JP2022136230A5 - - Google Patents
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- JP2022136230A5 JP2022136230A5 JP2022119527A JP2022119527A JP2022136230A5 JP 2022136230 A5 JP2022136230 A5 JP 2022136230A5 JP 2022119527 A JP2022119527 A JP 2022119527A JP 2022119527 A JP2022119527 A JP 2022119527A JP 2022136230 A5 JP2022136230 A5 JP 2022136230A5
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- 239000008194 pharmaceutical composition Substances 0.000 claims 15
- RWQNBRDOKXIBIV-UHFFFAOYSA-N thymine Chemical compound CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 claims 12
- 108700026244 Open Reading Frames Proteins 0.000 claims 10
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical compound O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 claims 10
- 108020004999 messenger RNA Proteins 0.000 claims 9
- 125000002947 alkylene group Chemical group 0.000 claims 6
- 102000005840 alpha-Galactosidase Human genes 0.000 claims 6
- 108010030291 alpha-Galactosidase Proteins 0.000 claims 6
- 229920001184 polypeptide Polymers 0.000 claims 6
- 102000004196 processed proteins & peptides Human genes 0.000 claims 6
- 108090000765 processed proteins & peptides Proteins 0.000 claims 6
- 125000000325 methylidene group Chemical group [H]C([H])=* 0.000 claims 5
- 229940113082 thymine Drugs 0.000 claims 5
- 229940035893 uracil Drugs 0.000 claims 5
- OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical compound NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 claims 4
- UYTPUPDQBNUYGX-UHFFFAOYSA-N guanine Chemical compound O=C1NC(N)=NC2=C1N=CN2 UYTPUPDQBNUYGX-UHFFFAOYSA-N 0.000 claims 4
- 150000002632 lipids Chemical class 0.000 claims 4
- 239000002773 nucleotide Substances 0.000 claims 4
- 125000003729 nucleotide group Chemical group 0.000 claims 4
- 125000000547 substituted alkyl group Chemical group 0.000 claims 4
- 108020003589 5' Untranslated Regions Proteins 0.000 claims 3
- 125000003342 alkenyl group Chemical group 0.000 claims 3
- 125000000217 alkyl group Chemical group 0.000 claims 3
- 125000000304 alkynyl group Chemical group 0.000 claims 3
- 125000000732 arylene group Chemical group 0.000 claims 3
- 125000005549 heteroarylene group Chemical group 0.000 claims 3
- 239000001257 hydrogen Substances 0.000 claims 3
- 229910052739 hydrogen Inorganic materials 0.000 claims 3
- 125000004435 hydrogen atom Chemical class [H]* 0.000 claims 3
- 150000003904 phospholipids Chemical class 0.000 claims 3
- 108020005345 3' Untranslated Regions Proteins 0.000 claims 2
- KELXHQACBIUYSE-UHFFFAOYSA-N 5-methoxy-1h-pyrimidine-2,4-dione Chemical compound COC1=CNC(=O)NC1=O KELXHQACBIUYSE-UHFFFAOYSA-N 0.000 claims 2
- 208000024720 Fabry Disease Diseases 0.000 claims 2
- 108700011259 MicroRNAs Proteins 0.000 claims 2
- -1 amino lipid Chemical class 0.000 claims 2
- 125000004429 atom Chemical group 0.000 claims 2
- 125000004452 carbocyclyl group Chemical group 0.000 claims 2
- 229940104302 cytosine Drugs 0.000 claims 2
- 229940124447 delivery agent Drugs 0.000 claims 2
- 125000001072 heteroaryl group Chemical group 0.000 claims 2
- 125000000623 heterocyclic group Chemical group 0.000 claims 2
- 239000002679 microRNA Substances 0.000 claims 2
- QJGQUHMNIGDVPM-UHFFFAOYSA-N nitrogen group Chemical group [N] QJGQUHMNIGDVPM-UHFFFAOYSA-N 0.000 claims 2
- 150000003839 salts Chemical class 0.000 claims 2
- 125000003107 substituted aryl group Chemical group 0.000 claims 2
- LRSASMSXMSNRBT-UHFFFAOYSA-N 5-methylcytosine Chemical compound CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 claims 1
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 1
- QVGXLLKOCUKJST-UHFFFAOYSA-N atomic oxygen Chemical group [O] QVGXLLKOCUKJST-UHFFFAOYSA-N 0.000 claims 1
- 125000002618 bicyclic heterocycle group Chemical group 0.000 claims 1
- 239000000203 mixture Substances 0.000 claims 1
- 239000002105 nanoparticle Substances 0.000 claims 1
- 150000007523 nucleic acids Chemical group 0.000 claims 1
- 230000004962 physiological condition Effects 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
Applications Claiming Priority (4)
| 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 |
| JP2018560595A 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 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018560595A Division 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 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022136230A JP2022136230A (ja) | 2022-09-15 |
| JP2022136230A5 true JP2022136230A5 (https=) | 2023-02-13 |
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 Before (1)
| 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 |
Country Status (6)
| Country | Link |
|---|---|
| US (5) | US10519455B2 (https=) |
| EP (2) | EP3896164A1 (https=) |
| JP (2) | JP7114485B2 (https=) |
| AU (1) | AU2017266932B2 (https=) |
| CA (1) | CA3024507A1 (https=) |
| WO (1) | WO2017201328A1 (https=) |
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-
2017
- 2017-05-18 WO PCT/US2017/033398 patent/WO2017201328A1/en not_active Ceased
- 2017-05-18 EP EP21153545.5A patent/EP3896164A1/en not_active Withdrawn
- 2017-05-18 AU AU2017266932A patent/AU2017266932B2/en not_active Ceased
- 2017-05-18 JP JP2018560595A patent/JP7114485B2/ja active Active
- 2017-05-18 CA CA3024507A patent/CA3024507A1/en active Pending
- 2017-05-18 EP EP17728702.6A patent/EP3458590B9/en active Active
-
2018
- 2018-08-23 US US16/110,309 patent/US10519455B2/en active Active
- 2018-08-23 US US16/110,788 patent/US20190000932A1/en not_active Abandoned
- 2018-08-23 US US16/110,833 patent/US10494636B2/en active Active
-
2019
- 2019-09-13 US US16/570,351 patent/US11649461B2/en active Active
-
2022
- 2022-07-27 JP JP2022119527A patent/JP2022136230A/ja active Pending
-
2023
- 2023-04-06 US US18/296,596 patent/US20230323371A1/en not_active Abandoned
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