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PL2158322T3 - Terapia genowa dla lizosomalnych chorób spichrzeniowych - Google Patents

Terapia genowa dla lizosomalnych chorób spichrzeniowych

Info

Publication number
PL2158322T3
PL2158322T3 PL08756596T PL08756596T PL2158322T3 PL 2158322 T3 PL2158322 T3 PL 2158322T3 PL 08756596 T PL08756596 T PL 08756596T PL 08756596 T PL08756596 T PL 08756596T PL 2158322 T3 PL2158322 T3 PL 2158322T3
Authority
PL
Poland
Prior art keywords
gene therapy
lysosomal storage
storage diseases
diseases
lysosomal
Prior art date
Application number
PL08756596T
Other languages
English (en)
Inventor
James Dodge
Seng H Cheng
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 PL2158322T3 publication Critical patent/PL2158322T3/pl

Links

Classifications

    • 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
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Obesity (AREA)
  • Diabetes (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Hematology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
PL08756596T 2007-06-06 2008-06-02 Terapia genowa dla lizosomalnych chorób spichrzeniowych PL2158322T3 (pl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US92494707P 2007-06-06 2007-06-06
EP08756596.6A EP2158322B1 (en) 2007-06-06 2008-06-02 Gene therapy for lysosomal storage diseases
PCT/US2008/065481 WO2008154198A1 (en) 2007-06-06 2008-06-02 Gene therapy for lysosomal storage diseases

Publications (1)

Publication Number Publication Date
PL2158322T3 true PL2158322T3 (pl) 2017-10-31

Family

ID=40130114

Family Applications (2)

Application Number Title Priority Date Filing Date
PL08756596T PL2158322T3 (pl) 2007-06-06 2008-06-02 Terapia genowa dla lizosomalnych chorób spichrzeniowych
PL17168189T PL3252161T3 (pl) 2007-06-06 2008-06-02 Terapia genowa dla lizosomalnych chorób spichrzeniowych

Family Applications After (1)

Application Number Title Priority Date Filing Date
PL17168189T PL3252161T3 (pl) 2007-06-06 2008-06-02 Terapia genowa dla lizosomalnych chorób spichrzeniowych

Country Status (14)

Country Link
US (2) US8796236B2 (pl)
EP (2) EP2158322B1 (pl)
AR (1) AR070000A1 (pl)
CY (1) CY1119295T1 (pl)
DK (1) DK2158322T3 (pl)
ES (2) ES2635726T3 (pl)
HR (1) HRP20171165T1 (pl)
HU (1) HUE035779T2 (pl)
IL (3) IL202522A (pl)
LT (1) LT2158322T (pl)
PL (2) PL2158322T3 (pl)
PT (2) PT3252161T (pl)
SI (1) SI2158322T1 (pl)
WO (1) WO2008154198A1 (pl)

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PL1988823T3 (pl) 2006-02-09 2019-01-31 Genzyme Corporation Powolne dostarczanie dokomorowe
PL2158322T3 (pl) 2007-06-06 2017-10-31 Genzyme Corp Terapia genowa dla lizosomalnych chorób spichrzeniowych
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
EA023938B1 (ru) 2009-04-30 2016-07-29 Оспедале Сан Рафаэлле С.Р.Л. Вектор гена
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
EP3514232A1 (en) 2010-04-23 2019-07-24 University of Massachusetts Cns targeting aav vectors and methods of use thereof
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
AU2012300252B2 (en) 2011-08-31 2017-10-19 St. Jude Children's Research Hospital Methods and compositions to detect the level of lysosomal exocytosis activity and methods of use
BR112015028605A8 (pt) * 2013-05-15 2019-12-24 Univ Minnesota uso de uma composição e uso de um imunossupressor e uma composição
US11078464B2 (en) * 2013-08-30 2021-08-03 Amgen Inc. High titer recombinant AAV vector production in adherent and suspension cells
BR112017007737A2 (pt) 2014-10-21 2018-01-30 Univ Massachusetts variantes de aav recombinantes e usos das mesmas
SG11201703148TA (en) 2014-11-05 2017-05-30 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
EP3285788A4 (en) * 2015-04-23 2018-12-05 University of Massachusetts Modulation of aav vector transgene expression
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
JP7347932B2 (ja) * 2015-12-15 2023-09-20 ジェンザイム・コーポレーション ムコリピドーシスｉｉ型を治療するためのアデノ随伴ウイルスベクター
WO2017127565A1 (en) * 2016-01-19 2017-07-27 The Regents Of The University Of California Methods for the treatment of danon disease and other disorders of autophagy
EP3452104A1 (en) 2016-04-15 2019-03-13 The Trustees of The University of Pennsylvania Gene therapy for treating mucopolysaccharidosis type ii
CA3040483A1 (en) 2016-10-13 2018-04-19 University Of Massachusetts Aav capsid designs
US11219695B2 (en) 2016-10-20 2022-01-11 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of Fabry disease
CN110198712A (zh) * 2016-11-04 2019-09-03 费城儿童医院 用于治疗神经退行性疾病的基因转移组合物、方法和用途
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
WO2018237066A1 (en) 2017-06-20 2018-12-27 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services CODON OPTIMIZED HUMAN NPC1 GENES FOR THE TREATMENT OF C1-TYPE NIEMANN-PICK DISEASE AND ASSOCIATED STATES
US11819539B2 (en) 2017-09-22 2023-11-21 The Trustees Of The University Of Pennsylvania Gene therapy for treating Mucopolysaccharidosis type II
WO2019222314A1 (en) * 2018-05-15 2019-11-21 University Of Massachusetts Raav vectors encoding of lysosomal beta-galactorsidase (glb1) and cathepsin a
MX2021000443A (es) 2018-07-12 2021-05-28 Spacecraft Seven Llc Vectores de terapia génica para el tratamiento de la enfermedad de danon.
CN113438954A (zh) * 2018-10-01 2021-09-24 宾夕法尼亚州大学信托人 可用于治疗gm1神经节苷脂病的组合物
AU2020229772A1 (en) * 2019-02-26 2021-08-26 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of krabbe disease
SG11202111195VA (en) * 2019-04-12 2021-11-29 Encoded Therapeutics Inc Compositions and methods for administration of therapeutics
US11981912B2 (en) 2019-12-10 2024-05-14 Takeda Pharma ceutical Company Limited Adeno associated virus vectors for the treatment of hunter disease
EP4135776A1 (en) * 2020-04-14 2023-02-22 Genethon Vectors for the treatment of acid ceramidase deficiency

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CN105999238A (zh) 2009-08-28 2016-10-12 西奈山医学院 用于治疗酸性鞘磷脂酶缺乏的剂量递增酶替代疗法

Also Published As

Publication number Publication date
PL3252161T3 (pl) 2022-03-07
EP2158322A1 (en) 2010-03-03
IL252898A0 (en) 2017-08-31
EP2158322B1 (en) 2017-05-03
WO2008154198A1 (en) 2008-12-18
US11369693B2 (en) 2022-06-28
HRP20171165T1 (hr) 2017-12-15
US8796236B2 (en) 2014-08-05
PT3252161T (pt) 2022-02-01
IL202522A (en) 2017-10-31
EP2158322A4 (en) 2011-06-01
AR070000A1 (es) 2010-03-10
SI2158322T1 (sl) 2017-10-30
CY1119295T1 (el) 2018-02-14
IL202522A0 (en) 2011-08-01
LT2158322T (lt) 2017-08-25
EP3252161A1 (en) 2017-12-06
ES2905616T3 (es) 2022-04-11
PT2158322T (pt) 2017-08-09
US20150151007A1 (en) 2015-06-04
HUE035779T2 (en) 2018-05-28
IL266734A (en) 2019-07-31
EP3252161B1 (en) 2021-11-17
IL266734B (en) 2021-10-31
DK2158322T3 (en) 2017-08-28
US20100173979A1 (en) 2010-07-08
IL252898B (en) 2019-06-30
ES2635726T3 (es) 2017-10-04

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