CA3187261A1 - Lnp compositions comprising mrna therapeutics with extended half-life - Google Patents
Lnp compositions comprising mrna therapeutics with extended half-lifeInfo
- Publication number
- CA3187261A1 CA3187261A1 CA3187261A CA3187261A CA3187261A1 CA 3187261 A1 CA3187261 A1 CA 3187261A1 CA 3187261 A CA3187261 A CA 3187261A CA 3187261 A CA3187261 A CA 3187261A CA 3187261 A1 CA3187261 A1 CA 3187261A1
- Authority
- CA
- Canada
- 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 (1)
| Publication Number | Publication Date |
|---|---|
| CA3187261A1 true CA3187261A1 (en) | 2021-12-30 |
Family
ID=77155849
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3187261A Pending CA3187261A1 (en) | 2020-06-23 | 2021-06-23 | Lnp compositions comprising mrna therapeutics with extended half-life |
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 |
Family Cites Families (22)
| 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. |
| JP2022500444A (ja) * | 2018-09-14 | 2022-01-04 | モダーナティエックス・インコーポレイテッドModernaTX, Inc. | クリグラー−ナジャー症候群の治療のためのウリジン二リン酸グリコシルトランスフェラーゼ１ファミリー、ポリペプチドａ１をコードするポリヌクレオチド |
| 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
Also Published As
| Publication number | Publication date |
|---|---|
| US20230242908A1 (en) | 2023-08-03 |
| WO2021262909A2 (en) | 2021-12-30 |
| WO2021262909A3 (en) | 2022-04-21 |
| JP2023531511A (ja) | 2023-07-24 |
| AU2021297248A1 (en) | 2023-02-02 |
| 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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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| W00 | Other event occurred |
Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT Effective date: 20251210 |
|
| W00 | Other event occurred |
Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT Effective date: 20251216 |
|
| W00 | Other event occurred |
Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT Effective date: 20260206 |
|
| U13 | Renewal or maintenance fee not paid |
Free format text: ST27 STATUS EVENT CODE: N-1-6-U10-U13-U300 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: DEEMED ABANDONED - FAILURE TO RESPOND TO MAINTENANCE FEE NOTICE Effective date: 20260210 |
|
| U13 | Renewal or maintenance fee not paid |
Free format text: ST27 STATUS EVENT CODE: N-6-6-U10-U13-U300 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: DEEMED ABANDONED - FAILURE TO RESPOND TO MAINTENANCE FEE NOTICE Effective date: 20260216 |