RU2019120721A - Генная терапия для лечения мукополисахаридоза i типа - Google Patents
Генная терапия для лечения мукополисахаридоза i типа Download PDFInfo
- Publication number
- RU2019120721A RU2019120721A RU2019120721A RU2019120721A RU2019120721A RU 2019120721 A RU2019120721 A RU 2019120721A RU 2019120721 A RU2019120721 A RU 2019120721A RU 2019120721 A RU2019120721 A RU 2019120721A RU 2019120721 A RU2019120721 A RU 2019120721A
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- polynucleotide
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Links
- 102100035028 Alpha-L-iduronidase Human genes 0.000 title claims 11
- 101001019502 Homo sapiens Alpha-L-iduronidase Proteins 0.000 title claims 10
- 206010056886 Mucopolysaccharidosis I Diseases 0.000 title claims 3
- 208000028781 Mucopolysaccharidosis type 1 Diseases 0.000 title 1
- 238000001415 gene therapy Methods 0.000 title 1
- 108091033319 polynucleotide Proteins 0.000 claims 38
- 102000040430 polynucleotide Human genes 0.000 claims 38
- 239000002157 polynucleotide Substances 0.000 claims 38
- 229920001184 polypeptide Polymers 0.000 claims 9
- 108090000765 processed proteins & peptides Proteins 0.000 claims 9
- 102000004196 processed proteins & peptides Human genes 0.000 claims 9
- 210000004027 cell Anatomy 0.000 claims 8
- 210000004962 mammalian cell Anatomy 0.000 claims 8
- 238000000034 method Methods 0.000 claims 7
- 241000713772 Human immunodeficiency virus 1 Species 0.000 claims 6
- 241000700605 Viruses Species 0.000 claims 5
- 241000701022 Cytomegalovirus Species 0.000 claims 3
- 241000725303 Human immunodeficiency virus Species 0.000 claims 3
- 241000713666 Lentivirus Species 0.000 claims 3
- 238000012217 deletion Methods 0.000 claims 3
- 230000037430 deletion Effects 0.000 claims 3
- 238000012856 packing Methods 0.000 claims 3
- 239000008194 pharmaceutical composition Substances 0.000 claims 3
- 230000001177 retroviral effect Effects 0.000 claims 3
- 208000024891 symptom Diseases 0.000 claims 3
- 108091010877 Allograft inflammatory factor 1 Proteins 0.000 claims 2
- 102100040121 Allograft inflammatory factor 1 Human genes 0.000 claims 2
- 241000713704 Bovine immunodeficiency virus Species 0.000 claims 2
- 241000282693 Cercopithecidae Species 0.000 claims 2
- 241000713800 Feline immunodeficiency virus Species 0.000 claims 2
- 101000740205 Homo sapiens Sal-like protein 1 Proteins 0.000 claims 2
- 241000713340 Human immunodeficiency virus 2 Species 0.000 claims 2
- 102100022338 Integrin alpha-M Human genes 0.000 claims 2
- 102100037204 Sal-like protein 1 Human genes 0.000 claims 2
- 102100037029 Transmembrane protein 119 Human genes 0.000 claims 2
- 101710170979 Transmembrane protein 119 Proteins 0.000 claims 2
- 239000012634 fragment Substances 0.000 claims 2
- 230000008488 polyadenylation Effects 0.000 claims 2
- 210000000130 stem cell Anatomy 0.000 claims 2
- 201000004569 Blindness Diseases 0.000 claims 1
- 108010017009 CD11b Antigen Proteins 0.000 claims 1
- 108090000835 CX3C Chemokine Receptor 1 Proteins 0.000 claims 1
- 102100039196 CX3C chemokine receptor 1 Human genes 0.000 claims 1
- 241000283707 Capra Species 0.000 claims 1
- 102000009410 Chemokine receptor Human genes 0.000 claims 1
- 108050000299 Chemokine receptor Proteins 0.000 claims 1
- 206010011878 Deafness Diseases 0.000 claims 1
- 206010066054 Dysmorphism Diseases 0.000 claims 1
- 241000713730 Equine infectious anemia virus Species 0.000 claims 1
- 108091006027 G proteins Proteins 0.000 claims 1
- 102000030782 GTP binding Human genes 0.000 claims 1
- 108091000058 GTP-Binding Proteins 0.000 claims 1
- 208000031886 HIV Infections Diseases 0.000 claims 1
- 102100031573 Hematopoietic progenitor cell antigen CD34 Human genes 0.000 claims 1
- 102100021519 Hemoglobin subunit beta Human genes 0.000 claims 1
- 108091005904 Hemoglobin subunit beta Proteins 0.000 claims 1
- 101000777663 Homo sapiens Hematopoietic progenitor cell antigen CD34 Proteins 0.000 claims 1
- 101001046686 Homo sapiens Integrin alpha-M Proteins 0.000 claims 1
- 101000934372 Homo sapiens Macrosialin Proteins 0.000 claims 1
- 208000015178 Hurler syndrome Diseases 0.000 claims 1
- 108010003381 Iduronidase Proteins 0.000 claims 1
- 206010061598 Immunodeficiency Diseases 0.000 claims 1
- 208000029462 Immunodeficiency disease Diseases 0.000 claims 1
- 102100025136 Macrosialin Human genes 0.000 claims 1
- 108010057466 NF-kappa B Proteins 0.000 claims 1
- 102000003945 NF-kappa B Human genes 0.000 claims 1
- 241000283973 Oryctolagus cuniculus Species 0.000 claims 1
- 102000010292 Peptide Elongation Factor 1 Human genes 0.000 claims 1
- 108010077524 Peptide Elongation Factor 1 Proteins 0.000 claims 1
- 241000714474 Rous sarcoma virus Species 0.000 claims 1
- 206010039491 Sarcoma Diseases 0.000 claims 1
- 108700026226 TATA Box Proteins 0.000 claims 1
- 108091023040 Transcription factor Proteins 0.000 claims 1
- 102000040945 Transcription factor Human genes 0.000 claims 1
- 208000010094 Visna Diseases 0.000 claims 1
- 238000009825 accumulation Methods 0.000 claims 1
- 102000019997 adhesion receptor Human genes 0.000 claims 1
- 108010013985 adhesion receptor Proteins 0.000 claims 1
- 206010003246 arthritis Diseases 0.000 claims 1
- 108010006025 bovine growth hormone Proteins 0.000 claims 1
- 230000003920 cognitive function Effects 0.000 claims 1
- 239000002299 complementary DNA Substances 0.000 claims 1
- 230000003247 decreasing effect Effects 0.000 claims 1
- 230000001934 delay Effects 0.000 claims 1
- 238000011161 development Methods 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 206010014599 encephalitis Diseases 0.000 claims 1
- 239000003623 enhancer Substances 0.000 claims 1
- 230000010370 hearing loss Effects 0.000 claims 1
- 231100000888 hearing loss Toxicity 0.000 claims 1
- 208000016354 hearing loss disease Diseases 0.000 claims 1
- 208000019622 heart disease Diseases 0.000 claims 1
- 210000003958 hematopoietic stem cell Anatomy 0.000 claims 1
- 230000007813 immunodeficiency Effects 0.000 claims 1
- 102000006495 integrins Human genes 0.000 claims 1
- 108010044426 integrins Proteins 0.000 claims 1
- 239000000203 mixture Substances 0.000 claims 1
- 230000004048 modification Effects 0.000 claims 1
- 238000012986 modification Methods 0.000 claims 1
- 239000013642 negative control Substances 0.000 claims 1
- 208000023504 respiratory system disease Diseases 0.000 claims 1
- 208000011580 syndromic disease Diseases 0.000 claims 1
- 230000029812 viral genome replication Effects 0.000 claims 1
- 230000006490 viral transcription Effects 0.000 claims 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
- A61K48/005—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
- A61P11/00—Drugs for disorders of the respiratory system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
-
- 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
-
- 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
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
-
- 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
- A61P27/16—Otologicals
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
-
- 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
- A61P9/00—Drugs for disorders of the cardiovascular system
-
- 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
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
- C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/01076—L-Iduronidase (3.2.1.76)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- 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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/16011—Human Immunodeficiency Virus, HIV
- C12N2740/16041—Use of virus, viral particle or viral elements as a vector
- C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Cardiology (AREA)
- Heart & Thoracic Surgery (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Hospice & Palliative Care (AREA)
- Physical Education & Sports Medicine (AREA)
- Obesity (AREA)
- Psychiatry (AREA)
- Ophthalmology & Optometry (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662430795P | 2016-12-06 | 2016-12-06 | |
| US62/430,795 | 2016-12-06 | ||
| PCT/US2017/064913 WO2018106807A1 (en) | 2016-12-06 | 2017-12-06 | Gene therapy for mucopolysaccharidosis, type i |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| RU2019120721A true RU2019120721A (ru) | 2021-01-11 |
Family
ID=62491308
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2019120721A RU2019120721A (ru) | 2016-12-06 | 2017-12-06 | Генная терапия для лечения мукополисахаридоза i типа |
Country Status (13)
| Country | Link |
|---|---|
| US (1) | US20220339296A1 (enExample) |
| EP (1) | EP3551750A4 (enExample) |
| JP (1) | JP2019536484A (enExample) |
| KR (1) | KR20190089988A (enExample) |
| CN (1) | CN110214182A (enExample) |
| AU (1) | AU2017370662A1 (enExample) |
| BR (1) | BR112019011635A2 (enExample) |
| CA (1) | CA3046079A1 (enExample) |
| IL (1) | IL267057A (enExample) |
| MA (1) | MA47847A (enExample) |
| MX (1) | MX2019006552A (enExample) |
| RU (1) | RU2019120721A (enExample) |
| WO (1) | WO2018106807A1 (enExample) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN109641063B (zh) | 2016-04-20 | 2022-11-15 | 能源环境和技术研究中心O.A., M.P. | 用于增强pklr的基因表达的组合物和方法 |
| US11214789B2 (en) | 2016-05-03 | 2022-01-04 | Flodesign Sonics, Inc. | Concentration and washing of particles with acoustics |
| SG11202002263TA (en) | 2017-10-16 | 2020-04-29 | Centro De Investigaciones Energeticas Medioambientales Y Tecnologicas | Lentiviral vectors for delivery of pklr to treat pyruvate kinase deficiency |
| JP7576754B2 (ja) | 2018-04-11 | 2024-11-01 | スペースクラフト セブン リミテッド ライアビリティ カンパニー | 幹細胞移植のための組成物および方法 |
| MX2021001292A (es) * | 2018-07-30 | 2021-07-15 | Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P | Metodos para la modificacion genica de celulas hematopoyeticas. |
| CN115109790A (zh) * | 2021-03-23 | 2022-09-27 | 北京据德医药科技有限公司 | 一种重组a-L-艾杜糖醛酸普酶及其制备方法 |
| CA3222751A1 (en) * | 2021-06-08 | 2022-12-15 | Touchlight IP Limited | Lentiviral vector |
| WO2024201066A1 (en) * | 2023-03-31 | 2024-10-03 | Dawn Therapeutics Limited | Novel vector |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| FR2872170B1 (fr) * | 2004-06-25 | 2006-11-10 | Centre Nat Rech Scient Cnrse | Lentivirus non interactif et non replicatif, preparation et utilisations |
| US20110294114A1 (en) * | 2009-12-04 | 2011-12-01 | Cincinnati Children's Hospital Medical Center | Optimization of determinants for successful genetic correction of diseases, mediated by hematopoietic stem cells |
| AU2012267512B2 (en) * | 2011-06-10 | 2014-11-20 | Bluebird Bio, Inc. | Gene therapy vectors for adrenoleukodystrophy and adrenomyeloneuropathy |
| US9695443B2 (en) * | 2012-05-25 | 2017-07-04 | Commissariat A L'energie Atomique Et Aux Energies Alternatives | Vector for the selective silencing of a gene in astrocytes |
| WO2014201252A2 (en) * | 2013-06-13 | 2014-12-18 | Shire Human Genetic Therapies, Inc. | Messenger rna based viral production |
| EP3413896B1 (en) * | 2016-02-12 | 2021-03-17 | Bluebird Bio, Inc. | Vcn enhancer compositions and methods of using the same |
-
2017
- 2017-12-06 RU RU2019120721A patent/RU2019120721A/ru not_active Application Discontinuation
- 2017-12-06 CA CA3046079A patent/CA3046079A1/en not_active Abandoned
- 2017-12-06 CN CN201780083351.5A patent/CN110214182A/zh active Pending
- 2017-12-06 MA MA047847A patent/MA47847A/fr unknown
- 2017-12-06 AU AU2017370662A patent/AU2017370662A1/en not_active Abandoned
- 2017-12-06 KR KR1020197019344A patent/KR20190089988A/ko not_active Ceased
- 2017-12-06 WO PCT/US2017/064913 patent/WO2018106807A1/en not_active Ceased
- 2017-12-06 JP JP2019551256A patent/JP2019536484A/ja active Pending
- 2017-12-06 EP EP17877511.0A patent/EP3551750A4/en not_active Withdrawn
- 2017-12-06 BR BR112019011635-4A patent/BR112019011635A2/pt not_active IP Right Cessation
- 2017-12-06 US US16/466,130 patent/US20220339296A1/en not_active Abandoned
- 2017-12-06 MX MX2019006552A patent/MX2019006552A/es unknown
-
2019
- 2019-06-03 IL IL267057A patent/IL267057A/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| EP3551750A4 (en) | 2020-07-22 |
| JP2019536484A (ja) | 2019-12-19 |
| CN110214182A (zh) | 2019-09-06 |
| CA3046079A1 (en) | 2018-06-14 |
| AU2017370662A1 (en) | 2019-06-27 |
| MX2019006552A (es) | 2019-10-15 |
| US20220339296A1 (en) | 2022-10-27 |
| MA47847A (fr) | 2020-01-29 |
| EP3551750A1 (en) | 2019-10-16 |
| IL267057A (en) | 2019-08-29 |
| BR112019011635A2 (pt) | 2019-11-12 |
| WO2018106807A1 (en) | 2018-06-14 |
| KR20190089988A (ko) | 2019-07-31 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| FA93 | Acknowledgement of application withdrawn (no request for examination) |
Effective date: 20201207 |