PT2158322T - Terapia genética para doenças do armazenamento lisossomal - Google Patents

Terapia genética para doenças do armazenamento lisossomal

Info

Publication number
PT2158322T
PT2158322T PT87565966T PT08756596T PT2158322T PT 2158322 T PT2158322 T PT 2158322T PT 87565966 T PT87565966 T PT 87565966T PT 08756596 T PT08756596 T PT 08756596T PT 2158322 T PT2158322 T PT 2158322T
Authority
PT
Portugal
Prior art keywords
gene therapy
lysosomal storage
storage diseases
diseases
lysosomal
Prior art date
Application number
PT87565966T
Other languages
English (en)
Inventor
Dodge James
H Cheng Seng
Original Assignee
Genzyme Corp
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 Genzyme Corp filed Critical Genzyme Corp
Publication of PT2158322T publication Critical patent/PT2158322T/pt

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
    • A61K48/0075—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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • 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
    • 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
    • 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)
    • C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
PT87565966T 2007-06-06 2008-06-02 Terapia genética para doenças do armazenamento lisossomal PT2158322T (pt)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US92494707P 2007-06-06 2007-06-06

Publications (1)

Publication Number Publication Date
PT2158322T true PT2158322T (pt) 2017-08-09

Family

ID=40130114

Family Applications (2)

Application Number Title Priority Date Filing Date
PT87565966T PT2158322T (pt) 2007-06-06 2008-06-02 Terapia genética para doenças do armazenamento lisossomal
PT171681893T PT3252161T (pt) 2007-06-06 2008-06-02 Terapia genética para doenças do armazenamento lisossomal

Family Applications After (1)

Application Number Title Priority Date Filing Date
PT171681893T PT3252161T (pt) 2007-06-06 2008-06-02 Terapia genética para doenças do armazenamento lisossomal

Country Status (14)

Country Link
US (2) US8796236B2 (pt)
EP (2) EP3252161B1 (pt)
AR (1) AR070000A1 (pt)
CY (1) CY1119295T1 (pt)
DK (1) DK2158322T3 (pt)
ES (2) ES2635726T3 (pt)
HR (1) HRP20171165T1 (pt)
HU (1) HUE035779T2 (pt)
IL (3) IL202522A (pt)
LT (1) LT2158322T (pt)
PL (2) PL3252161T3 (pt)
PT (2) PT2158322T (pt)
SI (1) SI2158322T1 (pt)
WO (1) WO2008154198A1 (pt)

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ES2635726T3 (es) 2007-06-06 2017-10-04 Genzyme Corporation Terapia génica para enfermedades de almacenamiento lisosómico
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
US8242086B2 (en) * 2008-11-12 2012-08-14 Duke University Methods and compositions for treating disorders caused by a deficiency in a gene product of a CLN gene
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US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
DK2826860T3 (en) 2010-04-23 2018-12-03 Univ Massachusetts CNS targeting AAV vectors and methods for their use
WO2011133874A1 (en) 2010-04-23 2011-10-27 University Of Massachusetts Multicistronic expression constructs
US9849195B2 (en) 2011-03-31 2017-12-26 University Of Iowa Research Foundation Methods and compositions for treating brain diseases
US20130090374A1 (en) * 2011-04-20 2013-04-11 Miguel Sena-Esteves Methods for the treatment of tay-sachs disease, sandhoff disease, and gm1-gangliosidosis
US20130039888A1 (en) 2011-06-08 2013-02-14 Nationwide Children's Hospital Inc. Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders
LT2751279T (lt) 2011-08-31 2017-12-11 St. Jude Children`S Research Hospital Būdai ir kompozicijos lizosominės egzocitozės aktyvumo lygio nustatymui ir panaudojimo būdai
CN115120746A (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系统转移
US11078464B2 (en) 2013-08-30 2021-08-03 Amgen Inc. High titer recombinant AAV vector production in adherent and suspension cells
CN107073051B (zh) 2014-10-21 2021-08-24 马萨诸塞大学 重组aav变体及其用途
MX2017005834A (es) 2014-11-05 2017-11-17 Voyager Therapeutics Inc Polinucleotidos aad para el tratamiento de la enfermedad de parkinson.
CA3019315A1 (en) * 2015-04-23 2016-10-27 University Of Massachusetts Modulation of aav vector transgene expression
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
RU2742612C2 (ru) * 2015-12-15 2021-02-09 Джензим Корпорейшн Векторы на основе аденоассоциированного вируса для лечения муколипидоза типа ii
AU2017209189B2 (en) * 2016-01-19 2024-03-28 The Regents Of The University Of California Methods for the treatment of Danon disease and other disorders of autophagy
JP7171439B2 (ja) 2016-04-15 2022-11-15 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型を処置するための遺伝子療法
US11578340B2 (en) 2016-10-13 2023-02-14 University Of Massachusetts AAV capsid designs
CA3039673A1 (en) 2016-10-20 2018-04-26 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of fabry disease
WO2018085688A1 (en) * 2016-11-04 2018-05-11 The Children's Hospital Of Philadelphia Gene transfer compositions, methods and uses for treating neurodegenerative diseases
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
US11819539B2 (en) 2017-09-22 2023-11-21 The Trustees Of The University Of Pennsylvania Gene therapy for treating Mucopolysaccharidosis type II
EP3794131A4 (en) * 2018-05-15 2022-01-26 University of Massachusetts RECOMBINANT AAV VECTORS CODING FOR LYSOSOMIAL BETA-GALACTOSIDASE (GLB1) AND CATHEPSIN A
WO2020014523A1 (en) 2018-07-12 2020-01-16 Rocket Pharmaceuticals, Ltd. Gene therapy vectors for treatment of danon disease
KR20220007601A (ko) * 2019-04-12 2022-01-18 엔코디드 테라퓨틱스, 인크. 치료제 투여를 위한 조성물 및 방법
CN115379863A (zh) * 2020-04-14 2022-11-22 吉尼松公司 用于治疗酸性神经酰胺酶缺乏症的载体

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ATE525092T1 (de) * 2005-05-02 2011-10-15 Genzyme Corp Gentherapie für neurometabolische erkrankungen
ES2700048T3 (es) * 2006-02-08 2019-02-13 Genzyme Corp Terapia génica par la enfermedad de Niemann-Pick tipo A
EP1988823B1 (en) * 2006-02-09 2018-08-15 Genzyme Corporation Slow intraventricular delivery
ES2635726T3 (es) 2007-06-06 2017-10-04 Genzyme Corporation Terapia génica para enfermedades de almacenamiento lisosómico
EP3482767B1 (en) 2009-08-28 2021-10-06 Icahn School of Medicine at Mount Sinai Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency

Also Published As

Publication number Publication date
PT3252161T (pt) 2022-02-01
IL202522A (en) 2017-10-31
CY1119295T1 (el) 2018-02-14
PL3252161T3 (pl) 2022-03-07
HUE035779T2 (en) 2018-05-28
US8796236B2 (en) 2014-08-05
EP3252161A1 (en) 2017-12-06
IL266734A (en) 2019-07-31
EP2158322A4 (en) 2011-06-01
WO2008154198A1 (en) 2008-12-18
IL266734B (en) 2021-10-31
SI2158322T1 (sl) 2017-10-30
HRP20171165T1 (hr) 2017-12-15
ES2905616T3 (es) 2022-04-11
US20100173979A1 (en) 2010-07-08
EP2158322B1 (en) 2017-05-03
LT2158322T (lt) 2017-08-25
IL202522A0 (en) 2011-08-01
DK2158322T3 (en) 2017-08-28
ES2635726T3 (es) 2017-10-04
PL2158322T3 (pl) 2017-10-31
AR070000A1 (es) 2010-03-10
IL252898B (en) 2019-06-30
US20150151007A1 (en) 2015-06-04
EP3252161B1 (en) 2021-11-17
EP2158322A1 (en) 2010-03-03
IL252898A0 (en) 2017-08-31
US11369693B2 (en) 2022-06-28

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