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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- 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
- 125000000217 alkyl group Chemical group 0.000 claims description 57
- 230000008685 targeting Effects 0.000 claims description 43
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical group O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 claims description 40
- RWQNBRDOKXIBIV-UHFFFAOYSA-N thymine Chemical compound CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 claims description 40
- 150000001875 compounds Chemical class 0.000 claims description 33
- 102000020313 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
- 239000002773 nucleotide Substances 0.000 claims description 20
- 229940113082 thymine Drugs 0.000 claims description 20
- 229940035893 uracil Drugs 0.000 claims description 20
- 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 effect Effects 0.000 claims description 16
- 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 compound 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 14
- 101001018026 Homo sapiens Lysosomal alpha-glucosidase Proteins 0.000 claims description 14
- 102000045921 human GAA Human genes 0.000 claims description 14
- 108020004999 messenger RNA Proteins 0.000 claims description 14
- 108090000623 proteins and genes Proteins 0.000 claims description 14
- 239000008194 pharmaceutical composition Substances 0.000 claims description 13
- 108091093037 Peptide nucleic acid Proteins 0.000 claims description 12
- QVGXLLKOCUKJST-UHFFFAOYSA-N atomic oxygen Chemical group [O] QVGXLLKOCUKJST-UHFFFAOYSA-N 0.000 claims description 12
- 229910052736 halogen Inorganic materials 0.000 claims description 12
- 150000002367 halogens Chemical class 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
- 125000004433 nitrogen atom Chemical group N* 0.000 claims description 12
- 125000001997 phenyl group Chemical group [H]C1=C([H])C([H])=C(*)C([H])=C1[H] 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 effect Effects 0.000 claims description 10
- 208000007345 glycogen storage disease Diseases 0.000 claims description 8
- 150000003839 salts Chemical class 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 atom Chemical group 0.000 claims description 6
- 229910052799 carbon Inorganic materials 0.000 claims description 6
- 125000002915 carbonyl group Chemical group [*:2]C([*:1])=O 0.000 claims description 6
- 108020004707 nucleic acids Proteins 0.000 claims description 6
- 102000039446 nucleic acids Human genes 0.000 claims description 6
- 150000007523 nucleic acids Chemical class 0.000 claims description 6
- 229910052760 oxygen Inorganic materials 0.000 claims description 6
- 239000001301 oxygen Substances 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
- -1 C 1 -C 6 alkyl Substances 0.000 claims description 5
- OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical compound NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 claims description 4
- 239000003937 drug carrier Substances 0.000 claims description 4
- UYTPUPDQBNUYGX-UHFFFAOYSA-N guanine Chemical compound O=C1NC(N)=NC2=C1N=CN2 UYTPUPDQBNUYGX-UHFFFAOYSA-N 0.000 claims description 4
- FDGQSTZJBFJUBT-UHFFFAOYSA-N hypoxanthine Chemical compound O=C1NC=NC2=C1NC=N2 FDGQSTZJBFJUBT-UHFFFAOYSA-N 0.000 claims description 4
- 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
- MSSXOMSJDRHRMC-UHFFFAOYSA-N 9H-purine-2,6-diamine Chemical compound NC1=NC(N)=C2NC=NC2=N1 MSSXOMSJDRHRMC-UHFFFAOYSA-N 0.000 claims description 2
- GFFGJBXGBJISGV-UHFFFAOYSA-N Adenine Chemical compound NC1=NC=NC2=C1N=CN2 GFFGJBXGBJISGV-UHFFFAOYSA-N 0.000 claims description 2
- 229930024421 Adenine Natural products 0.000 claims description 2
- UGQMRVRMYYASKQ-UHFFFAOYSA-N Hypoxanthine nucleoside Natural products OC1C(O)C(CO)OC1N1C(NC=NC2=O)=C2N=C1 UGQMRVRMYYASKQ-UHFFFAOYSA-N 0.000 claims description 2
- 229960000643 adenine Drugs 0.000 claims description 2
- 229940104302 cytosine Drugs 0.000 claims description 2
- 239000000203 mixture Substances 0.000 claims 7
- 108090000765 processed proteins & peptides Proteins 0.000 claims 3
- 238000000034 method Methods 0.000 description 6
- 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
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 (OSRAM) | 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)
| Country | Link |
|---|---|
| US (3) | US11236338B2 (OSRAM) |
| EP (1) | EP3041935A1 (OSRAM) |
| JP (3) | JP6618910B2 (OSRAM) |
| KR (2) | KR20210088009A (OSRAM) |
| CN (2) | CN111235149B (OSRAM) |
| AU (2) | AU2014317961B2 (OSRAM) |
| CA (1) | CA2922838A1 (OSRAM) |
| IL (4) | IL300444A (OSRAM) |
| MX (1) | MX384377B (OSRAM) |
| TW (1) | TWI736514B (OSRAM) |
| WO (1) | WO2015035231A1 (OSRAM) |
Families Citing this family (24)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2852549T3 (es) | 2005-02-09 | 2021-09-13 | Sarepta Therapeutics Inc | Composición antisentido para tratamiento de la atrofia muscular |
| US20130085139A1 (en) | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
| IL300444A (en) | 2013-09-05 | 2023-04-01 | Sarepta Therapeutics Inc | Inclusion of exon 2 is induced by antisense in acid alpha-glucosidase |
| EP3480312A1 (en) * | 2013-09-11 | 2019-05-08 | Synthena AG | Nucleic acids and methods for the treatment of pompe disease |
| KR102524543B1 (ko) * | 2014-06-10 | 2023-04-20 | 에라스무스 유니버시티 메디컬 센터 로테르담 | 폼페병의 치료에 유용한 안티센스 올리고뉴클레오티드 |
| 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 |
| MX2017011004A (es) * | 2015-02-27 | 2018-02-09 | Sarepta Therapeutics Inc | Inclusion del exon2 inducida por antisentido en alfa-glucosidasa.acida. |
| MA41795A (fr) | 2015-03-18 | 2018-01-23 | Sarepta Therapeutics Inc | Exclusion d'un exon induite par des composés antisens dans la myostatine |
| WO2017062835A2 (en) * | 2015-10-09 | 2017-04-13 | Sarepta Therapeutics, Inc. | Compositions and methods for treating duchenne muscular dystrophy and related disorders |
| AU2016334804B2 (en) | 2015-10-09 | 2022-03-31 | University Of Southampton | Modulation of gene expression and screening for deregulated protein expression |
| US10993995B2 (en) | 2015-12-07 | 2021-05-04 | 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 |
| WO2017106377A1 (en) | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome |
| US11060089B2 (en) * | 2016-04-18 | 2021-07-13 | Sarepta Therapeutics, Inc. | Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene |
| 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. |
| MX2019006882A (es) * | 2016-12-19 | 2019-08-16 | Sarepta Therapeutics Inc | Conjugados de oligomeros de omision de exon para distrofia muscular. |
| LT3673080T (lt) | 2017-08-25 | 2023-12-27 | Stoke Therapeutics, Inc. | Priešprasminiai oligomerai, skirti būklėms ir ligoms gydyti |
| NL2019517B1 (en) * | 2017-09-08 | 2019-03-19 | Univ Erasmus Med Ct Rotterdam | New therapy for Pompe disease |
| EP3672601B1 (en) * | 2017-09-25 | 2023-09-13 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers via fast-flow synthesis |
| BR112020022512A2 (pt) | 2018-05-04 | 2021-05-04 | Stoke Therapeutics, Inc. | métodos e composições para tratamento de doença de armazenamento de éster de colesteril |
| EP4150092A4 (en) | 2020-05-11 | 2024-11-06 | Stoke Therapeutics, Inc. | OPA1 ANTISENSE OLIGOMERS FOR THE TREATMENT OF DISEASES AND CONDITIONS |
| KR20210151593A (ko) * | 2020-06-05 | 2021-12-14 | 넥스올리고(주) | 신규한 몰포리노 올리고뉴클레오티드 유도체 |
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