EA201690588A1 - Доставка мрнк в цнс и ее применение - Google Patents
Доставка мрнк в цнс и ее применениеInfo
- Publication number
- EA201690588A1 EA201690588A1 EA201690588A EA201690588A EA201690588A1 EA 201690588 A1 EA201690588 A1 EA 201690588A1 EA 201690588 A EA201690588 A EA 201690588A EA 201690588 A EA201690588 A EA 201690588A EA 201690588 A1 EA201690588 A1 EA 201690588A1
- Authority
- EA
- Eurasian Patent Office
- Prior art keywords
- mrna
- cns
- delivery
- compositions
- methods
- Prior art date
Links
- 108020004999 messenger RNA Proteins 0.000 abstract 4
- 239000000203 mixture Substances 0.000 abstract 4
- 210000003169 central nervous system Anatomy 0.000 abstract 3
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 2
- 238000000034 method Methods 0.000 abstract 2
- 210000004556 brain Anatomy 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000035475 disorder Diseases 0.000 abstract 1
- 230000003834 intracellular effect Effects 0.000 abstract 1
- 238000007913 intrathecal administration Methods 0.000 abstract 1
- 239000002502 liposome Substances 0.000 abstract 1
- 210000002569 neuron Anatomy 0.000 abstract 1
- 102000004169 proteins and genes Human genes 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
- 210000000278 spinal cord Anatomy 0.000 abstract 1
- 208000002320 spinal muscular atrophy Diseases 0.000 abstract 1
Classifications
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- 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
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
-
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- 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
- 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
-
- 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
-
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- Epidemiology (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Neurology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Neurosurgery (AREA)
- Immunology (AREA)
- Marine Sciences & Fisheries (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Dispersion Chemistry (AREA)
- Physical Education & Sports Medicine (AREA)
- Hospice & Palliative Care (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Psychiatry (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361894246P | 2013-10-22 | 2013-10-22 | |
| US201462020161P | 2014-07-02 | 2014-07-02 | |
| PCT/US2014/061786 WO2015061461A1 (en) | 2013-10-22 | 2014-10-22 | Cns delivery of mrna and uses thereof |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| EA201690588A1 true EA201690588A1 (ru) | 2016-09-30 |
Family
ID=51844900
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| EA201690588A EA201690588A1 (ru) | 2013-10-22 | 2014-10-22 | Доставка мрнк в цнс и ее применение |
Country Status (10)
| Country | Link |
|---|---|
| US (4) | US20150110857A1 (https=) |
| EP (2) | EP4036241A1 (https=) |
| JP (6) | JP6893785B2 (https=) |
| CN (1) | CN105658800A (https=) |
| AU (3) | AU2014340149B2 (https=) |
| BR (1) | BR112016008832A2 (https=) |
| CA (1) | CA2928040A1 (https=) |
| EA (1) | EA201690588A1 (https=) |
| MX (1) | MX2016005236A (https=) |
| WO (1) | WO2015061461A1 (https=) |
Families Citing this family (70)
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|---|---|---|---|---|
| ES2646630T3 (es) | 2008-11-07 | 2017-12-14 | Massachusetts Institute Of Technology | Lipidoides aminoalcohólicos y usos de los mismos |
| HUE042177T2 (hu) | 2009-12-01 | 2019-06-28 | Translate Bio Inc | Szteroidszármazék mRNS szállítására humán genetikai betegségekben |
| US9238716B2 (en) | 2011-03-28 | 2016-01-19 | Massachusetts Institute Of Technology | Conjugated lipomers and uses thereof |
| EP3354644A1 (en) | 2011-06-08 | 2018-08-01 | Translate Bio, Inc. | Cleavable lipids |
| PL3586861T3 (pl) | 2011-06-08 | 2022-05-23 | Translate Bio, Inc. | Kompozycje nanocząstek lipidowych i sposoby dostarczania mrna |
| KR102451116B1 (ko) | 2011-10-27 | 2022-10-06 | 메사추세츠 인스티튜트 오브 테크놀로지 | 약물 캡슐화 마이크로스피어를 형성할 수 있는, ｎ-말단 상에 관능화된 아미노산 유도체 |
| EP3536787A1 (en) | 2012-06-08 | 2019-09-11 | Translate Bio, Inc. | Nuclease resistant polynucleotides and uses thereof |
| KR20210122917A (ko) | 2013-03-14 | 2021-10-12 | 샤이어 휴먼 지네틱 테라피즈 인크. | Cftr mrna 조성물 및 관련 방법 및 사용 |
| DK2970456T3 (da) | 2013-03-14 | 2021-07-05 | Translate Bio Inc | Fremgangsmåder og sammensætninger til levering af mrna-kodede antistoffer |
| PL3467108T3 (pl) | 2013-03-14 | 2024-09-30 | Translate Bio, Inc. | Sposoby oczyszczania informacyjnego rna |
| ES2707966T3 (es) | 2013-10-22 | 2019-04-08 | Translate Bio Inc | Terapia de ARNm para la deficiencia en síntesis de argininosuccinato |
| EA034103B1 (ru) | 2013-10-22 | 2019-12-27 | Транслейт Био, Инк. | СПОСОБ ЛЕЧЕНИЯ ФЕНИЛКЕТОНУРИИ С ПРИМЕНЕНИЕМ мРНК |
| PT3134506T (pt) | 2014-04-25 | 2019-10-31 | Translate Bio Inc | Métodos de purificação de rna mensageiro |
| JP6384863B2 (ja) * | 2014-10-24 | 2018-09-05 | 国立大学法人北海道大学 | 脂質膜構造体 |
| ES2949540T3 (es) * | 2015-06-19 | 2023-09-29 | Massachusetts Inst Technology | 2,5-piperazinadionas sustituidas con alquenilo y su uso en composiciones para suministrar un agente a un sujeto o una célula |
| BR112018011975A2 (pt) * | 2015-12-14 | 2018-12-11 | Univ Pennsylvania | composições úteis no tratamento de atrofia muscular espinhal |
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| WO2018089801A1 (en) * | 2016-11-10 | 2018-05-17 | Translate Bio, Inc. | Improved process of preparing mrna-loaded lipid nanoparticles |
| KR102313431B1 (ko) | 2016-11-21 | 2021-10-18 | 나노스트링 테크놀로지스, 인크. | 화학적 조성물 및 이것을 사용하는 방법 |
| ES2973040T3 (es) | 2017-01-27 | 2024-06-18 | Methodist Hospital | Plataforma de estructura de núcleo/cáscara para inmunoterapia |
| WO2018157133A1 (en) | 2017-02-27 | 2018-08-30 | Translate Bio, Inc. | Methods for purification of messenger rna |
| EP3585892B8 (en) | 2017-02-27 | 2022-07-13 | Translate Bio, Inc. | Methods for purification of messenger rna |
| EA201991747A1 (ru) | 2017-02-27 | 2020-06-04 | Транслейт Био, Инк. | НОВАЯ КОДОН-ОПТИМИЗИРОВАННАЯ мРНК CFTR |
| JOP20190200A1 (ar) * | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
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| BR102017016440A2 (pt) * | 2017-07-31 | 2019-03-19 | Universidade Federal Do Rio Grande Do Sul | Composição para terapia gênica do sistema nervoso central, processo de obtenção e uso da mesma |
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| EP4629972A1 (en) * | 2022-12-07 | 2025-10-15 | The University of British Columbia | Compositions and methods for peptide or protein delivery to the delivery to the central nervous system |
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- 2014-10-22 US US14/521,168 patent/US20150110857A1/en not_active Abandoned
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| US20210196633A1 (en) | 2021-07-01 |
| US10780052B2 (en) | 2020-09-22 |
| MX2016005236A (es) | 2016-08-12 |
| AU2021201220B2 (en) | 2024-05-02 |
| AU2021201220A1 (en) | 2021-03-11 |
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| CA2928040A1 (en) | 2015-04-30 |
| JP2023041951A (ja) | 2023-03-24 |
| US12016954B2 (en) | 2024-06-25 |
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| EP4036241A1 (en) | 2022-08-03 |
| JP2019065047A (ja) | 2019-04-25 |
| JP6893785B2 (ja) | 2021-06-23 |
| EP3060671B1 (en) | 2021-12-29 |
| US20180028445A1 (en) | 2018-02-01 |
| US20150110857A1 (en) | 2015-04-23 |
| JP2016539916A (ja) | 2016-12-22 |
| EP4036241A9 (en) | 2023-03-22 |
| AU2014340149B2 (en) | 2020-12-24 |
| AU2024205165A1 (en) | 2024-08-15 |
| EP3060671A1 (en) | 2016-08-31 |
| JP2025013512A (ja) | 2025-01-24 |
| BR112016008832A2 (pt) | 2017-10-03 |
| US20240408013A1 (en) | 2024-12-12 |
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