JP2023531511A - 半減期が延長されたｍＲＮＡ治療薬を含むＬＮＰ組成物 - Google Patents
半減期が延長されたｍＲＮＡ治療薬を含むＬＮＰ組成物 Download PDFInfo
- Publication number
- JP2023531511A JP2023531511A JP2022579691A JP2022579691A JP2023531511A JP 2023531511 A JP2023531511 A JP 2023531511A JP 2022579691 A JP2022579691 A JP 2022579691A JP 2022579691 A JP2022579691 A JP 2022579691A JP 2023531511 A JP2023531511 A JP 2023531511A
- Authority
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- Japan
- Prior art keywords
- seq
- polynucleotide
- sequence
- utr
- fragment
- 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
Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
- A61K9/16—Agglomerates; Granulates; Microbeadlets ; Microspheres; Pellets; Solid products obtained by spray drying, spray freeze drying, spray congealing,(multiple) emulsion solvent evaporation or extraction
- A61K9/1605—Excipients; Inactive ingredients
- A61K9/1617—Organic compounds, e.g. phospholipids, fats
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/67—General methods for enhancing the expression
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Immunology (AREA)
- Nanotechnology (AREA)
- Optics & Photonics (AREA)
- Dispersion Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Medicinal Preparation (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063042822P | 2020-06-23 | 2020-06-23 | |
| US63/042,822 | 2020-06-23 | ||
| US202163165094P | 2021-03-23 | 2021-03-23 | |
| US63/165,094 | 2021-03-23 | ||
| US202163165469P | 2021-03-24 | 2021-03-24 | |
| US63/165,469 | 2021-03-24 | ||
| PCT/US2021/038780 WO2021262909A2 (en) | 2020-06-23 | 2021-06-23 | Lnp compositions comprising mrna therapeutics with extended half-life |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023531511A true JP2023531511A (ja) | 2023-07-24 |
| JP2023531511A5 JP2023531511A5 (https=) | 2024-07-01 |
Family
ID=77155849
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022579691A Pending JP2023531511A (ja) | 2020-06-23 | 2021-06-23 | 半減期が延長されたｍＲＮＡ治療薬を含むＬＮＰ組成物 |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20230242908A1 (https=) |
| EP (1) | EP4168556A2 (https=) |
| JP (1) | JP2023531511A (https=) |
| KR (1) | KR20230042005A (https=) |
| CN (1) | CN116194151A (https=) |
| AU (1) | AU2021297248A1 (https=) |
| BR (1) | BR112022025991A2 (https=) |
| CA (1) | CA3187261A1 (https=) |
| IL (1) | IL299196A (https=) |
| MX (1) | MX2022016550A (https=) |
| WO (1) | WO2021262909A2 (https=) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20250313830A1 (en) * | 2022-03-25 | 2025-10-09 | Modernatx, Inc. | Messenger ribonucleic acids with extended half-life |
| US20250221931A1 (en) * | 2022-03-25 | 2025-07-10 | Modernatx, Inc. | Polynucleotides encoding fanconi anemia, complementation group proteins for the treatment of fanconi anemia |
| WO2023196399A1 (en) * | 2022-04-06 | 2023-10-12 | Modernatx, Inc. | Lipid nanoparticles and polynucleotides encoding argininosuccinate lyase for the treatment of argininosuccinic aciduria |
| WO2023215498A2 (en) * | 2022-05-05 | 2023-11-09 | Modernatx, Inc. | Compositions and methods for cd28 antagonism |
| KR20250047488A (ko) * | 2023-09-27 | 2025-04-04 | 한미약품 주식회사 | 변형된 폴리아데닐 서열을 포함하는 핵산 분자 |
| KR20250047490A (ko) * | 2023-09-27 | 2025-04-04 | 한미약품 주식회사 | 변형된 폴리아데닐 서열 및 ox40l 단백질 코딩서열을 포함하는 핵산 분자 |
| KR20250047489A (ko) * | 2023-09-27 | 2025-04-04 | 한미약품 주식회사 | 변형된 폴리아데닐 서열 및 p53 단백질 코딩서열을 포함하는 핵산 분자 |
| CN117603029B (zh) * | 2023-11-21 | 2025-12-19 | 江苏禾本生化有限公司 | 一种戊菌唑关键中间体的合成方法 |
| WO2025255199A1 (en) * | 2024-06-05 | 2025-12-11 | Modernatx, Inc. | Argininosuccinate synthase 1 and argininosuccinate lyase polypeptides and polynucleotides and uses thereof |
| WO2026085234A1 (en) * | 2024-10-18 | 2026-04-23 | Modernatx, Inc. | 3' untranslated region sequences for extending messenger ribonucleic acids half-life |
Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2020056239A1 (en) * | 2018-09-14 | 2020-03-19 | Modernatx, Inc. | Polynucleotides encoding uridine diphosphate glycosyltransferase 1 family, polypeptide a1 for the treatment of crigler-najjar syndrome |
Family Cites Families (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB8613481D0 (en) | 1986-06-04 | 1986-07-09 | Diatech Ltd | Translation of mrna |
| GB2284210B (en) | 1993-11-26 | 1997-11-12 | British Tech Group | Translational enhancer DNA from a kinesin gene |
| US5824497A (en) | 1995-02-10 | 1998-10-20 | Mcmaster University | High efficiency translation of mRNA molecules |
| EP2447370B1 (en) | 2001-09-28 | 2018-07-18 | Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. | MicroRNA molecules |
| US20050222064A1 (en) | 2002-02-20 | 2005-10-06 | Sirna Therapeutics, Inc. | Polycationic compositions for cellular delivery of polynucleotides |
| US7683036B2 (en) | 2003-07-31 | 2010-03-23 | Regulus Therapeutics Inc. | Oligomeric compounds and compositions for use in modulation of small non-coding RNAs |
| DE102005046490A1 (de) | 2005-09-28 | 2007-03-29 | Johannes-Gutenberg-Universität Mainz | Modifikationen von RNA, die zu einer erhöhten Transkriptstabilität und Translationseffizienz führen |
| CA2715078C (en) | 2007-09-26 | 2019-07-23 | Intrexon Corporation | Synthetic 5'utrs, expression vectors, and methods for increasing transgene expression |
| EP2288336B8 (en) | 2008-04-25 | 2017-03-22 | Northwestern University | Nanostructures suitable for sequestering cholesterol |
| PL215513B1 (pl) | 2008-06-06 | 2013-12-31 | Univ Warszawski | Nowe boranofosforanowe analogi dinukleotydów, ich zastosowanie, czasteczka RNA, sposób otrzymywania RNA oraz sposób otrzymywania peptydów lub bialka |
| KR101766408B1 (ko) | 2009-06-10 | 2017-08-10 | 알닐람 파마슈티칼스 인코포레이티드 | 향상된 지질 조성물 |
| CA2824526C (en) | 2011-01-11 | 2020-07-07 | Alnylam Pharmaceuticals, Inc. | Pegylated lipids and their use for drug delivery |
| AU2013374345A1 (en) | 2013-01-17 | 2015-08-06 | Moderna Therapeutics, Inc. | Signal-sensor polynucleotides for the alteration of cellular phenotypes |
| US20160022840A1 (en) | 2013-03-09 | 2016-01-28 | Moderna Therapeutics, Inc. | Heterologous untranslated regions for mrna |
| US10821175B2 (en) | 2014-02-25 | 2020-11-03 | Merck Sharp & Dohme Corp. | Lipid nanoparticle vaccine adjuvants and antigen delivery systems |
| WO2017066797A1 (en) | 2015-10-16 | 2017-04-20 | Modernatx, Inc. | Trinucleotide mrna cap analogs |
| JP7194594B2 (ja) | 2016-05-18 | 2022-12-22 | モデルナティエックス インコーポレイテッド | 免疫調節ポリペプチドをコードするｍＲＮＡの組み合わせ及びその使用 |
| MX2021003015A (es) * | 2018-09-13 | 2021-07-15 | Modernatx Inc | Polinucleotidos que codifican glucosa-6-fosfatasa para el tratamiento de la glucogenosis. |
| AU2021205337A1 (en) * | 2020-01-10 | 2022-07-21 | Modernatx, Inc. | Methods of making tolerogenic dendritic cells |
| EP4096720A2 (en) * | 2020-01-30 | 2022-12-07 | ModernaTX, Inc. | Mrnas encoding metabolic reprogramming polypeptides and uses thereof |
| CN118369111A (zh) * | 2021-12-08 | 2024-07-19 | 摩登纳特斯有限公司 | 单纯疱疹病毒mRNA疫苗 |
-
2021
- 2021-06-23 EP EP21748991.3A patent/EP4168556A2/en active Pending
- 2021-06-23 IL IL299196A patent/IL299196A/en unknown
- 2021-06-23 KR KR1020237000782A patent/KR20230042005A/ko not_active Withdrawn
- 2021-06-23 JP JP2022579691A patent/JP2023531511A/ja active Pending
- 2021-06-23 CA CA3187261A patent/CA3187261A1/en active Pending
- 2021-06-23 AU AU2021297248A patent/AU2021297248A1/en not_active Abandoned
- 2021-06-23 US US18/012,094 patent/US20230242908A1/en active Pending
- 2021-06-23 MX MX2022016550A patent/MX2022016550A/es unknown
- 2021-06-23 CN CN202180051902.6A patent/CN116194151A/zh active Pending
- 2021-06-23 WO PCT/US2021/038780 patent/WO2021262909A2/en not_active Ceased
- 2021-06-23 BR BR112022025991A patent/BR112022025991A2/pt not_active Application Discontinuation
Patent Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2020056239A1 (en) * | 2018-09-14 | 2020-03-19 | Modernatx, Inc. | Polynucleotides encoding uridine diphosphate glycosyltransferase 1 family, polypeptide a1 for the treatment of crigler-najjar syndrome |
Non-Patent Citations (1)
| Title |
|---|
| "Mus musculus H1.5 linker histone, cluster member (H1f5), mRNA,", NCBI REFERENCE SEQUENCE [ONLINE], JPN6025024357, 3 May 2020 (2020-05-03), ISSN: 0005802030 * |
Also Published As
| Publication number | Publication date |
|---|---|
| US20230242908A1 (en) | 2023-08-03 |
| WO2021262909A2 (en) | 2021-12-30 |
| WO2021262909A3 (en) | 2022-04-21 |
| AU2021297248A1 (en) | 2023-02-02 |
| CA3187261A1 (en) | 2021-12-30 |
| CN116194151A (zh) | 2023-05-30 |
| IL299196A (en) | 2023-02-01 |
| EP4168556A2 (en) | 2023-04-26 |
| MX2022016550A (es) | 2023-04-10 |
| BR112022025991A2 (pt) | 2023-03-14 |
| KR20230042005A (ko) | 2023-03-27 |
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