CO2017012562A2 - Vectores virales adenoasociados que codifican para la enzima iduronato-2-sulfatasa - Google Patents
Vectores virales adenoasociados que codifican para la enzima iduronato-2-sulfatasaInfo
- Publication number
- CO2017012562A2 CO2017012562A2 CONC2017/0012562A CO2017012562A CO2017012562A2 CO 2017012562 A2 CO2017012562 A2 CO 2017012562A2 CO 2017012562 A CO2017012562 A CO 2017012562A CO 2017012562 A2 CO2017012562 A2 CO 2017012562A2
- Authority
- CO
- Colombia
- Prior art keywords
- iduronate
- adeno
- sulfatase enzyme
- viral vectors
- vectors encoding
- Prior art date
Links
- 102100029199 Iduronate 2-sulfatase Human genes 0.000 title abstract 2
- 101710096421 Iduronate 2-sulfatase Proteins 0.000 title abstract 2
- 239000013603 viral vector Substances 0.000 title 1
- 241000702421 Dependoparvovirus Species 0.000 abstract 1
- 108091028043 Nucleic acid sequence Proteins 0.000 abstract 1
- 239000008194 pharmaceutical composition Substances 0.000 abstract 1
- 239000013598 vector 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
- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
-
- 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)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
-
- 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
-
- 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
- A61K48/0016—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 nucleic acid is delivered as a 'naked' nucleic acid, i.e. not combined with an entity such as a cationic lipid
-
- 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
- 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
- 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
- C12N15/63—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
-
- 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
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/06—Sulfuric ester hydrolases (3.1.6)
- C12Y301/06013—Iduronate-2-sulfatase (3.1.6.13)
-
- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Biomedical Technology (AREA)
- Epidemiology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Virology (AREA)
- Dermatology (AREA)
- Enzymes And Modification Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP15382297.8A EP3101125A1 (en) | 2015-06-05 | 2015-06-05 | Adenoassociated virus vectors for the treatment of mucopolysaccharidoses |
| PCT/EP2016/062655 WO2016193431A1 (en) | 2015-06-05 | 2016-06-03 | Adenoassociated virus vectors for the treatment of mucopolysaccharidoses |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CO2017012562A2 true CO2017012562A2 (es) | 2018-02-28 |
Family
ID=53398018
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CONC2017/0012562A CO2017012562A2 (es) | 2015-06-05 | 2017-12-05 | Vectores virales adenoasociados que codifican para la enzima iduronato-2-sulfatasa |
Country Status (21)
Families Citing this family (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9309534B2 (en) | 2010-07-12 | 2016-04-12 | Universidad Autonoma De Barcelona | Gene therapy composition for use in diabetes treatment |
| EP2692868A1 (en) | 2012-08-02 | 2014-02-05 | Universitat Autònoma De Barcelona | Adeno-associated viral (AAV) vectors useful for transducing adipose tissue |
| CA2960912A1 (en) | 2014-09-16 | 2016-03-24 | Universitat Autonoma De Barcelona | Adeno-associated viral vectors for the gene therapy of metabolic diseases |
| ES2824457T3 (es) | 2014-12-05 | 2021-05-12 | UNIV AUTòNOMA DE BARCELONA | Vectores virales para el tratamiento de la diabetes |
| EP3242945B1 (en) | 2015-01-07 | 2021-09-01 | Universitat Autònoma de Barcelona | Single-vector gene construct comprising insulin and glucokinase genes |
| CA3019427A1 (en) | 2016-04-15 | 2017-10-19 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type ii |
| UY37679A (es) * | 2017-04-14 | 2018-11-30 | Regenxbio Inc | Tratamiento de mucopolisacaridosis ii con iduronato-2-sulfatasa (ids) humana recombinante producida por células neurales o gliales humanas |
| JP7449223B2 (ja) | 2017-09-22 | 2024-03-13 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | ムコ多糖症ｉｉ型を治療するための遺伝子療法 |
| CN108715867A (zh) * | 2018-05-31 | 2018-10-30 | 深圳市免疫基因治疗研究院 | 一种Sanfilippo B综合症慢病毒载体、慢病毒及其制备方法和应用 |
| CN108795985A (zh) * | 2018-05-31 | 2018-11-13 | 深圳市免疫基因治疗研究院 | 一种黏多糖贮积症慢病毒载体、慢病毒及其制备方法和应用 |
| EP3829619A4 (en) * | 2018-07-27 | 2022-05-18 | REGENXBIO Inc. | TREATMENT OF IVA MUCOPOLYSACCHARIDOSIS |
| WO2020099548A1 (en) * | 2018-11-15 | 2020-05-22 | Esteve Pharmaceuticals, S.A. | Animal model of mucopolysaccharidoses type iva |
| TW202039856A (zh) * | 2019-03-28 | 2020-11-01 | 西班牙商艾斯提夫製藥股份有限公司 | 製造重組病毒載體之方法 |
| EP4252783A3 (en) | 2019-07-10 | 2023-11-15 | Masonic Medical Research Laboratory | Vgll4 with ucp-1 cis-regulatory element and method of use thereof |
| BR112022011332A2 (pt) * | 2019-12-10 | 2022-08-23 | Takeda Pharmaceuticals Co | Vetor de vírus adeno-associado recombinante, vírus adeno-associado recombinante, e, método para tratar um indivíduo com síndrome de hunter |
| WO2021207077A1 (en) * | 2020-04-06 | 2021-10-14 | Homology Medicines, Inc. | Adeno-associated virus compositions for ids gene transfer and methods of use thereof |
| CN111718947B (zh) * | 2020-06-18 | 2022-08-23 | 舒泰神（北京）生物制药股份有限公司 | 用于治疗ⅲa或ⅲb型粘多糖贮积症的腺相关病毒载体及用途 |
| CN114480454B (zh) * | 2020-10-27 | 2024-03-29 | 华东理工大学 | 用于治疗粘多糖贮积症ⅱ型的腺相关病毒载体、药物及其应用 |
| JP2024505739A (ja) | 2021-02-10 | 2024-02-07 | レジェンクスバイオ インコーポレーテッド | 組換えヒトイズロン酸－２－スルファターゼ（ｉｄｓ）によるムコ多糖症ｉｉの治療 |
| WO2022221421A2 (en) * | 2021-04-13 | 2022-10-20 | Capsida, Inc. | Aav compositions with high brain expression for treating mucopolysaccharidosis ii |
| CA3228179A1 (en) * | 2021-08-18 | 2023-02-23 | Joint-Stock Company "Generium" | Targeted delivery of therapeutic enzymes |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR100762945B1 (ko) * | 2004-09-08 | 2007-10-04 | 진동규 | 동물 세포주를 이용한 이듀로네이트-설파타제의 발현 방법및 그 발현을 위한 세포주 |
| PL2245145T3 (pl) * | 2008-01-18 | 2017-07-31 | Biomarin Pharmaceutical Inc. | Wytwarzanie aktywnych wysoce fosforylowanych ludzkich enzymów sulfatazy lizosomalnej i ich zastosowania |
| HRP20190690T1 (hr) * | 2009-10-09 | 2019-06-28 | Armagen, Inc. | Postupci i pripravci za povećanje aktiviteta iduronat-2-sulfataze u centralnom nervnom sustavu |
| JPWO2011108451A1 (ja) * | 2010-03-01 | 2013-06-27 | 日本ケミカルリサーチ株式会社 | 遺伝子ノックアウト細胞を用いた組換え体リソソーム酵素の製造方法 |
| EP2394667A1 (en) * | 2010-06-10 | 2011-12-14 | Laboratorios Del Dr. Esteve, S.A. | Vectors and sequences for the treatment of diseases |
| KR101535791B1 (ko) * | 2010-11-12 | 2015-07-10 | 주식회사 녹십자 | 개량형 이듀로네이트-2-설파타제 및 이의 용도 |
| WO2012101671A1 (en) * | 2011-01-25 | 2012-08-02 | Jcr Pharmaceuticals Co., Ltd. | Method for production of human recombinant iduronate 2-sulfatase |
| WO2012138453A1 (en) * | 2011-04-03 | 2012-10-11 | The General Hospital Corporation | Efficient protein expression in vivo using modified rna (mod-rna) |
| CN108949772A (zh) * | 2012-04-02 | 2018-12-07 | 现代泰克斯公司 | 用于产生与人类疾病相关的生物制剂和蛋白质的修饰多核苷酸 |
| US9150841B2 (en) * | 2012-06-29 | 2015-10-06 | Shire Human Genetic Therapies, Inc. | Cells for producing recombinant iduronate-2-sulfatase |
| WO2015126729A1 (en) * | 2014-02-19 | 2015-08-27 | Bioasis Technologies, Inc. | P97-ids fusion proteins |
-
2015
- 2015-06-05 EP EP15382297.8A patent/EP3101125A1/en not_active Withdrawn
-
2016
- 2016-06-03 MY MYPI2017704615A patent/MY184586A/en unknown
- 2016-06-03 RU RU2017142006A patent/RU2744593C2/ru active
- 2016-06-03 CN CN201680032931.7A patent/CN108138154B/zh active Active
- 2016-06-03 TN TNP/2017/000506A patent/TN2017000506A1/en unknown
- 2016-06-03 WO PCT/EP2016/062655 patent/WO2016193431A1/en not_active Ceased
- 2016-06-03 PT PT167307164T patent/PT3303576T/pt unknown
- 2016-06-03 CA CA2988201A patent/CA2988201C/en active Active
- 2016-06-03 NZ NZ738018A patent/NZ738018A/en unknown
- 2016-06-03 US US15/578,366 patent/US10617771B2/en active Active
- 2016-06-03 ES ES16730716T patent/ES2790834T3/es active Active
- 2016-06-03 MX MX2017015725A patent/MX386615B/es unknown
- 2016-06-03 JP JP2017563119A patent/JP6850736B2/ja active Active
- 2016-06-03 PH PH1/2017/502192A patent/PH12017502192B1/en unknown
- 2016-06-03 AU AU2016273343A patent/AU2016273343B2/en not_active Ceased
- 2016-06-03 KR KR1020187000048A patent/KR102609858B1/ko active Active
- 2016-06-03 UA UAA201711584A patent/UA125373C2/uk unknown
- 2016-06-03 EP EP16730716.4A patent/EP3303576B1/en active Active
-
2017
- 2017-11-27 IL IL255939A patent/IL255939B/en active IP Right Grant
- 2017-11-27 ZA ZA2017/08039A patent/ZA201708039B/en unknown
- 2017-12-04 CL CL2017003096A patent/CL2017003096A1/es unknown
- 2017-12-05 CO CONC2017/0012562A patent/CO2017012562A2/es unknown
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