0.000 claims description 14
- 102000027592 human GAA protein Human genes 0.000 claims description 14
- 229920002106 messenger RNA Polymers 0.000 claims description 14
- 239000008194 pharmaceutical composition Substances 0.000 claims description 13
- 229920002224 Peptide nucleic acid Polymers 0.000 claims description 12
- 125000002887 hydroxy group Chemical group [H]O* 0.000 claims description 12
- 229910052757 nitrogen Inorganic materials 0.000 claims description 12
- 150000002829 nitrogen Chemical group 0.000 claims description 12
- 125000001424 substituent group Chemical group 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
- 230000000295 complement Effects 0.000 claims description 10
- 229910052799 carbon Inorganic materials 0.000 claims description 9
- 125000001188 haloalkyl group Chemical group 0.000 claims description 9
- 125000003884 phenylalkyl group Chemical group 0.000 claims description 9
- 208000007345 Glycogen Storage Disease Diseases 0.000 claims description 8
- 150000003839 salts Chemical class 0.000 claims description 8
- 239000011780 sodium chloride Substances 0.000 claims description 8
- 125000003545 alkoxy group Chemical group 0.000 claims description 6
- 125000003282 alkyl amino group Chemical group 0.000 claims description 6
- 125000004429 atoms Chemical group 0.000 claims description 6
- 125000002915 carbonyl group Chemical group [*:2]C([*:1])=O 0.000 claims description 6
- 229920003013 deoxyribonucleic acid Polymers 0.000 claims description 6
- 108020004707 nucleic acids Proteins 0.000 claims description 6
- 150000007523 nucleic acids Chemical class 0.000 claims description 6
- MYMOFIZGZYHOMD-UHFFFAOYSA-N oxygen Chemical group O=O MYMOFIZGZYHOMD-UHFFFAOYSA-N 0.000 claims description 6
- 229910052760 oxygen Inorganic materials 0.000 claims description 6
- 239000001301 oxygen Substances 0.000 claims description 6
- QVGXLLKOCUKJST-UHFFFAOYSA-N oxygen atom Chemical group [O] QVGXLLKOCUKJST-UHFFFAOYSA-N 0.000 claims description 6
- 239000000126 substance Substances 0.000 claims description 6
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims description 6
- FDGQSTZJBFJUBT-UHFFFAOYSA-N Hypoxanthine Chemical compound O=C1NC=NC2=C1NC=N2 FDGQSTZJBFJUBT-UHFFFAOYSA-N 0.000 claims description 4
- 239000003937 drug carrier Substances 0.000 claims description 4
- -1 -C (= O) OR g Chemical group 0.000 claims description 2
- MSSXOMSJDRHRMC-UHFFFAOYSA-N 2,6-Diaminopurine Chemical compound NC1=NC(N)=C2NC=NC2=N1 MSSXOMSJDRHRMC-UHFFFAOYSA-N 0.000 claims description 2
- IZGFERLHOCMZQF-UHFFFAOYSA-N 2-(10H-phenoxazin-1-yloxy)ethanamine Chemical compound O1C2=CC=CC=C2NC2=C1C=CC=C2OCCN IZGFERLHOCMZQF-UHFFFAOYSA-N 0.000 claims description 2
- LRSASMSXMSNRBT-UHFFFAOYSA-N 5-Methylcytosine Chemical compound CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 claims description 2
- 229960000643 Adenine Drugs 0.000 claims description 2
- GFFGJBXGBJISGV-UHFFFAOYSA-N Adenine Natural products NC1=NC=NC2=C1N=CN2 GFFGJBXGBJISGV-UHFFFAOYSA-N 0.000 claims description 2
- OPTASPLRGRRNAP-UHFFFAOYSA-N Cytosine Chemical compound NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 claims description 2
- 229940104302 Cytosine Drugs 0.000 claims description 2
- UYTPUPDQBNUYGX-UHFFFAOYSA-N Guanine Chemical compound O=C1NC(N)=NC2=C1N=CN2 UYTPUPDQBNUYGX-UHFFFAOYSA-N 0.000 claims description 2
- OIRDTQYFTABQOQ-KQYNXXCUSA-N adenosine Chemical compound C1=NC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O OIRDTQYFTABQOQ-KQYNXXCUSA-N 0.000 claims description 2
- 229910052736 halogen Inorganic materials 0.000 claims 3
- 150000002367 halogens Chemical class 0.000 claims 3
- 125000001997 phenyl group Chemical group [H]C1=C([H])C([H])=C(*)C([H])=C1[H] 0.000 claims 3
- 0 CN1C*CCCC1 Chemical compound CN1C*CCCC1 0.000 description 2
- LJLWVVCWBURGCC-UHFFFAOYSA-N CC(C)C(C)NC Chemical compound CC(C)C(C)NC LJLWVVCWBURGCC-UHFFFAOYSA-N 0.000 description 1
- 210000002356 Skeleton Anatomy 0.000 description 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361874261P | 2013-09-05 | 2013-09-05 | |
| US61/874,261 | 2013-09-05 | ||
| US201461932195P | 2014-01-27 | 2014-01-27 | |
| US61/932,195 | 2014-01-27 | ||
| PCT/US2014/054384 WO2015035231A1 (en) | 2013-09-05 | 2014-09-05 | Antisense-induced exon2 inclusion in acid alpha-glucosidase |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018227359A Division JP6671449B2 (ja) | 2013-09-05 | 2018-12-04 | 酸性α−グルコシダーゼにおけるアンチセンス誘導エクソン２包含 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2016533761A JP2016533761A (ja) | 2016-11-04 |
| JP2016533761A5 true JP2016533761A5 (US07794700-20100914-C00152.png) | 2017-10-12 |
| JP6618910B2 JP6618910B2 (ja) | 2019-12-11 |
Family
ID=51541398
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016540437A Active JP6618910B2 (ja) | 2013-09-05 | 2014-09-05 | 酸性α−グルコシダーゼにおけるアンチセンス誘導エクソン２包含 |
| JP2018227359A Active JP6671449B2 (ja) | 2013-09-05 | 2018-12-04 | 酸性α−グルコシダーゼにおけるアンチセンス誘導エクソン２包含 |
| JP2020035519A Active JP6865871B2 (ja) | 2013-09-05 | 2020-03-03 | 酸性α−グルコシダーゼにおけるアンチセンス誘導エクソン２包含 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018227359A Active JP6671449B2 (ja) | 2013-09-05 | 2018-12-04 | 酸性α−グルコシダーゼにおけるアンチセンス誘導エクソン２包含 |
| JP2020035519A Active JP6865871B2 (ja) | 2013-09-05 | 2020-03-03 | 酸性α−グルコシダーゼにおけるアンチセンス誘導エクソン２包含 |
Country Status (11)
Families Citing this family (24)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2006086667A2 (en) | 2005-02-09 | 2006-08-17 | Avi Bio Pharma, Inc. | Antisense composition and method for treating muscle atrophy |
| US20130085139A1 (en) | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
| JP6618910B2 (ja) | 2013-09-05 | 2019-12-11 | サレプタ セラピューティクス，インコーポレイテッド | 酸性α−グルコシダーゼにおけるアンチセンス誘導エクソン２包含 |
| EP3480312A1 (en) | 2013-09-11 | 2019-05-08 | Synthena AG | Nucleic acids and methods for the treatment of pompe disease |
| WO2015190922A1 (en) * | 2014-06-10 | 2015-12-17 | Erasmus University Medical Center Rotterdam | Antisense oligonucleotides useful in treatment of pompe disease |
| GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| CA2963288A1 (en) | 2014-10-03 | 2016-04-07 | Cold Spring Harbor Laboratory | Targeted augmentation of nuclear gene output |
| WO2016138534A2 (en) * | 2015-02-27 | 2016-09-01 | Sarepta Therapeutics, Inc. | Antisense-induced exon2 inclusion in acid alpha-glucosidase |
| MA41795A (fr) | 2015-03-18 | 2018-01-23 | Sarepta Therapeutics Inc | Exclusion d'un exon induite par des composés antisens dans la myostatine |
| US10196639B2 (en) | 2015-10-09 | 2019-02-05 | University Of Southampton | Modulation of gene expression and screening for deregulated protein expression |
| CN108699555A (zh) * | 2015-10-09 | 2018-10-23 | 萨勒普塔医疗公司 | 用于治疗杜兴肌营养不良和相关病症的组合物和方法 |
| CA3007152A1 (en) | 2015-12-07 | 2017-06-15 | Erasmus University Medical Center Rotterdam | Enzymatic replacement therapy and antisense therapy for pompe disease |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| EP3390636B1 (en) | 2015-12-14 | 2021-05-19 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of dravet syndrome |
| EA201892366A1 (ru) * | 2016-04-18 | 2019-03-29 | Сарепта Терапьютикс, Инк. | Антисмысловые олигомеры и способы их применения для лечения заболеваний, связанных с геном кислой альфа-глюкозидазы |
| NL2017295B1 (en) * | 2016-08-05 | 2018-02-14 | Univ Erasmus Med Ct Rotterdam | Antisense oligomeric compound for Pompe disease |
| NL2017294B1 (en) * | 2016-08-05 | 2018-02-14 | Univ Erasmus Med Ct Rotterdam | Natural cryptic exon removal by pairs of antisense oligonucleotides. |
| DK3554553T3 (da) * | 2016-12-19 | 2022-09-19 | Sarepta Therapeutics Inc | Exon-overspringnings-oligomerkonjugat til muskeldystrofi |
| WO2019040923A1 (en) | 2017-08-25 | 2019-02-28 | Stoke Therapeutics, Inc. | ANTISENSE OLIGOMERS FOR THE TREATMENT OF DISEASE CONDITIONS AND OTHER DISEASES |
| NL2019517B1 (en) * | 2017-09-08 | 2019-03-19 | Univ Erasmus Med Ct Rotterdam | New therapy for Pompe disease |
| TWI812647B (zh) * | 2017-09-25 | 2023-08-21 | 美商薩羅塔治療公司 | 經由速流合成以製備磷醯二胺嗎啉代寡聚物之製程 |
| MX2020011695A (es) | 2018-05-04 | 2021-02-26 | Stoke Therapeutics Inc | Métodos y composiciones para el tratamiento de la enfermedad por almacenamiento de éster de colesterilo. |
| EP4150092A1 (en) | 2020-05-11 | 2023-03-22 | Stoke Therapeutics, Inc. | Opa1 antisense oligomers for treatment of conditions and diseases |
| KR20210151593A (ko) * | 2020-06-05 | 2021-12-14 | 넥스올리고(주) | 신규한 몰포리노 올리고뉴클레오티드 유도체 |
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