JP2020522244A5 - - Google Patents
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- Publication number
- JP2020522244A5 JP2020522244A5 JP2019565348A JP2019565348A JP2020522244A5 JP 2020522244 A5 JP2020522244 A5 JP 2020522244A5 JP 2019565348 A JP2019565348 A JP 2019565348A JP 2019565348 A JP2019565348 A JP 2019565348A JP 2020522244 A5 JP2020522244 A5 JP 2020522244A5
- Authority
- JP
- Japan
- Prior art keywords
- polynucleotide according
- polynucleotide
- utr
- seq
- composition
- 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.)
- Granted
Links
- 108091033319 polynucleotide Proteins 0.000 claims 16
- 239000002157 polynucleotide Substances 0.000 claims 16
- 102000040430 polynucleotide Human genes 0.000 claims 16
- DRTQHJPVMGBUCF-XVFCMESISA-N Uridine Chemical group O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-XVFCMESISA-N 0.000 claims 6
- 239000003795 chemical substances by application Substances 0.000 claims 4
- 108020005345 3' Untranslated Regions Proteins 0.000 claims 3
- 108020003589 5' Untranslated Regions Proteins 0.000 claims 3
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 3
- 239000002105 nanoparticle Substances 0.000 claims 3
- 101000893559 Homo sapiens Amylo-alpha-1,6-glucosidase Proteins 0.000 claims 2
- 241000723792 Tobacco etch virus Species 0.000 claims 2
- DRTQHJPVMGBUCF-PSQAKQOGSA-N beta-L-uridine Natural products O[C@H]1[C@@H](O)[C@H](CO)O[C@@H]1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-PSQAKQOGSA-N 0.000 claims 2
- 201000010099 disease Diseases 0.000 claims 2
- DRTQHJPVMGBUCF-UHFFFAOYSA-N uracil arabinoside Natural products OC1C(O)C(CO)OC1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-UHFFFAOYSA-N 0.000 claims 2
- 229940045145 uridine Drugs 0.000 claims 2
- 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 1
- 102100040894 Amylo-alpha-1,6-glucosidase Human genes 0.000 claims 1
- 208000032008 Glycogen storage disease due to glycogen debranching enzyme deficiency Diseases 0.000 claims 1
- 206010053250 Glycogen storage disease type III Diseases 0.000 claims 1
- 108091026898 Leader sequence (mRNA) Proteins 0.000 claims 1
- 108091036066 Three prime untranslated region Proteins 0.000 claims 1
- 241000269368 Xenopus laevis Species 0.000 claims 1
- XSWXHOQMWXTMEH-QOCHGBHMSA-N [(Z)-non-2-enyl] 4-[2-(dimethylamino)ethylsulfanylcarbonyl-(4-oxo-4-pentadecan-8-yloxybutyl)amino]butanoate Chemical compound CCCCCCCC(CCCCCCC)OC(=O)CCCN(CCCC(=O)OC\C=C/CCCCCC)C(=O)SCCN(C)C XSWXHOQMWXTMEH-QOCHGBHMSA-N 0.000 claims 1
- RGAIHNZNCGOCLA-ZDSKVHJSSA-N [(Z)-non-2-enyl] 8-[2-(dimethylamino)ethylsulfanylcarbonyl-[8-[(Z)-non-2-enoxy]-8-oxooctyl]amino]octanoate Chemical compound CCCCCC\C=C/COC(=O)CCCCCCCN(CCCCCCCC(=O)OC\C=C/CCCCCC)C(=O)SCCN(C)C RGAIHNZNCGOCLA-ZDSKVHJSSA-N 0.000 claims 1
- 150000003838 adenosines Chemical class 0.000 claims 1
- 230000003247 decreasing effect Effects 0.000 claims 1
- 208000035475 disorder Diseases 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 230000000694 effects Effects 0.000 claims 1
- 201000004543 glycogen storage disease III Diseases 0.000 claims 1
- 239000002502 liposome Substances 0.000 claims 1
- 239000002773 nucleotide Substances 0.000 claims 1
- 125000003729 nucleotide group Chemical group 0.000 claims 1
- 230000000069 prophylactic effect Effects 0.000 claims 1
- 229940124597 therapeutic agent Drugs 0.000 claims 1
- 239000012096 transfection reagent Substances 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023038257A JP2023075248A (ja) | 2017-05-31 | 2023-03-13 | 糖原病ｉｉｉ型のための治療薬 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762513350P | 2017-05-31 | 2017-05-31 | |
| US62/513,350 | 2017-05-31 | ||
| PCT/US2018/035477 WO2018222926A1 (en) | 2017-05-31 | 2018-05-31 | Therapeutics for glycogen storage disease type iii |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023038257A Division JP2023075248A (ja) | 2017-05-31 | 2023-03-13 | 糖原病ｉｉｉ型のための治療薬 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020522244A JP2020522244A (ja) | 2020-07-30 |
| JP2020522244A5 true JP2020522244A5 (enExample) | 2021-07-26 |
| JP7284101B2 JP7284101B2 (ja) | 2023-05-30 |
Family
ID=64456114
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019565348A Active JP7284101B2 (ja) | 2017-05-31 | 2018-05-31 | 糖原病ｉｉｉ型のための治療薬 |
| JP2023038257A Pending JP2023075248A (ja) | 2017-05-31 | 2023-03-13 | 糖原病ｉｉｉ型のための治療薬 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023038257A Pending JP2023075248A (ja) | 2017-05-31 | 2023-03-13 | 糖原病ｉｉｉ型のための治療薬 |
Country Status (14)
| Country | Link |
|---|---|
| US (2) | US11377643B2 (enExample) |
| EP (1) | EP3630964B1 (enExample) |
| JP (2) | JP7284101B2 (enExample) |
| KR (1) | KR102636537B1 (enExample) |
| CN (1) | CN110719954B (enExample) |
| AR (1) | AR112706A1 (enExample) |
| AU (1) | AU2018278315B2 (enExample) |
| BR (1) | BR112019025224A2 (enExample) |
| CA (1) | CA3063907A1 (enExample) |
| CO (1) | CO2019013332A2 (enExample) |
| ES (1) | ES3048960T3 (enExample) |
| MX (1) | MX2019014412A (enExample) |
| TW (1) | TWI794237B (enExample) |
| WO (1) | WO2018222926A1 (enExample) |
Families Citing this family (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PT2791160T (pt) | 2011-12-16 | 2022-07-04 | Modernatx Inc | Composições arnm modificado |
| EP3630964B1 (en) * | 2017-05-31 | 2025-08-13 | Ultragenyx Pharmaceutical Inc. | Therapeutics for glycogen storage disease type iii |
| ES2960692T3 (es) | 2018-12-06 | 2024-03-06 | Arcturus Therapeutics Inc | Composiciones y métodos para el tratamiento de la deficiencia de ornitina transcarbamilasa |
| CN110314407A (zh) * | 2019-08-01 | 2019-10-11 | 山东新希望六和集团有限公司 | 一种脂肪快速提取装置 |
| US20220347298A1 (en) | 2019-10-04 | 2022-11-03 | Ultragenyx Pharmaceutical Inc. | Methods for improved therapeutic use of recombinant aav |
| IT202000003371A1 (it) * | 2020-02-19 | 2021-08-19 | Enea Agenzia Naz Per Le Nuove Tecnologie Lenergia E Lo Sviluppo Economico Sostenibile | Composto per il trattamento di una glicogenosi |
| US12351834B2 (en) | 2020-03-03 | 2025-07-08 | Arcturus Therapeutics, Inc. | Compositions and methods for the treatment of ornithine transcarbamylase deficiency |
| US11759515B2 (en) | 2020-03-09 | 2023-09-19 | Arcturus Therapeutics, Inc. | Compositions and methods for inducing immune responses |
| WO2022023284A1 (en) | 2020-07-27 | 2022-02-03 | Anjarium Biosciences Ag | Compositions of dna molecules, methods of making therefor, and methods of use thereof |
| AU2021325945A1 (en) | 2020-08-14 | 2023-04-06 | Arcturus Therapeutics, Inc. | Method of lyophilizing lipid nanoparticles |
| CA3214538A1 (en) * | 2021-04-20 | 2022-10-27 | Joel DE BEER | Compositions of dna molecules encoding amylo-alpha-1, 6-glucosidase, 4-alpha-glucanotransferase, methods of making thereof, and methods of use thereof |
| WO2023131254A1 (zh) * | 2022-01-06 | 2023-07-13 | 上海吉量医药工程有限公司 | N1位修饰假尿嘧啶核苷及其在mRNA合成中的应用 |
| CN115960926A (zh) * | 2022-12-30 | 2023-04-14 | 南方科技大学 | 一种mRNA及含有mRNA的药物 |
| WO2025080939A1 (en) | 2023-10-13 | 2025-04-17 | Ultragenyx Pharmaceutical, Inc. | Compositions and methods for treating conditions associated with cartilage oligomeric matrix protein (comp) mutations |
| WO2025128853A2 (en) | 2023-12-13 | 2025-06-19 | Ultragenyx Pharmaceutical Inc. | Compositions and methods for treating conditions associated with ube3a overexpression |
Family Cites Families (45)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2457959C (en) | 2001-06-05 | 2014-10-28 | Curevac Gmbh | Pharmaceutical composition containing a stabilised mrna optimised for translation in its coding regions |
| WO2004039412A2 (en) | 2002-10-29 | 2004-05-13 | Engene, Inc. | Compositions for cancer treatment |
| TR201909609T4 (tr) | 2005-08-23 | 2019-07-22 | Univ Pennsylvania | Modifiye edilmiş nükleosidleri içeren rna ve kullanım yöntemleri. |
| JP2009544754A (ja) | 2006-07-28 | 2009-12-17 | アプライド バイオシステムズ， エルエルシー | ジヌクレオチドｍｒｎａキャップアナログ |
| WO2009058911A2 (en) | 2007-10-31 | 2009-05-07 | Applied Biosystems Inc. | Preparation and isolation of 5' capped mrna |
| ES2535419T3 (es) | 2007-12-27 | 2015-05-11 | Protiva Biotherapeutics Inc. | Silenciamiento de expresión de quinasa tipo polo usando ARN interferente |
| US20110117125A1 (en) | 2008-01-02 | 2011-05-19 | Tekmira Pharmaceuticals Corporation | Compositions and methods for the delivery of nucleic acids |
| ES2638448T3 (es) | 2008-04-15 | 2017-10-20 | Protiva Biotherapeutics Inc. | Novedosas formulaciones de lípidos para la administración de ácidos nucleicos |
| SI2318037T1 (sl) * | 2008-07-08 | 2015-07-31 | Duke University | Metoda za zdravljenje bolezni glikogenoze |
| WO2010037408A1 (en) | 2008-09-30 | 2010-04-08 | Curevac Gmbh | Composition comprising a complexed (m)rna and a naked mrna for providing or enhancing an immunostimulatory response in a mammal and uses thereof |
| CA2740000C (en) | 2008-10-09 | 2017-12-12 | Tekmira Pharmaceuticals Corporation | Improved amino lipids and methods for the delivery of nucleic acids |
| WO2010048536A2 (en) | 2008-10-23 | 2010-04-29 | Alnylam Pharmaceuticals, Inc. | Processes for preparing lipids |
| PL4241767T3 (pl) | 2008-11-10 | 2025-03-03 | Arbutus Biopharma Corporation | Nowe lipidy i kompozycje do dostarczania środków terapeutycznych |
| EP2391343B1 (en) | 2009-01-29 | 2017-03-01 | Arbutus Biopharma Corporation | Improved lipid formulation for the delivery of nucleic acids |
| KR20180094137A (ko) | 2009-05-05 | 2018-08-22 | 알닐람 파마슈티칼스 인코포레이티드 | 지질 조성물 |
| PL3431076T3 (pl) | 2009-06-10 | 2022-01-31 | Arbutus Biopharma Corporation | Ulepszona formulacja lipidowa |
| AU2010326132B9 (en) | 2009-12-01 | 2014-10-02 | Translate Bio, Inc. | Delivery of mRNA for the augmentation of proteins and enzymes in human genetic diseases |
| NZ605079A (en) | 2010-06-03 | 2015-08-28 | Alnylam Pharmaceuticals Inc | Biodegradable lipids for the delivery of active agents |
| WO2012000104A1 (en) | 2010-06-30 | 2012-01-05 | Protiva Biotherapeutics, Inc. | Non-liposomal systems for nucleic acid delivery |
| EP4043025A1 (en) * | 2011-06-08 | 2022-08-17 | Translate Bio, Inc. | Lipid nanoparticle compositions and methods for mrna delivery |
| RS62993B1 (sr) | 2011-10-03 | 2022-03-31 | Modernatx Inc | Modifikovani nukleozidi, nukleotidi, i nukleinske kiseline, i njihove upotrebe |
| LT3421601T (lt) | 2011-12-30 | 2020-01-10 | Cellscript, Llc | In vitro susintetintos ssrnr gamyba ir panaudojimas įvedimui į žinduolių ląsteles biologinio arba biocheminio poveikio sukėlimui |
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| JP6506749B2 (ja) | 2013-10-22 | 2019-04-24 | シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド | フェニルケトン尿症のためのｍＲＮＡ療法 |
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| EP3630964B1 (en) * | 2017-05-31 | 2025-08-13 | Ultragenyx Pharmaceutical Inc. | Therapeutics for glycogen storage disease type iii |
-
2018
- 2018-05-31 EP EP18810235.4A patent/EP3630964B1/en active Active
- 2018-05-31 US US16/617,431 patent/US11377643B2/en active Active
- 2018-05-31 AR ARP180101448A patent/AR112706A1/es unknown
- 2018-05-31 BR BR112019025224-0A patent/BR112019025224A2/pt unknown
- 2018-05-31 ES ES18810235T patent/ES3048960T3/es active Active
- 2018-05-31 CA CA3063907A patent/CA3063907A1/en active Pending
- 2018-05-31 KR KR1020197036416A patent/KR102636537B1/ko active Active
- 2018-05-31 MX MX2019014412A patent/MX2019014412A/es unknown
- 2018-05-31 AU AU2018278315A patent/AU2018278315B2/en active Active
- 2018-05-31 WO PCT/US2018/035477 patent/WO2018222926A1/en not_active Ceased
- 2018-05-31 CN CN201880036551.XA patent/CN110719954B/zh active Active
- 2018-05-31 JP JP2019565348A patent/JP7284101B2/ja active Active
- 2018-05-31 TW TW107118693A patent/TWI794237B/zh active
-
2019
- 2019-11-27 CO CONC2019/0013332A patent/CO2019013332A2/es unknown
-
2022
- 2022-05-04 US US17/736,798 patent/US20220340886A1/en active Pending
-
2023
- 2023-03-13 JP JP2023038257A patent/JP2023075248A/ja active Pending