EA201890543A1 - Способы и материалы для генной терапии galgt2 - Google Patents
Способы и материалы для генной терапии galgt2Info
- Publication number
- EA201890543A1 EA201890543A1 EA201890543A EA201890543A EA201890543A1 EA 201890543 A1 EA201890543 A1 EA 201890543A1 EA 201890543 A EA201890543 A EA 201890543A EA 201890543 A EA201890543 A EA 201890543A EA 201890543 A1 EA201890543 A1 EA 201890543A1
- Authority
- EA
- Eurasian Patent Office
- Prior art keywords
- methods
- galgt2
- gene therapy
- materials
- raav
- Prior art date
Links
- 102100031504 Beta-1,4 N-acetylgalactosaminyltransferase 2 Human genes 0.000 title abstract 3
- 101000729812 Homo sapiens Beta-1,4 N-acetylgalactosaminyltransferase 2 Proteins 0.000 title abstract 3
- 238000001415 gene therapy Methods 0.000 title abstract 2
- 238000000034 method Methods 0.000 title abstract 2
- 201000006938 muscular dystrophy Diseases 0.000 abstract 2
- 208000018360 neuromuscular disease Diseases 0.000 abstract 2
- 241000702421 Dependoparvovirus Species 0.000 abstract 1
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 abstract 1
- 102100022745 Laminin subunit alpha-2 Human genes 0.000 abstract 1
- 201000006815 congenital muscular dystrophy Diseases 0.000 abstract 1
- 102000040430 polynucleotide Human genes 0.000 abstract 1
- 108091033319 polynucleotide Proteins 0.000 abstract 1
- 239000002157 polynucleotide Substances 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
- 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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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- 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
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/45—Transferases (2)
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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
- A61K48/0075—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 delivery route, e.g. oral, subcutaneous
-
- 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
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- 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
- C07H21/04—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 deoxyribosyl as saccharide radical
-
- 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
- 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
- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
- C12N15/8645—Adeno-associated virus
-
- 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
- C12N9/10—Transferases (2.)
- C12N9/1048—Glycosyltransferases (2.4)
-
- 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
- C12N9/10—Transferases (2.)
- C12N9/1048—Glycosyltransferases (2.4)
- C12N9/1051—Hexosyltransferases (2.4.1)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
- C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y204/00—Glycosyltransferases (2.4)
- C12Y204/01—Hexosyltransferases (2.4.1)
- C12Y204/01165—N-Acetylneuraminylgalactosylglucosylceramide beta-1,4-N-acetylgalactosaminyltransferase (2.4.1.165)
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Biomedical Technology (AREA)
- Microbiology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Immunology (AREA)
- Virology (AREA)
- Plant Pathology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Neurology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Analytical Chemistry (AREA)
- Gastroenterology & Hepatology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562220107P | 2015-09-17 | 2015-09-17 | |
| US201562221068P | 2015-09-20 | 2015-09-20 | |
| US201662301260P | 2016-02-29 | 2016-02-29 | |
| PCT/US2016/052051 WO2017049031A1 (en) | 2015-09-17 | 2016-09-16 | Methods and materials for galgt2 gene therapy |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| EA201890543A1 true EA201890543A1 (ru) | 2018-08-31 |
Family
ID=58289891
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| EA201890543A EA201890543A1 (ru) | 2015-09-17 | 2016-09-16 | Способы и материалы для генной терапии galgt2 |
Country Status (14)
| Country | Link |
|---|---|
| US (3) | US10980897B2 (enExample) |
| EP (1) | EP3350331A4 (enExample) |
| JP (3) | JP7338970B2 (enExample) |
| KR (1) | KR102799163B1 (enExample) |
| CN (1) | CN108136049A (enExample) |
| AU (3) | AU2016323575B2 (enExample) |
| CA (1) | CA2998636A1 (enExample) |
| CO (1) | CO2018003960A2 (enExample) |
| EA (1) | EA201890543A1 (enExample) |
| HK (1) | HK1257519A1 (enExample) |
| IL (2) | IL258005B2 (enExample) |
| MX (2) | MX2018003234A (enExample) |
| SA (1) | SA518391113B1 (enExample) |
| WO (1) | WO2017049031A1 (enExample) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| IL258005B2 (en) | 2015-09-17 | 2025-01-01 | Res Inst Nationwide Childrens Hospital | Methods and materials for gene therapy for beta-4,1-N-acetylgalactosaminyltransferase 2 |
| MA45477A (fr) * | 2016-04-15 | 2019-02-20 | Res Inst Nationwide Childrens Hospital | Administration à vecteurs de virus adéno-associé de microarn-29 et micro-dystrophine pour traiter la dystrophie musculaire |
| JP7162021B2 (ja) | 2017-03-17 | 2022-10-27 | ニューカッスル ユニバーシティ | 筋ジストロフィーを治療するためのマイクロジストロフィン断片のアデノ随伴ウイルスベクター送達 |
| WO2018191666A1 (en) * | 2017-04-14 | 2018-10-18 | Regenxbio Inc. | Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2 sulfatase (ids) produced by human neural or glial cells |
| US11534501B2 (en) | 2017-10-18 | 2022-12-27 | Research Institute At Nationwide Children's Hospital | Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy |
| EP3697916A4 (en) | 2017-10-20 | 2021-08-11 | Research Institute at Nationwide Children's Hospital | METHODS AND MATERIALS FOR NT-3 GENE THERAPY |
| MY208145A (en) | 2018-06-18 | 2025-04-18 | Res Inst Nationwide Childrens Hospital | Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy |
| EP3807309A1 (en) * | 2018-06-18 | 2021-04-21 | Research Institute at Nationwide Children's Hospital | Recombinant adeno-associated virus products and methods for treating dystroglycanopathies and laminin-deficient muscular dystrophies |
| KR20220105643A (ko) * | 2019-10-25 | 2022-07-27 | 오덴테스 테라퓨틱스, 인크. | 글리코겐 축적 장애를 치료하기 위한 조성물 및 방법 |
| US20220411821A1 (en) * | 2019-10-28 | 2022-12-29 | University Of Florida Research Foundation, Incorporated | Gene therapy vectors |
| CN116323925A (zh) * | 2020-08-07 | 2023-06-23 | 詹森生物科技公司 | 高度纯化的病毒颗粒的制剂 |
| EP4230196A1 (en) | 2022-02-21 | 2023-08-23 | Som Innovation Biotech, S.A. | Compounds for use in the treatment of dystrophinopathies |
Family Cites Families (23)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| ATE272123T1 (de) | 1993-11-09 | 2004-08-15 | Ohio Med College | Stabile zellinie, die in der lage ist, das replikationsgen des adenoassoziertenvirus zu exprimieren |
| EP0733103B1 (en) | 1993-11-09 | 2004-03-03 | Targeted Genetics Corporation | Generation of high titers of recombinant aav vectors |
| US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
| US5856152A (en) | 1994-10-28 | 1999-01-05 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV vector and methods of use therefor |
| EP0796339A1 (en) | 1994-12-06 | 1997-09-24 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant aav vectors |
| FR2737730B1 (fr) | 1995-08-10 | 1997-09-05 | Pasteur Merieux Serums Vacc | Procede de purification de virus par chromatographie |
| JPH11511326A (ja) | 1995-08-30 | 1999-10-05 | ジエンザイム コーポレイション | アデノウィルスおよびａａｖの精製 |
| US6632670B1 (en) | 1995-09-08 | 2003-10-14 | Genzyme Corporation | AAV vectors for gene therapy |
| US5910434A (en) | 1995-12-15 | 1999-06-08 | Systemix, Inc. | Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant |
| ATE465267T1 (de) | 1996-09-06 | 2010-05-15 | Univ Pennsylvania | Rekombinante aav zur herstellung eines medikaments für die gentherapie von muskelzellen |
| WO1999011764A2 (en) | 1997-09-05 | 1999-03-11 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of recombinant aav vectors |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6258595B1 (en) | 1999-03-18 | 2001-07-10 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
| CA2394976A1 (en) | 1999-12-13 | 2001-06-14 | Lexicon Genetics Incorporated | Novel human transferase proteins and polynucleotides encoding the same |
| CA2406743A1 (en) | 2000-04-28 | 2001-11-08 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids |
| EP1390490B1 (en) * | 2001-05-24 | 2009-04-15 | Genzyme Corporation | Muscle-specific expression vectors |
| US20060194265A1 (en) * | 2001-10-23 | 2006-08-31 | Morris David W | Novel therapeutic targets in cancer |
| WO2008048986A2 (en) * | 2006-10-17 | 2008-04-24 | Children's Hospital Medical Center | Gene array technique for predicting response in inflammatory bowel diseases |
| WO2013078316A1 (en) | 2011-11-23 | 2013-05-30 | Nationwide Children's Hospital, Inc. | Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides |
| JP2015503924A (ja) * | 2012-01-05 | 2015-02-05 | ハダシット メディカル リサーチ サービシーズ アンド ディベロップメント リミテッド | 遺伝子導入のための方法及び組成物 |
| BR112014020325A2 (pt) | 2012-02-17 | 2017-08-08 | Childrens Hospital Philadelphia | composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos |
| IL258005B2 (en) | 2015-09-17 | 2025-01-01 | Res Inst Nationwide Childrens Hospital | Methods and materials for gene therapy for beta-4,1-N-acetylgalactosaminyltransferase 2 |
-
2016
- 2016-09-16 IL IL258005A patent/IL258005B2/en unknown
- 2016-09-16 JP JP2018513504A patent/JP7338970B2/ja active Active
- 2016-09-16 US US15/759,474 patent/US10980897B2/en active Active
- 2016-09-16 HK HK18116463.4A patent/HK1257519A1/zh unknown
- 2016-09-16 WO PCT/US2016/052051 patent/WO2017049031A1/en not_active Ceased
- 2016-09-16 CN CN201680054261.9A patent/CN108136049A/zh active Pending
- 2016-09-16 EP EP16847351.0A patent/EP3350331A4/en active Pending
- 2016-09-16 IL IL314884A patent/IL314884A/en unknown
- 2016-09-16 CA CA2998636A patent/CA2998636A1/en active Pending
- 2016-09-16 KR KR1020187010628A patent/KR102799163B1/ko active Active
- 2016-09-16 MX MX2018003234A patent/MX2018003234A/es unknown
- 2016-09-16 AU AU2016323575A patent/AU2016323575B2/en active Active
- 2016-09-16 EA EA201890543A patent/EA201890543A1/ru unknown
-
2018
- 2018-03-13 SA SA518391113A patent/SA518391113B1/ar unknown
- 2018-03-14 MX MX2023013509A patent/MX2023013509A/es unknown
- 2018-04-13 CO CONC2018/0003960A patent/CO2018003960A2/es unknown
-
2021
- 2021-03-16 US US17/203,232 patent/US12121595B2/en active Active
- 2021-11-05 JP JP2021180893A patent/JP2022010079A/ja active Pending
-
2023
- 2023-01-31 AU AU2023200499A patent/AU2023200499B2/en active Active
-
2024
- 2024-05-30 JP JP2024087848A patent/JP2024109934A/ja active Pending
- 2024-10-03 US US18/905,388 patent/US20250025578A1/en active Pending
-
2025
- 2025-08-06 AU AU2025213610A patent/AU2025213610A1/en active Pending
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