BR112023005207A2 - Lipídios catiônicos à base de piperazina - Google Patents
Lipídios catiônicos à base de piperazinaInfo
- Publication number
- BR112023005207A2 BR112023005207A2 BR112023005207A BR112023005207A BR112023005207A2 BR 112023005207 A2 BR112023005207 A2 BR 112023005207A2 BR 112023005207 A BR112023005207 A BR 112023005207A BR 112023005207 A BR112023005207 A BR 112023005207A BR 112023005207 A2 BR112023005207 A2 BR 112023005207A2
- Authority
- BR
- Brazil
- Prior art keywords
- piperazine
- based cation
- cation lipids
- useful
- lipids
- Prior art date
Links
- -1 CATION LIPIDS Chemical class 0.000 title abstract 3
- GLUUGHFHXGJENI-UHFFFAOYSA-N Piperazine Chemical compound C1CNCCN1 GLUUGHFHXGJENI-UHFFFAOYSA-N 0.000 abstract 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 2
- 102000004169 proteins and genes Human genes 0.000 abstract 2
- 108090000623 proteins and genes Proteins 0.000 abstract 2
- 150000001875 compounds Chemical class 0.000 abstract 1
- 230000007812 deficiency Effects 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000035475 disorder Diseases 0.000 abstract 1
- 108020004999 messenger RNA Proteins 0.000 abstract 1
- 150000003839 salts Chemical class 0.000 abstract 1
Classifications
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D241/00—Heterocyclic compounds containing 1,4-diazine or hydrogenated 1,4-diazine rings
- C07D241/02—Heterocyclic compounds containing 1,4-diazine or hydrogenated 1,4-diazine rings not condensed with other rings
- C07D241/04—Heterocyclic compounds containing 1,4-diazine or hydrogenated 1,4-diazine rings not condensed with other rings having no double bonds between ring members or between ring members and non-ring members
-
- 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
- A61K9/10—Dispersions; Emulsions
- A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
- A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
- A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers
-
- 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
- A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
- A61K48/0033—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
-
- 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
- A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
- A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
- A61K9/51—Nanocapsules; Nanoparticles
- A61K9/5107—Excipients; Inactive ingredients
- A61K9/5123—Organic compounds, e.g. fats, sugars
-
- 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
- A61K48/0091—Purification or manufacturing processes for gene therapy compositions
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Biophysics (AREA)
- Zoology (AREA)
- Physics & Mathematics (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Dispersion Chemistry (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Nanotechnology (AREA)
- Optics & Photonics (AREA)
- Manufacturing & Machinery (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
- Detergent Compositions (AREA)
- Agricultural Chemicals And Associated Chemicals (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063082101P | 2020-09-23 | 2020-09-23 | |
| PCT/US2021/051763 WO2022066916A1 (en) | 2020-09-23 | 2021-09-23 | Piperazine-based cationic lipids |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112023005207A2 true BR112023005207A2 (pt) | 2023-04-25 |
Family
ID=78414070
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112023005207A BR112023005207A2 (pt) | 2020-09-23 | 2021-09-23 | Lipídios catiônicos à base de piperazina |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US20230357166A1 (https=) |
| EP (1) | EP4216933B1 (https=) |
| JP (1) | JP7791182B2 (https=) |
| KR (1) | KR20230074526A (https=) |
| CN (1) | CN116457027A (https=) |
| AU (1) | AU2021349262A1 (https=) |
| BR (1) | BR112023005207A2 (https=) |
| CA (1) | CA3193347A1 (https=) |
| ES (1) | ES2994955T3 (https=) |
| IL (1) | IL301536A (https=) |
| MX (1) | MX2023003344A (https=) |
| WO (1) | WO2022066916A1 (https=) |
Families Citing this family (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2022099003A1 (en) | 2020-11-06 | 2022-05-12 | Sanofi | Lipid nanoparticles for delivering mrna vaccines |
| BR112023020027A2 (pt) * | 2021-04-15 | 2023-11-14 | Translate Bio Inc | Lipídeos catiônicos baseados em tampão "good" |
| WO2023178167A1 (en) * | 2022-03-16 | 2023-09-21 | Translate Bio, Inc. | Asymmetric piperazine-based cationic lipids |
| US20250263385A1 (en) | 2022-04-13 | 2025-08-21 | Sanofi | "Good" buffer-based cationic lipids |
| US20240150306A1 (en) * | 2022-09-27 | 2024-05-09 | Reinvigoron Theratech, Inc. | Compounds with cleavable disulfide moieties |
| EP4637813A1 (en) | 2022-12-20 | 2025-10-29 | Sanofi Pasteur | Rhinovirus mrna vaccine |
| CN116082275B (zh) * | 2023-03-13 | 2023-07-14 | 北京悦康科创医药科技股份有限公司 | 一种脾脏中高表达的阳离子脂质化合物、包含其的组合物及用途 |
| EP4734960A1 (en) | 2023-06-28 | 2026-05-06 | Sanofi Pasteur Inc. | Sterol analogs in lipid nanoparticle formulations |
| US12599560B2 (en) | 2023-11-02 | 2026-04-14 | Longuide Biopharma Corporation | Lipids and lipid nanoparticle formulations |
| CN121219273A (zh) * | 2023-11-02 | 2025-12-26 | 深圳厚存纳米药业有限公司 | 脂质和脂质纳米颗粒制剂 |
| US12539279B2 (en) | 2023-12-11 | 2026-02-03 | The Governing Council Of The University Of Toronto | Supramolecular ionizable lipid molecules with heteroatomic tuning for nucleic acid delivery |
| WO2025141521A1 (en) | 2023-12-29 | 2025-07-03 | Sanofi | Lipids having dendritic moieties |
| WO2025160118A1 (en) * | 2024-01-22 | 2025-07-31 | The Trustees Of The University Of Pennsylvania | Compositions and methods for hematopoietic stem cell (hsc) targeted delivery of therapeutic agents |
| WO2025255534A1 (en) * | 2024-06-07 | 2025-12-11 | Innovac Therapeutics Hk Limited | Diol-based novel ionizable lipids and their uses thereof |
| WO2026008743A1 (en) | 2024-07-02 | 2026-01-08 | Sanofi Pasteur Inc. | Water-soluble polyanionic polymer as adjuvant for carrier-formulated nucleic acid |
| WO2026053147A1 (en) | 2024-09-05 | 2026-03-12 | Sanofi Pasteur Inc. | Helper lipids and lnp compositions comprising same |
Family Cites Families (27)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4458066A (en) | 1980-02-29 | 1984-07-03 | University Patents, Inc. | Process for preparing polynucleotides |
| US4500707A (en) | 1980-02-29 | 1985-02-19 | University Patents, Inc. | Nucleosides useful in the preparation of polynucleotides |
| US5132418A (en) | 1980-02-29 | 1992-07-21 | University Patents, Inc. | Process for preparing polynucleotides |
| US4668777A (en) | 1981-03-27 | 1987-05-26 | University Patents, Inc. | Phosphoramidite nucleoside compounds |
| US4415732A (en) | 1981-03-27 | 1983-11-15 | University Patents, Inc. | Phosphoramidite compounds and processes |
| US4973679A (en) | 1981-03-27 | 1990-11-27 | University Patents, Inc. | Process for oligonucleo tide synthesis using phosphormidite intermediates |
| US4373071A (en) | 1981-04-30 | 1983-02-08 | City Of Hope Research Institute | Solid-phase synthesis of polynucleotides |
| US4401796A (en) | 1981-04-30 | 1983-08-30 | City Of Hope Research Institute | Solid-phase synthesis of polynucleotides |
| US5153319A (en) | 1986-03-31 | 1992-10-06 | University Patents, Inc. | Process for preparing polynucleotides |
| US5262530A (en) | 1988-12-21 | 1993-11-16 | Applied Biosystems, Inc. | Automated system for polynucleotide synthesis and purification |
| US5047524A (en) | 1988-12-21 | 1991-09-10 | Applied Biosystems, Inc. | Automated system for polynucleotide synthesis and purification |
| US5885613A (en) | 1994-09-30 | 1999-03-23 | The University Of British Columbia | Bilayer stabilizing components and their use in forming programmable fusogenic liposomes |
| US5780014A (en) | 1995-04-14 | 1998-07-14 | Inhale Therapeutic Systems | Method and apparatus for pulmonary administration of dry powder alpha 1-antitrypsin |
| US5700642A (en) | 1995-05-22 | 1997-12-23 | Sri International | Oligonucleotide sizing using immobilized cleavable primers |
| US5744335A (en) | 1995-09-19 | 1998-04-28 | Mirus Corporation | Process of transfecting a cell with a polynucleotide mixed with an amphipathic compound and a DNA-binding protein |
| US7101995B2 (en) * | 2001-08-27 | 2006-09-05 | Mirus Bio Corporation | Compositions and processes using siRNA, amphipathic compounds and polycations |
| KR101766408B1 (ko) | 2009-06-10 | 2017-08-10 | 알닐람 파마슈티칼스 인코포레이티드 | 향상된 지질 조성물 |
| WO2014028487A1 (en) | 2012-08-13 | 2014-02-20 | Massachusetts Institute Of Technology | Amine-containing lipidoids and uses thereof |
| ES3032935T3 (en) * | 2013-10-22 | 2025-07-29 | Translate Bio Inc | Lipid formulations for delivery of messenger rna |
| KR102559979B1 (ko) * | 2014-06-24 | 2023-07-25 | 샤이어 휴먼 지네틱 테라피즈 인크. | 핵산의 전달용 입체화학적으로 풍부한 조성물 |
| WO2016205691A1 (en) * | 2015-06-19 | 2016-12-22 | Massachusetts Institute Of Technology | Alkenyl substituted 2,5-piperazinediones and their use in compositions for delivering an agent to a subject or cell |
| AU2017356699B2 (en) * | 2016-11-08 | 2021-11-11 | Ramot At Tel-Aviv University Ltd. | Cationic lipids for nucleic acid delivery and preparation thereof |
| AU2017357758B2 (en) | 2016-11-10 | 2023-11-16 | Translate Bio, Inc. | Improved process of preparing mRNA-loaded lipid nanoparticles |
| JP7422977B2 (ja) * | 2018-07-23 | 2024-01-29 | トランスレイト バイオ， インコーポレイテッド | メッセンジャーｒｎａ用乾燥粉末製剤 |
| AU2019376660B2 (en) | 2018-11-09 | 2025-06-26 | Translate Bio, Inc. | 2,5-dioxopiperazine lipids with intercalated ester, thioester, disulfide and anhydride moieities |
| EP4146680A1 (en) | 2020-05-07 | 2023-03-15 | Translate Bio, Inc. | Composition and methods for treatment of primary ciliary dyskinesia |
| WO2021226468A1 (en) | 2020-05-07 | 2021-11-11 | Translate Bio, Inc. | Improved compositions for cftr mrna therapy |
-
2021
- 2021-09-23 IL IL301536A patent/IL301536A/en unknown
- 2021-09-23 AU AU2021349262A patent/AU2021349262A1/en active Pending
- 2021-09-23 JP JP2023518356A patent/JP7791182B2/ja active Active
- 2021-09-23 MX MX2023003344A patent/MX2023003344A/es unknown
- 2021-09-23 BR BR112023005207A patent/BR112023005207A2/pt unknown
- 2021-09-23 EP EP21799143.9A patent/EP4216933B1/en active Active
- 2021-09-23 CN CN202180075719.XA patent/CN116457027A/zh active Pending
- 2021-09-23 ES ES21799143T patent/ES2994955T3/es active Active
- 2021-09-23 KR KR1020237013611A patent/KR20230074526A/ko not_active Withdrawn
- 2021-09-23 WO PCT/US2021/051763 patent/WO2022066916A1/en not_active Ceased
- 2021-09-23 US US18/246,263 patent/US20230357166A1/en active Pending
- 2021-09-23 CA CA3193347A patent/CA3193347A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| AU2021349262A1 (en) | 2023-06-08 |
| US20230357166A1 (en) | 2023-11-09 |
| KR20230074526A (ko) | 2023-05-30 |
| CN116457027A (zh) | 2023-07-18 |
| AU2021349262A9 (en) | 2025-03-13 |
| ES2994955T3 (en) | 2025-02-04 |
| JP7791182B2 (ja) | 2025-12-23 |
| MX2023003344A (es) | 2023-06-05 |
| EP4216933B1 (en) | 2024-09-18 |
| WO2022066916A1 (en) | 2022-03-31 |
| IL301536A (en) | 2023-05-01 |
| JP2023542944A (ja) | 2023-10-12 |
| EP4216933A1 (en) | 2023-08-02 |
| CA3193347A1 (en) | 2022-03-31 |
| EP4216933C0 (en) | 2024-09-18 |
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