AU2022381750A1 - Lipid formulations containing nucleic acids and methods of treatment for cystic fibrosis - Google Patents
Lipid formulations containing nucleic acids and methods of treatment for cystic fibrosis Download PDFInfo
- Publication number
- AU2022381750A1 AU2022381750A1 AU2022381750A AU2022381750A AU2022381750A1 AU 2022381750 A1 AU2022381750 A1 AU 2022381750A1 AU 2022381750 A AU2022381750 A AU 2022381750A AU 2022381750 A AU2022381750 A AU 2022381750A AU 2022381750 A1 AU2022381750 A1 AU 2022381750A1
- Authority
- AU
- Australia
- Prior art keywords
- composition
- lipid
- mrna
- mol
- sequence
- 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
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
- C07C333/00—Derivatives of thiocarbamic acids, i.e. compounds containing any of the groups, the nitrogen atom not being part of nitro or nitroso groups
- C07C333/02—Monothiocarbamic acids; Derivatives thereof
- C07C333/04—Monothiocarbamic acids; Derivatives thereof having nitrogen atoms of thiocarbamic groups bound to hydrogen atoms or to acyclic carbon atoms
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D519/00—Heterocyclic compounds containing more than one system of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring system not provided for in groups C07D453/00 or C07D455/00
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/24—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing atoms other than carbon, hydrogen, oxygen, halogen, nitrogen or sulfur, e.g. cyclomethicone or phospholipids
-
- 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
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- 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
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/007—Pulmonary tract; Aromatherapy
- A61K9/0073—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
- A61K9/0078—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy for inhalation via a nebulizer such as a jet nebulizer, ultrasonic nebulizer, e.g. in the form of aqueous drug solutions or dispersions
-
- 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
- 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
-
- 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
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Life Sciences & Earth Sciences (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- General Health & Medical Sciences (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Biophysics (AREA)
- Dispersion Chemistry (AREA)
- Oil, Petroleum & Natural Gas (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Optics & Photonics (AREA)
- Nanotechnology (AREA)
- Otolaryngology (AREA)
- Pulmonology (AREA)
- Biomedical Technology (AREA)
- Dermatology (AREA)
- Physics & Mathematics (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163275402P | 2021-11-03 | 2021-11-03 | |
| US63/275,402 | 2021-11-03 | ||
| PCT/US2022/079244 WO2023081776A1 (en) | 2021-11-03 | 2022-11-03 | Lipid formulations containing nucleic acids and methods of treatment for cystic fibrosis |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AU2022381750A1 true AU2022381750A1 (en) | 2024-05-16 |
Family
ID=86242208
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2022381750A Pending AU2022381750A1 (en) | 2021-11-03 | 2022-11-03 | Lipid formulations containing nucleic acids and methods of treatment for cystic fibrosis |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US20230159449A1 (https=) |
| EP (1) | EP4426266A4 (https=) |
| JP (1) | JP2024542099A (https=) |
| AU (1) | AU2022381750A1 (https=) |
| CA (1) | CA3236675A1 (https=) |
| WO (1) | WO2023081776A1 (https=) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US12070509B2 (en) * | 2020-05-01 | 2024-08-27 | Arcturus Therapeutics, Inc. | Nucleic acids and methods of treatment for cystic fibrosis |
| CZ310613B6 (cs) | 2020-09-23 | 2026-01-28 | Ústav organické chemie a biochemie AV ČR, v. v. i. | Lipidoidy pro transfekci nukleových kyselin a jejich použití |
| CZ310443B6 (cs) | 2021-07-19 | 2025-06-25 | Ústav organické chemie a biochemie AV ČR, v. v. i. | Cyklohexanové lipidoidy pro transfekci nukleových kyselin a jejich použití |
| CN117653606B (zh) * | 2023-11-14 | 2024-05-10 | 广东康百医药科技有限公司 | 一种间充质干细胞外泌体冻干保护剂及其应用 |
| WO2025111297A1 (en) * | 2023-11-21 | 2025-05-30 | Modernatx, Inc. | Polynucleotides encoding cystic fibrosis transmembrane conductance regulator for the treatment of cystic fibrosis |
| WO2025166323A2 (en) | 2024-02-02 | 2025-08-07 | Editas Medicine, Inc. | Crispr-related methods and compositions targeting lipoprotein (a) expression |
| WO2025213138A1 (en) | 2024-04-05 | 2025-10-09 | Editas Medicine, Inc. | Crispr/rna-guided nuclease related methods and compositions for treating primary open angle glaucoma |
| WO2026003582A2 (en) | 2024-06-27 | 2026-01-02 | Axelyf ehf. | Lipids and lipid nanoparticles |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MX2019013752A (es) * | 2017-05-16 | 2020-07-20 | Translate Bio Inc | Tratamiento de la fibrosis quística mediante el suministro de arnm que codifica cftr optimizado en codones. |
| ES3039647T3 (en) * | 2017-05-31 | 2025-10-23 | Arcturus Therapeutics Inc | Synthesis and structure of high potency rna therapeutics |
| US12083224B2 (en) * | 2018-03-30 | 2024-09-10 | Arcturus Therapeutics, Inc. | Lipid particles for nucleic acid delivery |
| KR20210091120A (ko) * | 2018-08-29 | 2021-07-21 | 트랜슬레이트 바이오 인코포레이티드 | Mrna-로딩된 지질 나노입자를 제조하는 개선된 공정 |
| WO2021183563A1 (en) * | 2020-03-09 | 2021-09-16 | Arcturus Therapeutics, Inc. | Coronavirus vaccine compositions and methods |
-
2022
- 2022-11-03 EP EP22891055.0A patent/EP4426266A4/en active Pending
- 2022-11-03 WO PCT/US2022/079244 patent/WO2023081776A1/en not_active Ceased
- 2022-11-03 US US18/052,505 patent/US20230159449A1/en active Pending
- 2022-11-03 CA CA3236675A patent/CA3236675A1/en active Pending
- 2022-11-03 AU AU2022381750A patent/AU2022381750A1/en active Pending
- 2022-11-03 JP JP2024526594A patent/JP2024542099A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| JP2024542099A (ja) | 2024-11-13 |
| EP4426266A4 (en) | 2025-11-05 |
| WO2023081776A1 (en) | 2023-05-11 |
| US20230159449A1 (en) | 2023-05-25 |
| CA3236675A1 (en) | 2023-05-11 |
| EP4426266A1 (en) | 2024-09-11 |
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