JP2019516719A5 - - Google Patents
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- JP2019516719A5 JP2019516719A5 JP2018560595A JP2018560595A JP2019516719A5 JP 2019516719 A5 JP2019516719 A5 JP 2019516719A5 JP 2018560595 A JP2018560595 A JP 2018560595A JP 2018560595 A JP2018560595 A JP 2018560595A JP 2019516719 A5 JP2019516719 A5 JP 2019516719A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmaceutical composition
- composition according
- group
- guanosine
- mrna
- Prior art date
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- 239000008194 pharmaceutical composition Substances 0.000 claims 16
- 150000001875 compounds Chemical class 0.000 claims 6
- 150000002632 lipids Chemical class 0.000 claims 6
- 108020004999 messenger RNA Proteins 0.000 claims 6
- 102000005840 alpha-Galactosidase Human genes 0.000 claims 4
- 108010030291 alpha-Galactosidase Proteins 0.000 claims 4
- 229940124447 delivery agent Drugs 0.000 claims 4
- 239000002105 nanoparticle Substances 0.000 claims 4
- RWQNBRDOKXIBIV-UHFFFAOYSA-N thymine Chemical compound CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 claims 4
- 108700026244 Open Reading Frames Proteins 0.000 claims 3
- 229940029575 guanosine Drugs 0.000 claims 3
- 150000003839 salts Chemical class 0.000 claims 3
- 108020005345 3' Untranslated Regions Proteins 0.000 claims 2
- 108020003589 5' Untranslated Regions Proteins 0.000 claims 2
- MIKUYHXYGGJMLM-GIMIYPNGSA-N Crotonoside Natural products C1=NC2=C(N)NC(=O)N=C2N1[C@H]1O[C@@H](CO)[C@H](O)[C@@H]1O MIKUYHXYGGJMLM-GIMIYPNGSA-N 0.000 claims 2
- NYHBQMYGNKIUIF-UHFFFAOYSA-N D-guanosine Natural products C1=2NC(N)=NC(=O)C=2N=CN1C1OC(CO)C(O)C1O NYHBQMYGNKIUIF-UHFFFAOYSA-N 0.000 claims 2
- 208000024720 Fabry Disease Diseases 0.000 claims 2
- NYHBQMYGNKIUIF-UUOKFMHZSA-N Guanosine Natural products C1=NC=2C(=O)NC(N)=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O NYHBQMYGNKIUIF-UUOKFMHZSA-N 0.000 claims 2
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical compound O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 claims 2
- LNUFLCYMSVYYNW-ZPJMAFJPSA-N [(2r,3r,4s,5r,6r)-2-[(2r,3r,4s,5r,6r)-6-[(2r,3r,4s,5r,6r)-6-[(2r,3r,4s,5r,6r)-6-[[(3s,5s,8r,9s,10s,13r,14s,17r)-10,13-dimethyl-17-[(2r)-6-methylheptan-2-yl]-2,3,4,5,6,7,8,9,11,12,14,15,16,17-tetradecahydro-1h-cyclopenta[a]phenanthren-3-yl]oxy]-4,5-disulfo Chemical compound O([C@@H]1[C@@H](COS(O)(=O)=O)O[C@@H]([C@@H]([C@H]1OS(O)(=O)=O)OS(O)(=O)=O)O[C@@H]1[C@@H](COS(O)(=O)=O)O[C@@H]([C@@H]([C@H]1OS(O)(=O)=O)OS(O)(=O)=O)O[C@@H]1[C@@H](COS(O)(=O)=O)O[C@H]([C@@H]([C@H]1OS(O)(=O)=O)OS(O)(=O)=O)O[C@@H]1C[C@@H]2CC[C@H]3[C@@H]4CC[C@@H]([C@]4(CC[C@@H]3[C@@]2(C)CC1)C)[C@H](C)CCCC(C)C)[C@H]1O[C@H](COS(O)(=O)=O)[C@@H](OS(O)(=O)=O)[C@H](OS(O)(=O)=O)[C@H]1OS(O)(=O)=O LNUFLCYMSVYYNW-ZPJMAFJPSA-N 0.000 claims 2
- 125000000217 alkyl group Chemical group 0.000 claims 2
- HVYWMOMLDIMFJA-DPAQBDIFSA-N cholesterol Chemical compound C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@H](C)CCCC(C)C)[C@@]1(C)CC2 HVYWMOMLDIMFJA-DPAQBDIFSA-N 0.000 claims 2
- 125000001072 heteroaryl group Chemical group 0.000 claims 2
- 238000000034 method Methods 0.000 claims 2
- 150000007523 nucleic acids Chemical group 0.000 claims 2
- 229920001184 polypeptide Polymers 0.000 claims 2
- 102000004196 processed proteins & peptides Human genes 0.000 claims 2
- 108090000765 processed proteins & peptides Proteins 0.000 claims 2
- NRJAVPSFFCBXDT-HUESYALOSA-N 1,2-distearoyl-sn-glycero-3-phosphocholine Chemical compound CCCCCCCCCCCCCCCCCC(=O)OC[C@H](COP([O-])(=O)OCC[N+](C)(C)C)OC(=O)CCCCCCCCCCCCCCCCC NRJAVPSFFCBXDT-HUESYALOSA-N 0.000 claims 1
- HWPZZUQOWRWFDB-UHFFFAOYSA-N 1-methylcytosine Chemical compound CN1C=CC(N)=NC1=O HWPZZUQOWRWFDB-UHFFFAOYSA-N 0.000 claims 1
- RGICCULPCWNRAB-UHFFFAOYSA-N 2-[2-(2-hexoxyethoxy)ethoxy]ethanol Chemical compound CCCCCCOCCOCCOCCO RGICCULPCWNRAB-UHFFFAOYSA-N 0.000 claims 1
- GOJUJUVQIVIZAV-UHFFFAOYSA-N 2-amino-4,6-dichloropyrimidine-5-carbaldehyde Chemical group NC1=NC(Cl)=C(C=O)C(Cl)=N1 GOJUJUVQIVIZAV-UHFFFAOYSA-N 0.000 claims 1
- JRYMOPZHXMVHTA-DAGMQNCNSA-N 2-amino-7-[(2r,3r,4s,5r)-3,4-dihydroxy-5-(hydroxymethyl)oxolan-2-yl]-1h-pyrrolo[2,3-d]pyrimidin-4-one Chemical compound C1=CC=2C(=O)NC(N)=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O JRYMOPZHXMVHTA-DAGMQNCNSA-N 0.000 claims 1
- BGTXMQUSDNMLDW-AEHJODJJSA-N 2-amino-9-[(2r,3s,4r,5r)-3-fluoro-3,4-dihydroxy-5-(hydroxymethyl)oxolan-2-yl]-3h-purin-6-one Chemical compound C1=2NC(N)=NC(=O)C=2N=CN1[C@@H]1O[C@H](CO)[C@@H](O)[C@]1(O)F BGTXMQUSDNMLDW-AEHJODJJSA-N 0.000 claims 1
- KELXHQACBIUYSE-UHFFFAOYSA-N 5-methoxy-1h-pyrimidine-2,4-dione Chemical compound COC1=CNC(=O)NC1=O KELXHQACBIUYSE-UHFFFAOYSA-N 0.000 claims 1
- HCAJQHYUCKICQH-VPENINKCSA-N 8-Oxo-7,8-dihydro-2'-deoxyguanosine Chemical compound C1=2NC(N)=NC(=O)C=2NC(=O)N1[C@H]1C[C@H](O)[C@@H](CO)O1 HCAJQHYUCKICQH-VPENINKCSA-N 0.000 claims 1
- 241000180579 Arca Species 0.000 claims 1
- 101150077194 CAP1 gene Proteins 0.000 claims 1
- 101150014715 CAP2 gene Proteins 0.000 claims 1
- 229930010555 Inosine Natural products 0.000 claims 1
- 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 1
- 101100245221 Mus musculus Prss8 gene Proteins 0.000 claims 1
- 101100260872 Mus musculus Tmprss4 gene Proteins 0.000 claims 1
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 1
- 125000003342 alkenyl group Chemical group 0.000 claims 1
- 125000003118 aryl group Chemical group 0.000 claims 1
- 235000012000 cholesterol Nutrition 0.000 claims 1
- 125000000592 heterocycloalkyl group Chemical group 0.000 claims 1
- 229960003786 inosine Drugs 0.000 claims 1
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 claims 1
- 150000003904 phospholipids Chemical class 0.000 claims 1
- 230000002265 prevention Effects 0.000 claims 1
- 235000000346 sugar Nutrition 0.000 claims 1
- 150000008163 sugars Chemical class 0.000 claims 1
- 208000024891 symptom Diseases 0.000 claims 1
- ZEMGGZBWXRYJHK-UHFFFAOYSA-N thiouracil Chemical compound O=C1C=CNC(=S)N1 ZEMGGZBWXRYJHK-UHFFFAOYSA-N 0.000 claims 1
- 229950000329 thiouracil Drugs 0.000 claims 1
- 229940113082 thymine Drugs 0.000 claims 1
- 229940035893 uracil Drugs 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022119527A JP2022136230A (ja) | 2016-05-18 | 2022-07-27 | ファブリー病の治療のためのα－ガラクトシダーゼＡをコードするポリヌクレオチド |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662338354P | 2016-05-18 | 2016-05-18 | |
| US62/338,354 | 2016-05-18 | ||
| PCT/US2017/033398 WO2017201328A1 (en) | 2016-05-18 | 2017-05-18 | POLYNUCLEOTIDES ENCODING α-GALACTOSIDASE A FOR THE TREATMENT OF FABRY DISEASE |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022119527A Division JP2022136230A (ja) | 2016-05-18 | 2022-07-27 | ファブリー病の治療のためのα－ガラクトシダーゼＡをコードするポリヌクレオチド |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019516719A JP2019516719A (ja) | 2019-06-20 |
| JP2019516719A5 true JP2019516719A5 (enExample) | 2020-06-25 |
| JP7114485B2 JP7114485B2 (ja) | 2022-08-08 |
Family
ID=59021568
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018560595A Active JP7114485B2 (ja) | 2016-05-18 | 2017-05-18 | ファブリー病の治療のためのα－ガラクトシダーゼＡをコードするポリヌクレオチド |
| JP2022119527A Pending JP2022136230A (ja) | 2016-05-18 | 2022-07-27 | ファブリー病の治療のためのα－ガラクトシダーゼＡをコードするポリヌクレオチド |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022119527A Pending JP2022136230A (ja) | 2016-05-18 | 2022-07-27 | ファブリー病の治療のためのα－ガラクトシダーゼＡをコードするポリヌクレオチド |
Country Status (6)
| Country | Link |
|---|---|
| US (5) | US20190000932A1 (enExample) |
| EP (2) | EP3458590B9 (enExample) |
| JP (2) | JP7114485B2 (enExample) |
| AU (1) | AU2017266932B2 (enExample) |
| CA (1) | CA3024507A1 (enExample) |
| WO (1) | WO2017201328A1 (enExample) |
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2018
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2019
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2022
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2023
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