BR112015028605A8 - uso de uma composição e uso de um imunossupressor e uma composição - Google Patents
uso de uma composição e uso de um imunossupressor e uma composição Download PDFInfo
- Publication number
- BR112015028605A8 BR112015028605A8 BR112015028605A BR112015028605A BR112015028605A8 BR 112015028605 A8 BR112015028605 A8 BR 112015028605A8 BR 112015028605 A BR112015028605 A BR 112015028605A BR 112015028605 A BR112015028605 A BR 112015028605A BR 112015028605 A8 BR112015028605 A8 BR 112015028605A8
- Authority
- BR
- Brazil
- Prior art keywords
- disease
- composition
- inhibiting
- treating
- preventing
- Prior art date
Links
- 229960003444 immunosuppressant agent Drugs 0.000 title 1
- 230000001861 immunosuppressant effect Effects 0.000 title 1
- 239000003018 immunosuppressive agent Substances 0.000 title 1
- 201000010099 disease Diseases 0.000 abstract 3
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 3
- 241000124008 Mammalia Species 0.000 abstract 2
- 208000015114 central nervous system disease Diseases 0.000 abstract 2
- 230000002401 inhibitory effect Effects 0.000 abstract 2
- 238000000034 method Methods 0.000 abstract 2
- 108090000623 proteins and genes Proteins 0.000 abstract 2
- 208000024891 symptom Diseases 0.000 abstract 2
- 238000000185 intracerebroventricular administration Methods 0.000 abstract 1
- 238000007913 intrathecal administration Methods 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
- 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
- A61K38/46—Hydrolases (3)
- A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
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- 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
-
- 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
- A61K9/0085—Brain, e.g. brain implants; Spinal cord
-
- 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
- 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
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- 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
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- 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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
-
- 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
-
- 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
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- 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
- C12N2750/14171—Demonstrated in vivo effect
-
- 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
- C12N2830/007—Vector systems having a special element relevant for transcription cell cycle specific enhancer/promoter combination
-
- 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)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Epidemiology (AREA)
- Molecular Biology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Immunology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Diabetes (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Orthopedic Medicine & Surgery (AREA)
Abstract
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201361823757P | 2013-05-15 | 2013-05-15 | |
PCT/US2014/038209 WO2014186579A1 (en) | 2013-05-15 | 2014-05-15 | Adeno-associated virus mediated gene transfer to the central nervous system |
Publications (2)
Publication Number | Publication Date |
---|---|
BR112015028605A2 BR112015028605A2 (pt) | 2017-07-25 |
BR112015028605A8 true BR112015028605A8 (pt) | 2019-12-24 |
Family
ID=51898870
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR112015028605A BR112015028605A8 (pt) | 2013-05-15 | 2014-05-15 | uso de uma composição e uso de um imunossupressor e uma composição |
Country Status (13)
Country | Link |
---|---|
US (11) | US9827295B2 (pt) |
EP (2) | EP2996475A4 (pt) |
JP (4) | JP2016523835A (pt) |
KR (3) | KR20240093939A (pt) |
CN (3) | CN115120745A (pt) |
AU (3) | AU2014265417B2 (pt) |
BR (1) | BR112015028605A8 (pt) |
CA (2) | CA2912678C (pt) |
HK (2) | HK1222093A1 (pt) |
MX (2) | MX2015015560A (pt) |
RU (1) | RU2692251C2 (pt) |
SG (2) | SG11201509419QA (pt) |
WO (1) | WO2014186579A1 (pt) |
Families Citing this family (55)
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EP3747998A1 (en) | 2013-03-15 | 2020-12-09 | The Trustees of the University of Pennsylvania | Compositions for treating mpsi |
CN115120745A (zh) * | 2013-05-15 | 2022-09-30 | 明尼苏达大学董事会 | 腺相关病毒介导的基因向中枢神经***转移 |
WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
BR112017009497A2 (pt) | 2014-11-05 | 2018-02-06 | Voyager Therapeutics, Inc. | polinucleotídeos de aadc para o tratamento da doença de parkinson |
KR20230145206A (ko) | 2014-11-14 | 2023-10-17 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
BR112017010087A2 (pt) | 2014-11-14 | 2018-06-05 | Voyager Therapeutics, Inc. | composições e métodos para tratar esclerose lateral amiotrófica (ela) |
EP3230441A4 (en) | 2014-12-12 | 2018-10-03 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scaav |
CA2970730A1 (en) * | 2015-01-16 | 2016-07-21 | Voyager Therapeutics, Inc. | Central nervous system targeting polynucleotides |
EP3285788A4 (en) | 2015-04-23 | 2018-12-05 | University of Massachusetts | Modulation of aav vector transgene expression |
GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
US20180289839A1 (en) | 2015-05-15 | 2018-10-11 | Regents Of The University Of Minnesota | Intranasal therapeutic delivery of adeno-associated virus to central nervous system |
WO2016196507A1 (en) * | 2015-05-29 | 2016-12-08 | University Of Iowa Research Foundation | Methods of delivery of transgenes for treating brain diseases |
CN116650668A (zh) * | 2015-10-23 | 2023-08-29 | 衣阿华大学研究基金会 | 使用基因疗法以推迟疾病发生和发展同时提供认知保护来治疗神经变性疾病的方法 |
EP3370705B1 (en) * | 2015-11-05 | 2022-03-09 | The General Hospital Corporation | Intrathecal delivery of nucleic acid sequences encoding abcd1 for treatment of adrenomyeloneuropathy |
CN109152847A (zh) * | 2016-01-15 | 2019-01-04 | 桑格摩生物治疗股份有限公司 | 用于治疗神经疾病的方法和组合物 |
SG11201806270XA (en) * | 2016-02-03 | 2018-08-30 | Univ Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
MX2018009426A (es) * | 2016-02-22 | 2018-12-19 | Univ North Carolina Chapel Hill | Vector aav-idua para el tratamiento de ceguera asociada con mucopolisacaridosis i (mps i). |
US11185555B2 (en) | 2016-04-11 | 2021-11-30 | Noah James Harrison | Method to kill pathogenic microbes in a patient |
IL305449A (en) * | 2016-04-15 | 2023-10-01 | Univ Pennsylvania | Gene therapy for the treatment of type II mucositis |
EP3448987A4 (en) | 2016-04-29 | 2020-05-27 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR THE TREATMENT OF DISEASES |
MX2018014154A (es) | 2016-05-18 | 2019-05-06 | Voyager Therapeutics Inc | Polinucleotidos moduladores. |
RU2764587C2 (ru) | 2016-05-18 | 2022-01-18 | Вояджер Терапьютикс, Инк. | Способы и композиции для лечения хореи гентингтона |
EP3831281A1 (en) | 2016-08-30 | 2021-06-09 | The Regents of The University of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
US11111271B2 (en) | 2016-09-28 | 2021-09-07 | Cohbar, Inc. | Therapeutic peptides |
US20190358303A1 (en) * | 2017-01-31 | 2019-11-28 | Regenxbio Inc. | Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua) |
US11117930B2 (en) | 2017-02-23 | 2021-09-14 | Adrx, Inc. | Peptide inhibitors of transcription factor aggregation |
TW202315946A (zh) | 2017-04-03 | 2023-04-16 | 美商編碼製藥公司 | 組織選擇性轉基因表現 |
BR112019021569A2 (pt) * | 2017-04-14 | 2020-05-12 | Regenxbio Inc. | Precursor de iduronato-2-sulfatase humana recombinante glicosilada (ids), método para tratar um sujeito humano diagnosticado com mucopolissacaridose tipo ii (mps ii) |
EP3619308A4 (en) | 2017-05-05 | 2021-01-27 | Voyager Therapeutics, Inc. | COMPOSITIONS AND METHODS OF TREATMENT FOR HUNTINGTON'S MORBUS |
AU2018261790A1 (en) | 2017-05-05 | 2019-11-28 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (ALS) |
BR112019025888A2 (pt) | 2017-06-07 | 2020-06-30 | Regeneron Pharmaceuticals, Inc. | polinucleotídeo que codifica uma proteína terapêutica multidomínio, vetor de terapia gênica, proteína terapêutica multidomínio recombinante, método de expressão, métodos para reduzir o acúmulo de glicogênio em um tecido em um paciente em necessidade, para reduzir o acúmulo de glicogênio em um tecido em um paciente em necessidade epara tratar a deficiência enzimática em um paciente em necessidade e/ou tolerar o paciente à enzima para a qual é deficiente, anticorpo anti-cd63 ou fragmento de ligação a antígeno, e, composição farmacêutica |
CA3066077A1 (en) | 2017-06-07 | 2018-12-13 | Adrx, Inc. | Tau aggregation inhibitors |
JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
BR112020000063A2 (pt) | 2017-07-06 | 2020-07-14 | The Trustees Of The University Of Pennsylvania | terapia gênica mediada por aav9 para tratamento de mucopolissacaridose tipo i |
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CA3076036A1 (en) | 2017-09-22 | 2019-03-28 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type ii |
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JP2021525245A (ja) * | 2018-05-22 | 2021-09-24 | ザ ブリガム アンド ウィメンズ ホスピタル インコーポレイテッドThe Brigham and Women’s Hospital, Inc. | アルツハイマー病のための遺伝子治療 |
EP3856913A4 (en) | 2018-09-26 | 2022-10-26 | California Institute Of Technology | ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR TARGETED GENE THERAPY |
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CA3134841A1 (en) * | 2019-04-10 | 2020-10-15 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
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