HUE042177T2 - Szteroidszármazék mRNS szállítására humán genetikai betegségekben - Google Patents
Szteroidszármazék mRNS szállítására humán genetikai betegségekbenInfo
- Publication number
- HUE042177T2 HUE042177T2 HUE18156531A HUE18156531A HUE042177T2 HU E042177 T2 HUE042177 T2 HU E042177T2 HU E18156531 A HUE18156531 A HU E18156531A HU E18156531 A HUE18156531 A HU E18156531A HU E042177 T2 HUE042177 T2 HU E042177T2
- Authority
- HU
- Hungary
- Prior art keywords
- mrna
- delivery
- genetic diseases
- human genetic
- steroid derivative
- Prior art date
Links
- 208000026350 Inborn Genetic disease Diseases 0.000 title 1
- 208000016361 genetic disease Diseases 0.000 title 1
- 108020004999 messenger RNA Proteins 0.000 title 1
- 150000003431 steroids Chemical class 0.000 title 1
Classifications
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- 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
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
-
- 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
-
- 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
-
- 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
- A61K9/10—Dispersions; Emulsions
- A61K9/107—Emulsions ; Emulsion preconcentrates; Micelles
- A61K9/1075—Microemulsions or submicron emulsions; Preconcentrates or solids thereof; Micelles, e.g. made of phospholipids or block copolymers
-
- 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
-
- 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
- A61K9/48—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
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07J—STEROIDS
- C07J43/00—Normal steroids having a nitrogen-containing hetero ring spiro-condensed or not condensed with the cyclopenta(a)hydrophenanthrene skeleton
- C07J43/003—Normal steroids having a nitrogen-containing hetero ring spiro-condensed or not condensed with the cyclopenta(a)hydrophenanthrene skeleton not condensed
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- Dispersion Chemistry (AREA)
- Biomedical Technology (AREA)
- Biophysics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Physics & Mathematics (AREA)
- Dermatology (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Gastroenterology & Hepatology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Nanotechnology (AREA)
- Optics & Photonics (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
- Steroid Compounds (AREA)
- Enzymes And Modification Thereof (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US26565309P | 2009-12-01 | 2009-12-01 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HUE042177T2 true HUE042177T2 (hu) | 2019-06-28 |
Family
ID=44115246
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HUE10835016A HUE038039T2 (hu) | 2009-12-01 | 2010-11-30 | mRNS bejuttatása fehérjék és enzimek kiegészítésére humán genetikai betegségekben |
| HUE18156531A HUE042177T2 (hu) | 2009-12-01 | 2010-11-30 | Szteroidszármazék mRNS szállítására humán genetikai betegségekben |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HUE10835016A HUE038039T2 (hu) | 2009-12-01 | 2010-11-30 | mRNS bejuttatása fehérjék és enzimek kiegészítésére humán genetikai betegségekben |
Country Status (20)
| Country | Link |
|---|---|
| US (10) | US20110244026A1 (hu) |
| EP (5) | EP3318248B1 (hu) |
| AU (1) | AU2010326132B9 (hu) |
| CA (2) | CA3077990A1 (hu) |
| CY (2) | CY1120272T1 (hu) |
| DK (2) | DK2506857T3 (hu) |
| ES (3) | ES2713852T3 (hu) |
| HR (2) | HRP20180696T1 (hu) |
| HU (2) | HUE038039T2 (hu) |
| LT (2) | LT3338765T (hu) |
| ME (2) | ME03327B (hu) |
| NO (1) | NO2506857T3 (hu) |
| NZ (5) | NZ750556A (hu) |
| PL (2) | PL2506857T3 (hu) |
| PT (2) | PT3338765T (hu) |
| RS (2) | RS57314B1 (hu) |
| SI (2) | SI2506857T1 (hu) |
| SM (2) | SMT201900113T1 (hu) |
| TR (1) | TR201901311T4 (hu) |
| WO (1) | WO2011068810A1 (hu) |
Families Citing this family (313)
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|---|---|---|---|---|
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| JP5940064B2 (ja) | 2010-07-06 | 2016-06-29 | ノバルティス アーゲー | 低用量のｒｎａを用いた大型哺乳動物の免疫化 |
| SI2590626T1 (sl) | 2010-07-06 | 2016-01-29 | Glaxosmithkline Biologicals S.A. | Liposomi z lipidi, ki imajo koristno pKa vrednost, za dostavo RNA |
| ES2600892T3 (es) | 2010-07-06 | 2017-02-13 | Glaxosmithkline Biologicals Sa | Partículas de administración de tipo virión para moléculas de ARN autorreplicante |
| SI3243526T1 (sl) | 2010-07-06 | 2020-02-28 | Glaxosmithkline Biologicals S.A. | Dostava RNA za sprožitev večih imunskih poti |
| US9770463B2 (en) | 2010-07-06 | 2017-09-26 | Glaxosmithkline Biologicals Sa | Delivery of RNA to different cell types |
| EP3578205A1 (en) | 2010-08-06 | 2019-12-11 | ModernaTX, Inc. | A pharmaceutical formulation comprising engineered nucleic acids and medical use thereof |
| US9193827B2 (en) | 2010-08-26 | 2015-11-24 | Massachusetts Institute Of Technology | Poly(beta-amino alcohols), their preparation, and uses thereof |
| PL2611467T3 (pl) | 2010-08-31 | 2022-08-16 | Glaxosmithkline Biologicals Sa | Małe liposomy do dostarczania rna kodującego immunogen |
| EP3970742B1 (en) | 2010-08-31 | 2022-05-25 | GlaxoSmithKline Biologicals S.A. | Pegylated liposomes for delivery of immunogen-encoding rna |
| WO2012031908A1 (de) * | 2010-09-09 | 2012-03-15 | Saint-Gobain Glass France | Transparente scheibe mit heizbeschichtung |
| FI4108671T3 (fi) | 2010-10-01 | 2024-12-27 | Modernatx Inc | Muokattuja nukleosideja, nukleotideja ja nukleiinihappoja sekä niiden käyttötapoja |
| BR112013008700B8 (pt) | 2010-10-11 | 2022-10-04 | Novartis Ag | Molécula de rna auto-replicante, partícula de replicon de alfavírus, composição, molécula de dna recombinante, uso da molécula de rna auto-replicante |
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| EP2691443B1 (en) | 2011-03-28 | 2021-02-17 | Massachusetts Institute of Technology | Conjugated lipomers and uses thereof |
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