AR100419A1 - Vectores de virus adenoasociados para el tratamiento de trastornos de almacenamiento lisosómico - Google Patents

Vectores de virus adenoasociados para el tratamiento de trastornos de almacenamiento lisosómico

Info

Publication number
AR100419A1
AR100419A1 ARP150101471A ARP150101471A AR100419A1 AR 100419 A1 AR100419 A1 AR 100419A1 AR P150101471 A ARP150101471 A AR P150101471A AR P150101471 A ARP150101471 A AR P150101471A AR 100419 A1 AR100419 A1 AR 100419A1
Authority
AR
Argentina
Prior art keywords
treatment
storage disorders
lisosomic
adenoasociated
venues
Prior art date
Application number
ARP150101471A
Other languages
English (en)
Inventor
Bosch Turbert Ftima
Haurigot Mendoa Virginia
Albert Ribera Sanchez Mr
Original Assignee
Esteve Labor Dr
UNIV AUTòNOMA DE BARCELONA
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Esteve Labor Dr, UNIV AUTòNOMA DE BARCELONA filed Critical Esteve Labor Dr
Publication of AR100419A1 publication Critical patent/AR100419A1/es

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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
    • 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
    • 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
    • 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
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary 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
    • A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • 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
    • 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
    • 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
    • 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)
    • C12N9/2474—Hyaluronoglucosaminidase (3.2.1.35), i.e. hyaluronidase
    • 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)
    • C12Y302/0105—Alpha-N-acetylglucosaminidase (3.2.1.50)
    • 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
    • A61K48/0075—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
    • 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
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/10—Vectors comprising a special translation-regulating system regulates levels of translation
    • C12N2840/105—Vectors comprising a special translation-regulating system regulates levels of translation enhancing translation

Abstract

Vectores de virus adenoasociados y composiciones farmacéuticas que contienen a los mismos para el tratamiento de trastornos de almacenamiento lisosómico y, en especial, para el tratamiento de la mucopolisacaridosis Tipo IIIB. Reivindicación 1: Un vector de AAV9 recombinante que contiene un promotor CAG, SEQ ID Nº 4 ligado a una secuencia de nucleótidos que codifica a-N-acetilglucosaminidasa, SEQ ID Nº 1.
ARP150101471A 2014-05-14 2015-05-13 Vectores de virus adenoasociados para el tratamiento de trastornos de almacenamiento lisosómico AR100419A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
EP14382171 2014-05-14

Publications (1)

Publication Number Publication Date
AR100419A1 true AR100419A1 (es) 2016-10-05

Family

ID=50771225

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP150101471A AR100419A1 (es) 2014-05-14 2015-05-13 Vectores de virus adenoasociados para el tratamiento de trastornos de almacenamiento lisosómico

Country Status (22)

Country Link
US (1) US11149285B2 (es)
EP (1) EP3143146B1 (es)
JP (1) JP6634073B2 (es)
KR (1) KR102446169B1 (es)
CN (1) CN107002095B (es)
AR (1) AR100419A1 (es)
AU (1) AU2015261519B2 (es)
BR (1) BR112016025819B1 (es)
CA (1) CA2947468C (es)
ES (1) ES2823948T3 (es)
IL (1) IL248538B (es)
MX (1) MX2016014773A (es)
MY (1) MY183472A (es)
NZ (1) NZ726316A (es)
PH (1) PH12016502248A1 (es)
PT (1) PT3143146T (es)
RU (1) RU2718248C2 (es)
SA (1) SA516380290B1 (es)
SG (1) SG11201609314XA (es)
TW (1) TWI701332B (es)
UA (1) UA123985C2 (es)
WO (1) WO2015173308A1 (es)

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CA3128549A1 (en) 2010-07-12 2012-01-19 Universitat 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
JP2016533741A (ja) 2013-07-29 2016-11-04 ダニスコ・ユーエス・インク 酵素の変異体
CA2960912A1 (en) 2014-09-16 2016-03-24 Universitat Autonoma De Barcelona Adeno-associated viral vectors for the gene therapy of metabolic diseases
EP3015135B1 (en) 2014-10-29 2020-09-09 Noxell Corporation Hair colouration with a cationic or anionic coloured polymer, method and kit thereof
EP3015134B1 (en) 2014-10-29 2020-08-26 Noxell Corporation Hair colouration with a cationic coloured polymer, method and kit thereof
CA2969599A1 (en) 2014-12-05 2016-06-09 Universitat Autonoma De Barcelona Viral vectors for the treatment of diabetes
EP3978614A3 (en) 2015-01-07 2022-07-27 Universitat Autònoma de Barcelona Single-vector gene construct comprising insulin and glucokinase genes
EP3485005A1 (en) 2016-07-12 2019-05-22 The University Of Manchester Gene therapy
CN109863243B (zh) * 2016-09-30 2023-07-28 埃斯蒂文制药股份有限公司 用于治疗粘多糖症的腺相关病毒载体
BR112020010977A2 (pt) * 2017-11-30 2020-11-17 The Trustees Of The University Of Pennsylvania terapia de gene para mucopolissacaridose iiib
CN112513072A (zh) * 2018-04-27 2021-03-16 威斯康星州医药大学股份有限公司 慢病毒载体转化的t-rapa细胞在改善溶酶体贮积症中的应用
CA3101659A1 (en) * 2018-05-30 2019-12-05 Esteve Pharmaceuticals, S.A. Adenoassociated virus vectors for the treatment of mucopolysaccharidoses type iv a
EP3880810A1 (en) * 2018-11-15 2021-09-22 Esteve Pharmaceuticals, S.A. Animal model of mucopolysaccharidoses type iva
ES2933275T3 (es) * 2018-12-18 2023-02-03 Neuway Pharma Gmbh Un sistema de suministro de fármacos novedoso y métodos para proporcionar los mismos
EP3897695A1 (en) * 2018-12-20 2021-10-27 Esteve Pharmaceuticals, S.A. Recombinant vectors for the long term treatment of mucchopolysacharidosis
US20230183741A1 (en) * 2020-05-11 2023-06-15 University Of Florida Research Foundation, Incorporated Disease correction by delivery of aav8 vectors expressing codon optimized naglu
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
CN116234901A (zh) * 2020-08-28 2023-06-06 Jcr制药股份有限公司 α-N-乙酰氨基葡萄糖苷酶的突变体

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BR112014008873A2 (pt) * 2011-10-12 2020-09-15 Synageva Biopharma Corp proteína naglu recombinante humana e usos da mesma
CN102618578A (zh) * 2012-03-16 2012-08-01 中国人民解放军第四军医大学 用于促进汉坦病毒融合蛋白g2s0.7有效提呈的重组腺病毒高表达载体
JP2015516143A (ja) * 2012-04-02 2015-06-08 モデルナ セラピューティクス インコーポレイテッドＭｏｄｅｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． ヒト疾患に関連するタンパク質の産生のための修飾ポリヌクレオチド
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Also Published As

Publication number Publication date
EP3143146A1 (en) 2017-03-22
KR102446169B1 (ko) 2022-09-22
US11149285B2 (en) 2021-10-19
KR20170026358A (ko) 2017-03-08
US20170088859A1 (en) 2017-03-30
EP3143146B1 (en) 2020-05-20
CA2947468A1 (en) 2015-11-19
IL248538A0 (en) 2016-12-29
SG11201609314XA (en) 2016-12-29
JP2017523802A (ja) 2017-08-24
TW201625793A (zh) 2016-07-16
BR112016025819B1 (pt) 2022-08-16
IL248538B (en) 2021-01-31
NZ726316A (en) 2023-07-28
RU2718248C2 (ru) 2020-03-31
MX2016014773A (es) 2017-05-04
SA516380290B1 (ar) 2021-05-17
AU2015261519B2 (en) 2021-08-05
AU2015261519A2 (en) 2017-02-02
TWI701332B (zh) 2020-08-11
MY183472A (en) 2021-02-19
RU2016149206A (ru) 2018-06-14
WO2015173308A1 (en) 2015-11-19
AU2015261519A1 (en) 2016-11-24
UA123985C2 (uk) 2021-07-07
PH12016502248A1 (en) 2017-02-27
JP6634073B2 (ja) 2020-01-22
CN107002095A (zh) 2017-08-01
BR112016025819A2 (pt) 2018-07-03
ES2823948T3 (es) 2021-05-10
CA2947468C (en) 2022-10-25
RU2016149206A3 (es) 2019-04-25
CN107002095B (zh) 2021-03-02
PT3143146T (pt) 2020-08-27

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