[go: up one dir, main page]

MX2017015725A - Vectores virales adenoasociados para el tratamiento de mucopolisacaridosis. - Google Patents

Vectores virales adenoasociados para el tratamiento de mucopolisacaridosis.

Info

Publication number
MX2017015725A
MX2017015725A MX2017015725A MX2017015725A MX2017015725A MX 2017015725 A MX2017015725 A MX 2017015725A MX 2017015725 A MX2017015725 A MX 2017015725A MX 2017015725 A MX2017015725 A MX 2017015725A MX 2017015725 A MX2017015725 A MX 2017015725A
Authority
MX
Mexico
Prior art keywords
treatment
mucopolysaccharidoses
virus vectors
adenoassociated virus
adenoassociated
Prior art date
Application number
MX2017015725A
Other languages
English (en)
Other versions
MX386615B (es
Inventor
Fátima Bosch Tubert Maria
Areba Haurigot Virginia
Motas Mallol Sandra
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 MX2017015725A publication Critical patent/MX2017015725A/es
Publication of MX386615B publication Critical patent/MX386615B/es

Links

Classifications

    • 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)
  • Zoology (AREA)
  • Wood Science & Technology (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)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)

Abstract

La presente invención proporciona nuevos vectores derivados de virus adeno-asociados y composiciones farmacéuticas que los contienen para el tratamiento de trastornos de almacenamiento lisosómico y especialmente, para el tratamiento de mucopolisacaridosis Tipo II.
MX2017015725A 2015-06-05 2016-06-03 Vectores virales adenoasociados para el tratamiento de mucopolisacaridosis. MX386615B (es)

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 (2)

Publication Number Publication Date
MX2017015725A true MX2017015725A (es) 2018-08-15
MX386615B MX386615B (es) 2025-03-19

Family

ID=53398018

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2017015725A MX386615B (es) 2015-06-05 2016-06-03 Vectores virales adenoasociados para el tratamiento de mucopolisacaridosis.

Country Status (21)

Country Link
US (1) US10617771B2 (es)
EP (2) EP3101125A1 (es)
JP (1) JP6850736B2 (es)
KR (1) KR102609858B1 (es)
CN (1) CN108138154B (es)
AU (1) AU2016273343B2 (es)
CA (1) CA2988201C (es)
CL (1) CL2017003096A1 (es)
CO (1) CO2017012562A2 (es)
ES (1) ES2790834T3 (es)
IL (1) IL255939B (es)
MX (1) MX386615B (es)
MY (1) MY184586A (es)
NZ (1) NZ738018A (es)
PH (1) PH12017502192B1 (es)
PT (1) PT3303576T (es)
RU (1) RU2744593C2 (es)
TN (1) TN2017000506A1 (es)
UA (1) UA125373C2 (es)
WO (1) WO2016193431A1 (es)
ZA (1) ZA201708039B (es)

Families Citing this family (21)

* Cited by examiner, † Cited by third party
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
EP3227449B1 (en) 2014-12-05 2020-07-15 Universitat Autònoma de Barcelona Viral vectors for the treatment of diabetes
WO2016110518A1 (en) 2015-01-07 2016-07-14 Universitat Autònoma De Barcelona Single-vector gene construct comprising insulin and glucokinase genes
SG11201808176TA (en) 2016-04-15 2018-10-30 The Trustees Of The Univ Of Pennsyvania Gene therapy for treating mucopolysaccharidosis type ii
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)
CA3076036A1 (en) * 2017-09-22 2019-03-28 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type ii
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
KR20210040984A (ko) * 2018-07-27 2021-04-14 리젠엑스바이오 인크. 점액다당류증 iva의 치료
JP7462234B2 (ja) * 2018-11-15 2024-04-05 エステベ ファーマシューティカルズ，ソシエダッド アノニマ ムコ多糖症ｉｖａ型の動物モデル
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
EP3914720B9 (en) 2019-07-10 2023-09-27 Masonic Medical Research Laboratory Vgll4 with ucp-1 cis-regulatory element and method of use thereof
IL293684A (en) * 2019-12-10 2022-08-01 Takeda Pharmaceuticals Co Adeno associated virus vectors for the treatment of hunter disease
BR112022019182A2 (pt) * 2020-04-06 2022-11-29 Homology Medicines Inc Composições de vírus adeno-associadas para transferência do gene ids e métodos de uso da mesma
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
WO2022173605A2 (en) 2021-02-10 2022-08-18 Regenxbio Inc. Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids)
WO2022221421A2 (en) * 2021-04-13 2022-10-20 Capsida, Inc. Aav compositions with high brain expression for treating mucopolysaccharidosis ii
US20240350653A1 (en) * 2021-08-18 2024-10-24 Joint-Stock Company "Generium" Targeted delivery of therapeutic enzymes

Family Cites Families (11)

* Cited by examiner, † Cited by third party
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
PL3219795T3 (pl) * 2008-01-18 2024-09-16 Biomarin Pharmaceutical Inc. Wytwarzanie aktywnych wysoce fosforylowanych ludzkich enzymów sulfatazy lizosomalnej i ich zastosowania
US8834874B2 (en) * 2009-10-09 2014-09-16 Armagen Technologies, Inc. Methods and compositions for increasing iduronate 2-sulfatase activity in the CNS
WO2011108451A1 (ja) * 2010-03-01 2011-09-09 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
US20130236442A1 (en) * 2010-11-12 2013-09-12 Green Cross Corporation Iduronate-2-sulfatase and use thereof
WO2012101671A1 (en) * 2011-01-25 2012-08-02 Jcr Pharmaceuticals Co., Ltd. Method for production of human recombinant iduronate 2-sulfatase
EP3460064B8 (en) * 2011-04-03 2024-03-20 The General Hospital Corporation d/b/a Massachusetts General Hospital Efficient protein expression in vivo using modified rna (mod-rna)
AU2013243949A1 (en) * 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of biologics and proteins associated with human disease
US9150841B2 (en) * 2012-06-29 2015-10-06 Shire Human Genetic Therapies, Inc. Cells for producing recombinant iduronate-2-sulfatase
AU2015219339B2 (en) * 2014-02-19 2020-03-05 Bioasis Technologies Inc. P97-IDS fusion proteins

Also Published As

Publication number Publication date
WO2016193431A1 (en) 2016-12-08
UA125373C2 (uk) 2022-03-02
PH12017502192B1 (en) 2022-09-02
AU2016273343B2 (en) 2022-04-28
CN108138154B (zh) 2022-01-18
MY184586A (en) 2021-04-06
CA2988201A1 (en) 2016-12-08
CN108138154A (zh) 2018-06-08
PT3303576T (pt) 2020-05-08
RU2744593C2 (ru) 2021-03-11
PH12017502192A1 (en) 2018-06-11
TN2017000506A1 (en) 2019-04-12
RU2017142006A (ru) 2019-07-09
US20180169272A1 (en) 2018-06-21
US10617771B2 (en) 2020-04-14
ZA201708039B (en) 2018-11-28
ES2790834T3 (es) 2020-10-29
NZ738018A (en) 2025-03-28
KR20180012843A (ko) 2018-02-06
CO2017012562A2 (es) 2018-02-28
AU2016273343A1 (en) 2017-12-21
EP3303576B1 (en) 2020-02-12
CA2988201C (en) 2024-01-16
BR112017025892A2 (pt) 2018-08-14
EP3101125A1 (en) 2016-12-07
JP2018517414A (ja) 2018-07-05
CL2017003096A1 (es) 2018-08-10
IL255939A (en) 2018-01-31
KR102609858B1 (ko) 2023-12-05
JP6850736B2 (ja) 2021-03-31
EP3303576A1 (en) 2018-04-11
RU2017142006A3 (es) 2020-02-03
IL255939B (en) 2021-05-31
MX386615B (es) 2025-03-19

Similar Documents

Publication Publication Date Title
MX2017015725A (es) Vectores virales adenoasociados para el tratamiento de mucopolisacaridosis.
MX2016014773A (es) Vectores de virus adenoasociados para el tratamiento de trastornos de almacenamiento lisosomico.
AR109752A1 (es) Vectores de virus adenoasociados para el tratamiento de mucopolisacaridosis
ECSP19003773A (es) Compuestos heterocíclicos como inmunomoduladores
GT201600233A (es) Compuestos y composiciones como agonistas del receptor tipo toll 7
CL2018001685A1 (es) Compuestos heterociclicos como inmuno moduladores.
CL2016001889A1 (es) (2s)-n-[(1s)-1-ciano-2-feniletil]-1,4-oxazepan-2-carboxamidas como inhibidores de dipeptidil peptidasa 1.
CL2015001733A1 (es) Compuestos derivados de lactamas fusionadas de arilo y heteroarilo, moduladores de ezh2; composicion farmaceutica que los comprende; uso del compuesto para el tratamiento del cancer.
MX378273B (es) Compuestos activos hacia bromodominios.
CL2015000295A1 (es) Compuestos derivados de alquilpirimidina; composicion farmaceutica que los comprende; uso para el tratamiento de infecciones víricas y otras enfermedades.
CL2014001793A1 (es) Compuestos derivados de 1,3,5-triazinas sustituidas y sus sales, como inhibidores de la idh2 mutante; composicion farmaceutica que los comprende; y su uso para el tratamiento del cancer.
CL2016003212A1 (es) Derivados de indolín-2-ona o pirrolo-piridín-2-ona.
ECSP18049420A (es) Composiciones novedosas, usos y métodos para hacerlas
MX2016014308A (es) Compuestos y composiciones como agonistas del receptor tipo toll 7.
UY36654A (es) Azabenzimidazoles con propiedades moduladoras del receptor ampa y composiciones farmacéuticas que los contienen
CL2014002257A1 (es) Compuestos derivados de 6-alquinilo piridinas, como mimeticos smac; composicion farmaceutica que los comprende; y su uso para el tratamiento y/o prevencion del cancer.
ECSP14014778A (es) Composiciones derivadas de la quitosana
CL2016000148A1 (es) Nuevos compuestos isoindolina o isoquinolina, un proceso para su preparación y composiciones farmacéuticas que los contienen.
MX2017000183A (es) Amidas de benzoxazinona como moduladores del receptor de mineralocorticoides.
CL2018003338A1 (es) Formulaciones inyectables fisiológicamente balanceadas de fosnetupitant.
BR112017024908A2 (pt) usos terapêuticos da 4-cloroquinurenina
MX384447B (es) Composiciones farmacéuticas y usos dirigidos a los trastornos del almacenamiento lisosomal.
CO2020015942A2 (es) Vectores de virus adenoasociados para el tratamiento de mucopolisacaridosis tipo iv a
BR112017004552A2 (pt) composições farmacêuticas
MX2017003949A (es) Derivados peptidicos novedosos y sus usos.