ME03091B - Isporuka irnk za povećanje ekspresije proteina i enzima u humanim genetskim oboljenjima - Google Patents
Isporuka irnk za povećanje ekspresije proteina i enzima u humanim genetskim oboljenjimaInfo
- Publication number
- ME03091B ME03091B MEP-2018-113A MEP11318A ME03091B ME 03091 B ME03091 B ME 03091B ME P11318 A MEP11318 A ME P11318A ME 03091 B ME03091 B ME 03091B
- Authority
- ME
- Montenegro
- Prior art keywords
- augmentation
- mrna
- enzymes
- proteins
- delivery
- Prior art date
Links
- 102000004190 Enzymes Human genes 0.000 title 1
- 108090000790 Enzymes Proteins 0.000 title 1
- 208000026350 Inborn Genetic disease Diseases 0.000 title 1
- 230000003416 augmentation Effects 0.000 title 1
- 208000016361 genetic disease Diseases 0.000 title 1
- 108020004999 messenger RNA Proteins 0.000 title 1
- 102000004169 proteins and genes Human genes 0.000 title 1
- 108090000623 proteins and genes Proteins 0.000 title 1
Classifications
-
- 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)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Medicinal Chemistry (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)
- Biomedical Technology (AREA)
- Dispersion Chemistry (AREA)
- Biophysics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Physics & Mathematics (AREA)
- Dermatology (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Gastroenterology & Hepatology (AREA)
- Chemical Kinetics & Catalysis (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 (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US26565309P | 2009-12-01 | 2009-12-01 | |
| PCT/US2010/058457 WO2011068810A1 (en) | 2009-12-01 | 2010-11-30 | Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases |
| EP10835016.6A EP2506857B1 (en) | 2009-12-01 | 2010-11-30 | Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| ME03091B true ME03091B (me) | 2019-01-20 |
Family
ID=44115246
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MEP-2019-46A ME03327B (me) | 2009-12-01 | 2010-11-30 | Stereoidni derivati za isporuku irnk u humanim genetskim oboljenjima |
| MEP-2018-113A ME03091B (me) | 2009-12-01 | 2010-11-30 | Isporuka irnk za povećanje ekspresije proteina i enzima u humanim genetskim oboljenjima |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MEP-2019-46A ME03327B (me) | 2009-12-01 | 2010-11-30 | Stereoidni derivati za isporuku irnk u humanim genetskim oboljenjima |
Country Status (20)
| Country | Link |
|---|---|
| US (10) | US20110244026A1 (me) |
| EP (5) | EP3403647A1 (me) |
| AU (1) | AU2010326132B9 (me) |
| CA (2) | CA3077990A1 (me) |
| CY (2) | CY1120272T1 (me) |
| DK (2) | DK3338765T3 (me) |
| ES (3) | ES2713852T3 (me) |
| HR (2) | HRP20180696T1 (me) |
| HU (2) | HUE042177T2 (me) |
| LT (2) | LT2506857T (me) |
| ME (2) | ME03327B (me) |
| NO (1) | NO2506857T3 (me) |
| NZ (5) | NZ716192A (me) |
| PL (2) | PL3338765T3 (me) |
| PT (2) | PT2506857T (me) |
| RS (2) | RS58405B1 (me) |
| SI (2) | SI3338765T1 (me) |
| SM (2) | SMT201800220T1 (me) |
| TR (1) | TR201901311T4 (me) |
| WO (1) | WO2011068810A1 (me) |
Families Citing this family (318)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MX353900B (es) | 2008-11-07 | 2018-02-01 | Massachusetts Inst Technology | Lipidoides de aminoalcohol y usos de los mismos. |
| EP3403647A1 (en) | 2009-12-01 | 2018-11-21 | Translate Bio, Inc. | Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases |
| DK3243526T3 (da) | 2010-07-06 | 2020-02-17 | Glaxosmithkline Biologicals Sa | Levering af rna til at udløse flere immunsignalveje |
| EP2591114B1 (en) | 2010-07-06 | 2016-06-08 | GlaxoSmithKline Biologicals SA | Immunisation of large mammals with low doses of rna |
| US9770463B2 (en) | 2010-07-06 | 2017-09-26 | Glaxosmithkline Biologicals Sa | Delivery of RNA to different cell types |
| PL2590676T3 (pl) | 2010-07-06 | 2017-02-28 | Glaxosmithkline Biologicals Sa | Wirionopodobne cząstki dostarczające dla autoreplikujących się cząsteczek rna |
| BR112013000244A2 (pt) | 2010-07-06 | 2016-05-17 | Novartis Ag | lipossomas com lipídeos apresentando pka vantajoso para administração de rna |
| CA2807552A1 (en) | 2010-08-06 | 2012-02-09 | Moderna Therapeutics, Inc. | Engineered nucleic acids and methods of use thereof |
| WO2012027675A2 (en) | 2010-08-26 | 2012-03-01 | Massachusetts Institute Of Technology | Poly(beta-amino alcohols), their preparation, and uses thereof |
| MX341989B (es) | 2010-08-31 | 2016-09-09 | Novartis Ag * | Liposomas pequeños para el suministro de arn que codifica el inmunogeno. |
| RS63315B1 (sr) | 2010-08-31 | 2022-07-29 | Glaxosmithkline Biologicals Sa | Pegilovani lipozomi za isporuku rnk koja kodira imunogen |
| DE212011100045U1 (de) * | 2010-09-09 | 2012-10-25 | Saint-Gobain Glass France | Transparente Scheibe mit Heizbeschichtung |
| EP3431485B2 (en) | 2010-10-01 | 2024-09-04 | ModernaTX, Inc. | Engineered nucleic acids and methods of use thereof |
| CA2814386C (en) | 2010-10-11 | 2019-08-20 | Novartis Ag | Antigen delivery platforms |
| US8853377B2 (en) | 2010-11-30 | 2014-10-07 | Shire Human Genetic Therapies, Inc. | mRNA for use in treatment of human genetic diseases |
| US9238716B2 (en) | 2011-03-28 | 2016-01-19 | Massachusetts Institute Of Technology | Conjugated lipomers and uses thereof |
| WO2012135805A2 (en) | 2011-03-31 | 2012-10-04 | modeRNA Therapeutics | Delivery and formulation of engineered nucleic acids |
| US20140206753A1 (en) | 2011-06-08 | 2014-07-24 | Shire Human Genetic Therapies, Inc. | Lipid nanoparticle compositions and methods for mrna delivery |
| CA3198966A1 (en) | 2011-06-08 | 2012-12-13 | Translate Bio, Inc. | Cleavable lipids |
| CA2840989A1 (en) | 2011-07-06 | 2013-01-10 | Novartis Ag | Immunogenic combination compositions and uses thereof |
| CN102253778B (zh) * | 2011-07-22 | 2013-06-19 | 苏州瀚瑞微电子有限公司 | 一种电容传感器的定位方法 |
| WO2013066438A2 (en) | 2011-07-22 | 2013-05-10 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| US9464124B2 (en) | 2011-09-12 | 2016-10-11 | Moderna Therapeutics, Inc. | Engineered nucleic acids and methods of use thereof |
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| PE20181541A1 (es) | 2011-10-27 | 2018-09-26 | Massachusetts Inst Technology | Derivados de aminoacidos funcionalizados en la terminal n capaces de formar microesferas encapsuladoras de farmaco |
| US9579338B2 (en) | 2011-11-04 | 2017-02-28 | Nitto Denko Corporation | Method of producing lipid nanoparticles for drug delivery |
| CA2853689C (en) * | 2011-11-04 | 2020-06-30 | Nitto Denko Corporation | Method of producing lipid nanoparticles for drug delivery |
| KR20140102759A (ko) | 2011-12-16 | 2014-08-22 | 모더나 세라퓨틱스, 인코포레이티드 | 변형된 뉴클레오사이드, 뉴클레오타이드 및 핵산 조성물 |
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| AU2013243949A1 (en) | 2012-04-02 | 2014-10-30 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of biologics and proteins associated with human disease |
| DE18200782T1 (de) | 2012-04-02 | 2021-10-21 | Modernatx, Inc. | Modifizierte polynukleotide zur herstellung von proteinen im zusammenhang mit erkrankungen beim menschen |
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| EP2859102A4 (en) | 2012-06-08 | 2016-05-11 | Shire Human Genetic Therapies | NUCLEASE RESISTANT POLYNUCLEOTIDES AND USES THEREOF |
| WO2014028487A1 (en) | 2012-08-13 | 2014-02-20 | Massachusetts Institute Of Technology | Amine-containing lipidoids and uses thereof |
| EP2885419A4 (en) | 2012-08-14 | 2016-05-25 | Moderna Therapeutics Inc | ENZYMES AND POLYMERASES FOR RNA SYNTHESIS |
| CA2892529C (en) | 2012-11-26 | 2023-04-25 | Moderna Therapeutics, Inc. | Terminally modified rna |
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| HK1217215A1 (zh) | 2013-01-17 | 2016-12-30 | Modernatx, Inc. | 用於改变细胞表型的信号传感器多核苷酸 |
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| ES2981185T3 (es) | 2013-03-14 | 2024-10-07 | Translate Bio Inc | Métodos para la purificación de ARN mensajero |
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| MX2015011945A (es) | 2013-03-14 | 2015-12-01 | Shire Human Genetic Therapies | Evaluacion cuantitativa para la eficacia de los casquetes de arn mensajero. |
| US8980864B2 (en) | 2013-03-15 | 2015-03-17 | Moderna Therapeutics, Inc. | Compositions and methods of altering cholesterol levels |
| PL4332576T3 (pl) * | 2013-03-15 | 2025-09-08 | Translate Bio, Inc. | Synergistyczne wzmocnienie dostarczania kwasów nukleinowych poprzez formulacje zmieszane |
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| US11377470B2 (en) | 2013-03-15 | 2022-07-05 | Modernatx, Inc. | Ribonucleic acid purification |
| EP2971033B8 (en) | 2013-03-15 | 2019-07-10 | ModernaTX, Inc. | Manufacturing methods for production of rna transcripts |
| US10077439B2 (en) | 2013-03-15 | 2018-09-18 | Modernatx, Inc. | Removal of DNA fragments in mRNA production process |
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| WO2014179562A1 (en) | 2013-05-01 | 2014-11-06 | Massachusetts Institute Of Technology | 1,3,5-triazinane-2,4,6-trione derivatives and uses thereof |
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| EP3164112A1 (en) * | 2014-07-02 | 2017-05-10 | Shire Human Genetic Therapies, Inc. | Encapsulation of messenger rna |
| US9840479B2 (en) | 2014-07-02 | 2017-12-12 | Massachusetts Institute Of Technology | Polyamine-fatty acid derived lipidoids and uses thereof |
| US10195280B2 (en) | 2014-07-15 | 2019-02-05 | Life Technologies Corporation | Compositions and methods for efficient delivery of molecules to cells |
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- 2010-11-30 EP EP17207662.2A patent/EP3318248B1/en not_active Revoked
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- 2010-11-30 NO NO10835016A patent/NO2506857T3/no unknown
- 2010-11-30 TR TR2019/01311T patent/TR201901311T4/tr unknown
- 2010-11-30 SI SI201031681T patent/SI2506857T1/en unknown
- 2010-11-30 NZ NZ750556A patent/NZ750556A/en unknown
- 2010-11-30 RS RS20180509A patent/RS57314B1/sr unknown
- 2010-11-30 PT PT18156531T patent/PT3338765T/pt unknown
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- 2010-11-30 WO PCT/US2010/058457 patent/WO2011068810A1/en not_active Ceased
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2013
- 2013-03-13 US US13/800,501 patent/US10143758B2/en active Active
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2014
- 2014-06-19 US US14/309,387 patent/US20140294940A1/en not_active Abandoned
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2016
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2018
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2019
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- 2019-02-26 US US16/286,423 patent/US20190192690A1/en not_active Abandoned
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2021
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- 2021-12-22 US US17/560,197 patent/US20220111076A1/en not_active Abandoned
- 2021-12-22 US US17/560,195 patent/US20220111075A1/en not_active Abandoned
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2023
- 2023-06-13 US US18/334,330 patent/US20240123084A1/en active Pending
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2024
- 2024-02-02 US US18/431,597 patent/US20240342307A1/en active Pending
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