CA3175957A1 - Novel lipids and nanoparticle compositions thereof - Google Patents
Novel lipids and nanoparticle compositions thereofInfo
- Publication number
- CA3175957A1 CA3175957A1 CA3175957A CA3175957A CA3175957A1 CA 3175957 A1 CA3175957 A1 CA 3175957A1 CA 3175957 A CA3175957 A CA 3175957A CA 3175957 A CA3175957 A CA 3175957A CA 3175957 A1 CA3175957 A1 CA 3175957A1
- Authority
- CA
- Canada
- Prior art keywords
- lipid
- lipid nanoparticle
- cedna
- itr
- nanoparticle
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
-
- 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
- A61K48/0041—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 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/06—Antianaemics
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
- C07C323/00—Thiols, sulfides, hydropolysulfides or polysulfides substituted by halogen, oxygen or nitrogen atoms, or by sulfur atoms not being part of thio groups
- C07C323/23—Thiols, sulfides, hydropolysulfides or polysulfides substituted by halogen, oxygen or nitrogen atoms, or by sulfur atoms not being part of thio groups containing thio groups and nitrogen atoms, not being part of nitro or nitroso groups, bound to the same carbon skeleton
- C07C323/24—Thiols, sulfides, hydropolysulfides or polysulfides substituted by halogen, oxygen or nitrogen atoms, or by sulfur atoms not being part of thio groups containing thio groups and nitrogen atoms, not being part of nitro or nitroso groups, bound to the same carbon skeleton having the sulfur atoms of the thio groups bound to acyclic carbon atoms of the carbon skeleton
- C07C323/25—Thiols, sulfides, hydropolysulfides or polysulfides substituted by halogen, oxygen or nitrogen atoms, or by sulfur atoms not being part of thio groups containing thio groups and nitrogen atoms, not being part of nitro or nitroso groups, bound to the same carbon skeleton having the sulfur atoms of the thio groups bound to acyclic carbon atoms of the carbon skeleton the carbon skeleton being acyclic and saturated
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D211/00—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings
- C07D211/04—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom
- C07D211/06—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members
- C07D211/08—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hydrocarbon or substituted hydrocarbon radicals directly attached to ring carbon atoms
- C07D211/18—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hydrocarbon or substituted hydrocarbon radicals directly attached to ring carbon atoms with substituted hydrocarbon radicals attached to ring carbon atoms
- C07D211/20—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hydrocarbon or substituted hydrocarbon radicals directly attached to ring carbon atoms with substituted hydrocarbon radicals attached to ring carbon atoms with hydrocarbon radicals, substituted by singly bound oxygen or sulphur atoms
- C07D211/24—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hydrocarbon or substituted hydrocarbon radicals directly attached to ring carbon atoms with substituted hydrocarbon radicals attached to ring carbon atoms with hydrocarbon radicals, substituted by singly bound oxygen or sulphur atoms by sulfur atoms to which a second hetero atom is attached
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Biomedical Technology (AREA)
- Genetics & Genomics (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Epidemiology (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Physics & Mathematics (AREA)
- Biochemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Nanotechnology (AREA)
- Optics & Photonics (AREA)
- Plant Pathology (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063026479P | 2020-05-18 | 2020-05-18 | |
| US63/026,479 | 2020-05-18 | ||
| PCT/US2021/032676 WO2021236479A2 (en) | 2020-05-18 | 2021-05-17 | Novel lipids and nanoparticle compositions thereof |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3175957A1 true CA3175957A1 (en) | 2021-11-25 |
Family
ID=78707483
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3175957A Pending CA3175957A1 (en) | 2020-05-18 | 2021-05-17 | Novel lipids and nanoparticle compositions thereof |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20230181764A1 (https=) |
| EP (1) | EP4153144A4 (https=) |
| JP (1) | JP2023527747A (https=) |
| CN (1) | CN115968280A (https=) |
| AU (1) | AU2021274481A1 (https=) |
| CA (1) | CA3175957A1 (https=) |
| IL (1) | IL298241A (https=) |
| MX (1) | MX2022014484A (https=) |
| WO (1) | WO2021236479A2 (https=) |
Families Citing this family (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN113166783B (zh) | 2018-10-09 | 2024-10-11 | 不列颠哥伦比亚大学 | 包含无有机溶剂和去污剂的转染活性囊泡的组合物和系统以及与之相关的方法 |
| MX2023000806A (es) * | 2020-07-17 | 2023-04-11 | Generation Bio Co | Métodos para encapsular polinucleótidos en tamaños reducidos de nanopartículas lipídicas y formulación novedosa de estos. |
| CN114716355B (zh) * | 2022-04-02 | 2023-09-05 | 华南理工大学 | 一种脂质化合物、包含其的组合物及应用 |
| CN121532216A (zh) * | 2023-04-18 | 2026-02-13 | 赛仁生物技术公司 | 腺相关病毒载体组合物和使用方法 |
| US12311033B2 (en) | 2023-05-31 | 2025-05-27 | Capstan Therapeutics, Inc. | Lipid nanoparticle formulations and compositions |
| CN117925729B (zh) * | 2024-01-19 | 2025-06-10 | 晟迪生物医药(苏州)有限公司 | 基于空白脂质纳米颗粒的转染试剂、其制备方法和应用 |
| WO2026003582A2 (en) | 2024-06-27 | 2026-01-02 | Axelyf ehf. | Lipids and lipid nanoparticles |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB0610479D0 (en) * | 2006-05-26 | 2006-07-05 | Ge Healthcare Bio Sciences Ab | A method for generating metal chelating affinity ligands |
| JP6093710B2 (ja) * | 2011-11-18 | 2017-03-08 | 日油株式会社 | 細胞内動態を改善したカチオン性脂質 |
| CA2975371C (en) * | 2015-01-30 | 2024-02-13 | Nof Corporation | Cationic lipid for nucleic acid delivery |
| US11395798B2 (en) * | 2015-10-08 | 2022-07-26 | Nof Corporation | O/W type emulsion |
| WO2018237369A2 (en) * | 2017-06-23 | 2018-12-27 | Vical Incorporated | Lipid nanoparticle (lnp)-mediated delivery of a crispr-expressing plasmid dna for treating chronic hepatitis b virus infection |
| JP2020532981A (ja) * | 2017-09-08 | 2020-11-19 | ジェネレーション バイオ カンパニー | 修飾型閉端dna(cedna) |
| WO2019188867A1 (ja) * | 2018-03-27 | 2019-10-03 | 日油株式会社 | 細胞内動態を改善した新規カチオン性脂質 |
| JP7700101B2 (ja) * | 2019-09-06 | 2025-06-30 | ジェネレーション バイオ カンパニー | 閉端dnaおよび切断可能脂質を含む脂質ナノ粒子組成物ならびにそれらの使用方法 |
| WO2021060440A1 (ja) * | 2019-09-26 | 2021-04-01 | 日油株式会社 | 脂質ナノ粒子の凍結乾燥組成物 |
| AU2020385378A1 (en) * | 2019-11-22 | 2022-04-07 | Generation Bio Co. | Ionizable lipids and nanoparticle compositions thereof |
| KR20220121246A (ko) * | 2019-12-20 | 2022-08-31 | 큐어백 아게 | 핵산 전달용 신규한 지질 나노입자 |
-
2021
- 2021-05-17 US US17/925,404 patent/US20230181764A1/en active Pending
- 2021-05-17 CN CN202180035954.4A patent/CN115968280A/zh active Pending
- 2021-05-17 WO PCT/US2021/032676 patent/WO2021236479A2/en not_active Ceased
- 2021-05-17 CA CA3175957A patent/CA3175957A1/en active Pending
- 2021-05-17 JP JP2022570302A patent/JP2023527747A/ja active Pending
- 2021-05-17 AU AU2021274481A patent/AU2021274481A1/en active Pending
- 2021-05-17 EP EP21808224.6A patent/EP4153144A4/en active Pending
- 2021-05-17 MX MX2022014484A patent/MX2022014484A/es unknown
- 2021-05-17 IL IL298241A patent/IL298241A/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| US20230181764A1 (en) | 2023-06-15 |
| WO2021236479A2 (en) | 2021-11-25 |
| EP4153144A2 (en) | 2023-03-29 |
| MX2022014484A (es) | 2022-12-13 |
| AU2021274481A1 (en) | 2022-11-03 |
| IL298241A (en) | 2023-01-01 |
| CN115968280A (zh) | 2023-04-14 |
| JP2023527747A (ja) | 2023-06-30 |
| EP4153144A4 (en) | 2024-07-03 |
| WO2021236479A8 (en) | 2022-02-24 |
| WO2021236479A3 (en) | 2021-12-23 |
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| US20240293574A1 (en) | Cationic lipids and compositions thereof | |
| US12263228B2 (en) | Lipids and compositions thereof | |
| US20230181764A1 (en) | Novel lipids and nanoparticle compositions thereof |
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