ZA202403337B - Lipid nanoparticles for oligonucleotide delivery - Google Patents
Lipid nanoparticles for oligonucleotide deliveryInfo
- Publication number
- ZA202403337B ZA202403337B ZA2024/03337A ZA202403337A ZA202403337B ZA 202403337 B ZA202403337 B ZA 202403337B ZA 2024/03337 A ZA2024/03337 A ZA 2024/03337A ZA 202403337 A ZA202403337 A ZA 202403337A ZA 202403337 B ZA202403337 B ZA 202403337B
- Authority
- ZA
- South Africa
- Prior art keywords
- lipid nanoparticles
- lipid
- oligonucleotide delivery
- formula
- compound according
- Prior art date
Links
Classifications
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D295/00—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms
- C07D295/04—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms
- C07D295/14—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals
- C07D295/145—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals with the ring nitrogen atoms and the carbon atoms with three bonds to hetero atoms attached to the same carbon chain, which is not interrupted by carbocyclic rings
- C07D295/15—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals with the ring nitrogen atoms and the carbon atoms with three bonds to hetero atoms attached to the same carbon chain, which is not interrupted by carbocyclic rings to an acyclic saturated chain
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/385—Haptens or antigens, bound to carriers
-
- 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
- A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
- A61K47/10—Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
-
- 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
- A61K47/22—Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
-
- 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
- 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
- A61K47/28—Steroids, e.g. cholesterol, bile acids or glycyrrhetinic acid
-
- 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
- A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
- A61K47/34—Macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyesters, polyamino acids, polysiloxanes, polyphosphazines, copolymers of polyalkylene glycol or poloxamers
-
- 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
-
- 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
- A61K9/513—Organic macromolecular compounds; Dendrimers
- A61K9/5146—Organic macromolecular compounds; Dendrimers obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyethylene glycol, polyamines, polyanhydrides
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D295/00—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms
- C07D295/04—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms
- C07D295/12—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by singly or doubly bound nitrogen atoms
- C07D295/125—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by singly or doubly bound nitrogen atoms with the ring nitrogen atoms and the substituent nitrogen atoms attached to the same carbon chain, which is not interrupted by carbocyclic rings
- C07D295/13—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by singly or doubly bound nitrogen atoms with the ring nitrogen atoms and the substituent nitrogen atoms attached to the same carbon chain, which is not interrupted by carbocyclic rings to an acyclic saturated chain
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K2039/51—Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
- A61K2039/53—DNA (RNA) vaccination
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K2039/60—Medicinal preparations containing antigens or antibodies characteristics by the carrier linked to the antigen
- A61K2039/6018—Lipids, e.g. in lipopeptides
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Engineering & Computer Science (AREA)
- Public Health (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Oil, Petroleum & Natural Gas (AREA)
- General Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Nanotechnology (AREA)
- Optics & Photonics (AREA)
- Physics & Mathematics (AREA)
- Molecular Biology (AREA)
- Mycology (AREA)
- Microbiology (AREA)
- Immunology (AREA)
- Biophysics (AREA)
- Inorganic Chemistry (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Genetics & Genomics (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP21205899 | 2021-11-02 | ||
| PCT/EP2022/080576 WO2023078946A1 (en) | 2021-11-02 | 2022-11-02 | Lipid nanoparticles for oligonucleotide delivery |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| ZA202403337B true ZA202403337B (en) | 2025-08-27 |
Family
ID=78500458
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ZA2024/03337A ZA202403337B (en) | 2021-11-02 | 2024-04-29 | Lipid nanoparticles for oligonucleotide delivery |
Country Status (16)
| Country | Link |
|---|---|
| US (1) | US20250073177A1 (https=) |
| EP (2) | EP4613740A3 (https=) |
| JP (1) | JP2024540101A (https=) |
| KR (1) | KR20240099375A (https=) |
| CN (1) | CN118215472A (https=) |
| AU (1) | AU2022381685A1 (https=) |
| CA (1) | CA3236638A1 (https=) |
| DK (1) | DK4426269T3 (https=) |
| ES (1) | ES3048411T3 (https=) |
| FI (1) | FI4426269T3 (https=) |
| IL (1) | IL312431B2 (https=) |
| MX (1) | MX2024005110A (https=) |
| PL (1) | PL4426269T3 (https=) |
| PT (1) | PT4426269T (https=) |
| WO (1) | WO2023078946A1 (https=) |
| ZA (1) | ZA202403337B (https=) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN118125994A (zh) * | 2022-11-25 | 2024-06-04 | 艾斯拓康医药科技(北京)有限公司 | 可离子化脂质及其应用 |
| WO2025088152A1 (en) | 2023-10-27 | 2025-05-01 | Ziphius Nv | MODIFIED 5'UTRs |
| US12599560B2 (en) | 2023-11-02 | 2026-04-14 | Longuide Biopharma Corporation | Lipids and lipid nanoparticle formulations |
| CN117550985B (zh) * | 2023-11-08 | 2025-02-18 | 深圳厚存纳米药业有限公司 | 一种脂质化合物及其用途 |
| WO2025104023A1 (en) | 2023-11-13 | 2025-05-22 | Ziphius Nv | MODIFIED ALPHAVIRAL nsP3 |
| WO2025140594A1 (zh) * | 2023-12-29 | 2025-07-03 | 厦门赛诺邦格生物科技股份有限公司 | 一种含有多级叔胺的不对称阳离子脂质 |
| WO2026046237A1 (zh) * | 2024-08-27 | 2026-03-05 | 北京剂泰生命科技有限公司 | 肺靶向的脂质化合物 |
| CN120943786A (zh) * | 2025-07-17 | 2025-11-14 | 深圳市艾迪贝克生物技术有限公司 | 脂质化合物、其纳米颗粒及应用 |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6451592B1 (en) | 1996-04-05 | 2002-09-17 | Chiron Corporation | Recombinant alphavirus-based vectors with reduced inhibition of cellular macromolecular synthesis |
| EP1751289B1 (en) | 2004-05-18 | 2009-01-14 | Alphavax, Inc. | Tc-83-derived alphavirus vectors, particles and methods |
| US7332322B2 (en) | 2004-09-14 | 2008-02-19 | Ilya Frolov | Venezuelan equine encephalitis virus replicons with adaptive mutations in the genome and uses thereof |
| US8691750B2 (en) * | 2011-05-17 | 2014-04-08 | Axolabs Gmbh | Lipids and compositions for intracellular delivery of biologically active compounds |
| EP2882706A1 (en) * | 2012-08-13 | 2015-06-17 | Massachusetts Institute of Technology | Amine-containing lipidoids and uses thereof |
| US12077501B2 (en) * | 2017-06-14 | 2024-09-03 | Modernatx, Inc. | Compounds and compositions for intracellular delivery of agents |
-
2022
- 2022-11-02 US US18/705,454 patent/US20250073177A1/en active Pending
- 2022-11-02 AU AU2022381685A patent/AU2022381685A1/en active Pending
- 2022-11-02 JP JP2024525401A patent/JP2024540101A/ja active Pending
- 2022-11-02 IL IL312431A patent/IL312431B2/en unknown
- 2022-11-02 DK DK22813503.4T patent/DK4426269T3/da active
- 2022-11-02 PL PL22813503.4T patent/PL4426269T3/pl unknown
- 2022-11-02 PT PT228135034T patent/PT4426269T/pt unknown
- 2022-11-02 FI FIEP22813503.4T patent/FI4426269T3/fi active
- 2022-11-02 ES ES22813503T patent/ES3048411T3/es active Active
- 2022-11-02 EP EP25189030.7A patent/EP4613740A3/en active Pending
- 2022-11-02 WO PCT/EP2022/080576 patent/WO2023078946A1/en not_active Ceased
- 2022-11-02 KR KR1020247018042A patent/KR20240099375A/ko active Pending
- 2022-11-02 CA CA3236638A patent/CA3236638A1/en active Pending
- 2022-11-02 MX MX2024005110A patent/MX2024005110A/es unknown
- 2022-11-02 EP EP22813503.4A patent/EP4426269B1/en active Active
- 2022-11-02 CN CN202280072360.5A patent/CN118215472A/zh active Pending
-
2024
- 2024-04-29 ZA ZA2024/03337A patent/ZA202403337B/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| PT4426269T (pt) | 2025-09-03 |
| PL4426269T3 (pl) | 2025-11-17 |
| US20250073177A1 (en) | 2025-03-06 |
| IL312431B2 (en) | 2026-04-01 |
| ES3048411T3 (en) | 2025-12-10 |
| JP2024540101A (ja) | 2024-10-31 |
| EP4426269B1 (en) | 2025-07-16 |
| AU2022381685A1 (en) | 2024-05-16 |
| EP4613740A3 (en) | 2025-10-29 |
| IL312431B1 (en) | 2025-12-01 |
| KR20240099375A (ko) | 2024-06-28 |
| WO2023078946A1 (en) | 2023-05-11 |
| DK4426269T3 (da) | 2025-10-20 |
| MX2024005110A (es) | 2024-05-31 |
| EP4426269A1 (en) | 2024-09-11 |
| CN118215472A (zh) | 2024-06-18 |
| CA3236638A1 (en) | 2023-05-11 |
| EP4613740A2 (en) | 2025-09-10 |
| FI4426269T3 (fi) | 2025-10-20 |
| IL312431A (en) | 2024-06-01 |
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