MX2016014773A - Vectores de virus adenoasociados para el tratamiento de trastornos de almacenamiento lisosomico. - Google Patents

Vectores de virus adenoasociados para el tratamiento de trastornos de almacenamiento lisosomico.

Info

Publication number
MX2016014773A
MX2016014773A MX2016014773A MX2016014773A MX2016014773A MX 2016014773 A MX2016014773 A MX 2016014773A MX 2016014773 A MX2016014773 A MX 2016014773A MX 2016014773 A MX2016014773 A MX 2016014773A MX 2016014773 A MX2016014773 A MX 2016014773A
Authority
MX
Mexico
Prior art keywords
treatment
virus vectors
lysosomal storage
storage disorders
adenoassociated virus
Prior art date
Application number
MX2016014773A
Other languages
English (en)
Inventor
Fàtima Bosch Tubert M
Virginia HAURIGOT MENDOÇA M
Ribera Sanche Albert
Original Assignee
Esteve Labor Dr
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 filed Critical Esteve Labor Dr
Publication of MX2016014773A publication Critical patent/MX2016014773A/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

La presente invención proporciona nuevos 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.
MX2016014773A 2014-05-14 2015-05-13 Vectores de virus adenoasociados para el tratamiento de trastornos de almacenamiento lisosomico. MX2016014773A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP14382171 2014-05-14
PCT/EP2015/060604 WO2015173308A1 (en) 2014-05-14 2015-05-13 Adenoassociated virus vectors for the treatment of lysosomal storage disorders

Publications (1)

Publication Number Publication Date
MX2016014773A true MX2016014773A (es) 2017-05-04

Family

ID=50771225

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2016014773A MX2016014773A (es) 2014-05-14 2015-05-13 Vectores de virus adenoasociados para el tratamiento de trastornos de almacenamiento lisosomico.

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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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
CN105916981A (zh) 2013-07-29 2016-08-31 丹尼斯科美国公司 变体酶
US10973931B2 (en) 2014-09-16 2021-04-13 Universitat Autònoma De Barcelona Adeno-associated viral vectors for the gene therapy of metabolic diseases
EP3015134B1 (en) 2014-10-29 2020-08-26 Noxell Corporation Hair colouration with a cationic coloured polymer, method and kit thereof
EP3015135B1 (en) 2014-10-29 2020-09-09 Noxell Corporation Hair colouration with a cationic or anionic coloured polymer, method and kit thereof
US11085055B2 (en) 2014-12-05 2021-08-10 Universitat Autonoma De Barcelona Viral vectors for the treatment of diabetes
ES2900973T3 (es) 2015-01-07 2022-03-21 UNIV AUTòNOMA DE BARCELONA Constructo genético de vector individual que comprende genes de insulina y glucoquinasa
WO2018011572A1 (en) * 2016-07-12 2018-01-18 The University Of Manchester Gene therapy
PT3519569T (pt) 2016-09-30 2021-03-04 UNIV AUTòNOMA DE BARCELONA Vetores de vírus adenoassociados para o tratamento de mucopolissacaridoses
JP7384797B2 (ja) * 2017-11-30 2023-11-21 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ｉｉｉｂ型のための遺伝子療法
EP4285921A3 (en) * 2018-04-27 2024-03-06 The Medical College of Wisconsin, Inc. Use of lentivector-transduced t-rapa cells for amelioration of lysosomal storage disorders
KR20210027266A (ko) * 2018-05-30 2021-03-10 에스테베 파마슈티칼스 에스에이 뮤코다당질축적증 ｉｖ ａ형의 치료를 위한 아데노관련 바이러스 벡터
EP3880810A1 (en) * 2018-11-15 2021-09-22 Esteve Pharmaceuticals, S.A. Animal model of mucopolysaccharidoses type iva
JP6884432B2 (ja) * 2018-12-18 2021-06-09 ノイウェイ ファルマ ゲーエムベーハー 新規薬物送達システムおよびそれを提供する方法
US20220042043A1 (en) * 2018-12-20 2022-02-10 Esteve Pharmaceuticals, S.A. Recombinant vectors for the long term treatment of mucchopolysacharidosis
WO2021231491A1 (en) * 2020-05-11 2021-11-18 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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Also Published As

Publication number Publication date
NZ726316A (en) 2023-07-28
KR102446169B1 (ko) 2022-09-22
RU2016149206A (ru) 2018-06-14
CA2947468C (en) 2022-10-25
RU2016149206A3 (es) 2019-04-25
PT3143146T (pt) 2020-08-27
AU2015261519A1 (en) 2016-11-24
ES2823948T3 (es) 2021-05-10
CN107002095B (zh) 2021-03-02
RU2718248C2 (ru) 2020-03-31
KR20170026358A (ko) 2017-03-08
BR112016025819A2 (pt) 2018-07-03
IL248538B (en) 2021-01-31
SA516380290B1 (ar) 2021-05-17
US11149285B2 (en) 2021-10-19
AR100419A1 (es) 2016-10-05
IL248538A0 (en) 2016-12-29
TW201625793A (zh) 2016-07-16
BR112016025819B1 (pt) 2022-08-16
UA123985C2 (uk) 2021-07-07
EP3143146A1 (en) 2017-03-22
JP2017523802A (ja) 2017-08-24
PH12016502248A1 (en) 2017-02-27
WO2015173308A1 (en) 2015-11-19
SG11201609314XA (en) 2016-12-29
JP6634073B2 (ja) 2020-01-22
CN107002095A (zh) 2017-08-01
TWI701332B (zh) 2020-08-11
MY183472A (en) 2021-02-19
AU2015261519A2 (en) 2017-02-02
AU2015261519B2 (en) 2021-08-05
US20170088859A1 (en) 2017-03-30
EP3143146B1 (en) 2020-05-20
CA2947468A1 (en) 2015-11-19

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