JP2023075248A5 - - Google Patents
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- Publication number
- JP2023075248A5 JP2023075248A5 JP2023038257A JP2023038257A JP2023075248A5 JP 2023075248 A5 JP2023075248 A5 JP 2023075248A5 JP 2023038257 A JP2023038257 A JP 2023038257A JP 2023038257 A JP2023038257 A JP 2023038257A JP 2023075248 A5 JP2023075248 A5 JP 2023075248A5
- Authority
- JP
- Japan
- Prior art keywords
- lnp
- polynucleotide
- therapeutic agent
- utr
- lipid
- 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.)
- Pending
Links
Applications Claiming Priority (4)
| 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 |
| JP2019565348A JP7284101B2 (ja) | 2017-05-31 | 2018-05-31 | 糖原病ｉｉｉ型のための治療薬 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019565348A Division JP7284101B2 (ja) | 2017-05-31 | 2018-05-31 | 糖原病ｉｉｉ型のための治療薬 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023075248A JP2023075248A (ja) | 2023-05-30 |
| JP2023075248A5 true JP2023075248A5 (enExample) | 2023-09-13 |
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 Before (1)
| 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 |
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 (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3018046A1 (en) | 2011-12-16 | 2013-06-20 | Moderna Therapeutics, Inc. | Modified nucleoside, nucleotide, and nucleic acid compositions |
| US10383952B2 (en) | 2016-12-21 | 2019-08-20 | Arcturus Therapeutics, Inc. | Ionizable cationic lipid for RNA delivery |
| BR112019025224A2 (pt) * | 2017-05-31 | 2020-12-08 | Ultragenyx Pharmaceutical Inc. | Terapêutica para doença de armazenamento de glicogênio tipo iii |
| AU2019394996B2 (en) | 2018-12-06 | 2025-11-06 | Arcturus Therapeutics, Inc. | Compositions and methods for treating ornithine transcarbamylase deficiency |
| 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 |
| WO2021067598A1 (en) | 2019-10-04 | 2021-04-08 | 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 |
| AU2021236068A1 (en) * | 2020-03-09 | 2022-10-06 | Arcturus Therapeutics, Inc. | Compositions and methods for inducing immune responses |
| JP2023535632A (ja) | 2020-07-27 | 2023-08-18 | アンジャリウム バイオサイエンシズ エージー | Ｄｎａ分子の組成物、その作製方法、及びその使用方法 |
| EP4196128A4 (en) | 2020-08-14 | 2024-06-12 | Arcturus Therapeutics, Inc. | METHOD FOR FREEZE DILIZATION OF LIPID NANOPARTICLES |
| JP2024517427A (ja) * | 2021-04-20 | 2024-04-22 | アンジャリウム バイオサイエンシズ エージー | アミロ－α－１，６－グルコシダーゼ、４－α－グルカノトランスフェラーゼをコードするＤＮＡ分子の組成物、その作製の方法、及びその使用の方法 |
| CN118369335A (zh) * | 2022-01-06 | 2024-07-19 | 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 |
|---|---|---|---|---|
| ES2340499T3 (es) | 2001-06-05 | 2010-06-04 | Curevac Gmbh | Arnm de antigeno tumoral estabilizado con un contenido de g/c aumentado. |
| JP2006508939A (ja) | 2002-10-29 | 2006-03-16 | エンジーン， インコーポレイテッド | 癌処置のための組成物 |
| PL2578685T3 (pl) | 2005-08-23 | 2020-01-31 | The Trustees Of The University Of Pennsylvania | Rna zawierający zmodyfikowane nukleozydy i sposoby jego zastosowania |
| US8304529B2 (en) | 2006-07-28 | 2012-11-06 | Life Technologies Corporation | Dinucleotide MRNA cap analogs |
| 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 |
| WO2009086558A1 (en) | 2008-01-02 | 2009-07-09 | Tekmira Pharmaceuticals Corporation | Improved compositions and methods for the delivery of nucleic acids |
| CA2721333C (en) | 2008-04-15 | 2020-12-01 | Protiva Biotherapeutics, Inc. | Novel lipid formulations for nucleic acid delivery |
| ES2535461T3 (es) | 2008-07-08 | 2015-05-11 | Duke University | Método de tratamiento de la enfermedad por almacenamiento de glucógeno |
| 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 |
| CN111808084A (zh) | 2008-11-10 | 2020-10-23 | 阿布特斯生物制药公司 | 用于递送治疗剂的新型脂质和组合物 |
| EP3243504A1 (en) | 2009-01-29 | 2017-11-15 | Arbutus Biopharma Corporation | Improved lipid formulation |
| SG10201911942UA (en) | 2009-05-05 | 2020-02-27 | Muthiah Manoharan | Lipid compositions |
| KR101766408B1 (ko) | 2009-06-10 | 2017-08-10 | 알닐람 파마슈티칼스 인코포레이티드 | 향상된 지질 조성물 |
| EP3318248B1 (en) | 2009-12-01 | 2019-04-10 | Translate Bio, Inc. | Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases |
| KR20190039347A (ko) | 2010-06-03 | 2019-04-10 | 알닐람 파마슈티칼스 인코포레이티드 | 활성제의 전달을 위한 생분해성 지질 |
| US9006417B2 (en) | 2010-06-30 | 2015-04-14 | Protiva Biotherapeutics, Inc. | Non-liposomal systems for nucleic acid delivery |
| RU2013154295A (ru) * | 2011-06-08 | 2015-07-20 | Шир Хьюман Дженетик Терапис, Инк. | КОМПОЗИЦИИ ЛИПИДНЫХ НАНОЧАСТИЦ И СПОСОБЫ ДЛЯ ДОСТАВКИ мРНК |
| DE19216461T1 (de) | 2011-10-03 | 2021-10-07 | Modernatx, Inc. | Modifizierte nukleoside, nukleotide und nukleinsäuren und verwendungen davon |
| EP3144389B1 (en) | 2011-12-30 | 2018-05-09 | Cellscript, Llc | Making and using in vitro-synthesized ssrna for introducing into mammalian cells to induce a biological or biochemical effect |
| US9254311B2 (en) | 2012-04-02 | 2016-02-09 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of proteins |
| WO2013151665A2 (en) | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of proteins associated with human disease |
| WO2014093924A1 (en) | 2012-12-13 | 2014-06-19 | Moderna Therapeutics, Inc. | Modified nucleic acid molecules and uses thereof |
| EP4331620A3 (en) * | 2012-12-07 | 2024-12-04 | Translate Bio, Inc. | Lipidic nanoparticles for mrna delivery |
| WO2014130722A1 (en) * | 2013-02-20 | 2014-08-28 | Valerion Therapeutics, Llc | Methods and compositions for treatment of forbes-cori disease |
| US20160022840A1 (en) | 2013-03-09 | 2016-01-28 | Moderna Therapeutics, Inc. | Heterologous untranslated regions for mrna |
| EP3750903A1 (en) | 2013-03-14 | 2020-12-16 | Translate Bio, Inc. | Ribonucleic acids with 4'-thio-modified nucleotides and related methods |
| EP3052511A4 (en) | 2013-10-02 | 2017-05-31 | Moderna Therapeutics, Inc. | Polynucleotide molecules and uses thereof |
| 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 |
| AU2014348212C1 (en) | 2013-11-18 | 2018-11-29 | Arcturus Therapeutics, Inc. | Ionizable cationic lipid for RNA delivery |
| WO2015192092A1 (en) * | 2014-06-13 | 2015-12-17 | Valerion Therapeutics, Llc | Methods and compositions for treatment of glycogen storage diseases and glycogen metabolism disorders |
| AU2015338923B2 (en) * | 2014-11-02 | 2021-10-21 | Arcturus Therapeutics, Inc. | Messenger UNA molecules and uses thereof |
| WO2016077125A1 (en) | 2014-11-10 | 2016-05-19 | Moderna Therapeutics, Inc. | Alternative nucleic acid molecules containing reduced uracil content and uses thereof |
| WO2016077123A1 (en) | 2014-11-10 | 2016-05-19 | Moderna Therapeutics, Inc. | Multiparametric nucleic acid optimization |
| US9709490B2 (en) | 2014-12-08 | 2017-07-18 | Canon Kabushiki Kaisha | Refractive index distribution measuring method, refractive index distribution measuring apparatus, and optical element manufacturing method |
| WO2017054086A1 (en) * | 2015-10-01 | 2017-04-06 | Exerkine Corporation | Treatment of genetic myopathies using bioengineered exosomes |
| WO2017100551A1 (en) | 2015-12-09 | 2017-06-15 | Alexion Pharmaceuticals, Inc. | HETEROLOGOUS UTR SEQUENCES FOR ENHANCED mRNA EXPRESSION |
| IL308824A (en) | 2016-08-17 | 2024-01-01 | Factor Bioscience Inc | Nucleic acid products and methods of administration thereof |
| CA3040337A1 (en) | 2016-10-26 | 2018-05-03 | Curevac Ag | Lipid nanoparticle mrna vaccines |
| US10526284B2 (en) | 2016-12-21 | 2020-01-07 | Arcturus Therapeutics, Inc. | Ionizable cationic lipid for RNA delivery |
| US10383952B2 (en) | 2016-12-21 | 2019-08-20 | Arcturus Therapeutics, Inc. | Ionizable cationic lipid for RNA delivery |
| US10227302B2 (en) | 2017-02-09 | 2019-03-12 | Arcturus Therapeutics, Inc. | Ionizable cationic lipid for RNA delivery |
| BR112019025224A2 (pt) * | 2017-05-31 | 2020-12-08 | Ultragenyx Pharmaceutical Inc. | Terapêutica para doença de armazenamento de glicogênio tipo iii |
-
2018
- 2018-05-31 BR BR112019025224-0A patent/BR112019025224A2/pt unknown
- 2018-05-31 EP EP18810235.4A patent/EP3630964B1/en active Active
- 2018-05-31 CN CN201880036551.XA patent/CN110719954B/zh active Active
- 2018-05-31 AR ARP180101448A patent/AR112706A1/es unknown
- 2018-05-31 MX MX2019014412A patent/MX2019014412A/es unknown
- 2018-05-31 KR KR1020197036416A patent/KR102636537B1/ko active Active
- 2018-05-31 JP JP2019565348A patent/JP7284101B2/ja active Active
- 2018-05-31 TW TW107118693A patent/TWI794237B/zh active
- 2018-05-31 US US16/617,431 patent/US11377643B2/en active Active
- 2018-05-31 CA CA3063907A patent/CA3063907A1/en active Pending
- 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 ES ES18810235T patent/ES3048960T3/es active 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
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