LT2158322T - Lizosomų kaupimo ligų genų terapija - Google Patents

Lizosomų kaupimo ligų genų terapija

Info

Publication number
LT2158322T
LT2158322T LTEP08756596.6T LT08756596T LT2158322T LT 2158322 T LT2158322 T LT 2158322T LT 08756596 T LT08756596 T LT 08756596T LT 2158322 T LT2158322 T LT 2158322T
Authority
LT
Lithuania
Prior art keywords
gene therapy
lysosomal storage
storage diseases
diseases
lysosomal
Prior art date
Application number
LTEP08756596.6T
Other languages
English (en)
Inventor
James Dodge
Seng H. Cheng
Original Assignee
Genzyme Corporation
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 Corporation filed Critical Genzyme Corporation
Publication of LT2158322T publication Critical patent/LT2158322T/lt

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
    • 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
    • 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

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)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
LTEP08756596.6T 2007-06-06 2008-06-02 Lizosomų kaupimo ligų genų terapija LT2158322T (lt)

Applications Claiming Priority (2)

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

Publications (1)

Publication Number Publication Date
LT2158322T true LT2158322T (lt) 2017-08-25

Family

ID=40130114

Family Applications (1)

Application Number Title Priority Date Filing Date
LTEP08756596.6T LT2158322T (lt) 2007-06-06 2008-06-02 Lizosomų kaupimo ligų genų terapija

Country Status (14)

Country Link
US (2) US8796236B2 (lt)
EP (2) EP3252161B1 (lt)
AR (1) AR070000A1 (lt)
CY (1) CY1119295T1 (lt)
DK (1) DK2158322T3 (lt)
ES (2) ES2635726T3 (lt)
HR (1) HRP20171165T1 (lt)
HU (1) HUE035779T2 (lt)
IL (3) IL202522A (lt)
LT (1) LT2158322T (lt)
PL (2) PL2158322T3 (lt)
PT (2) PT3252161T (lt)
SI (1) SI2158322T1 (lt)
WO (1) WO2008154198A1 (lt)

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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
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SG10201912761UA (en) 2016-04-15 2020-02-27 The Trustees Of The Univ Of Pennsyvania Gene therapy for treating mucopolysaccharidosis type ii
AU2017341849B2 (en) 2016-10-13 2024-03-21 University Of Massachusetts AAV capsid designs
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CA3041548A1 (en) * 2016-11-04 2018-05-11 The Children's Hospital Of Philadelphia Gene transfer compositions, methods and uses for treating neurodegenerative diseases
KR20190139951A (ko) * 2017-04-14 2019-12-18 리젠엑스바이오 인크. 인간 뉴런 또는 신경교 세포에 의해 생성된 재조합 인간 이두로네이트-2 설파타제(ids)를 이용한 뮤코다당증 ii형의 치료
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CA3068010A1 (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 niemann-pick type c1 deficiency and related conditions
KR102883778B1 (ko) 2017-09-22 2025-11-12 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Ii형 점액다당류증의 치료를 위한 유전자 요법
CN112203697A (zh) 2018-04-13 2021-01-08 马萨诸塞大学 编码氨基己糖苷酶alpha和beta亚基的双顺反子AAV载体及其用途
KR20210019996A (ko) 2018-05-15 2021-02-23 보이저 테라퓨틱스, 인크. 파킨슨병의 치료를 위한 조성물 및 방법
WO2019222314A1 (en) * 2018-05-15 2019-11-21 University Of Massachusetts Raav vectors encoding of lysosomal beta-galactorsidase (glb1) and cathepsin a
KR102918544B1 (ko) 2018-07-12 2026-01-27 로켓 파마슈티컬스, 리미티드 다논병을 치료하기 위한 유전자 요법 벡터
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EP3931337A1 (en) * 2019-02-26 2022-01-05 The Trustees of The University of Pennsylvania Compositions useful in treatment of krabbe disease
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Also Published As

Publication number Publication date
DK2158322T3 (en) 2017-08-28
IL202522A0 (en) 2011-08-01
HUE035779T2 (en) 2018-05-28
CY1119295T1 (el) 2018-02-14
PT3252161T (pt) 2022-02-01
HRP20171165T1 (hr) 2017-12-15
IL266734A (en) 2019-07-31
IL266734B (en) 2021-10-31
IL252898B (en) 2019-06-30
ES2635726T3 (es) 2017-10-04
PL2158322T3 (pl) 2017-10-31
US11369693B2 (en) 2022-06-28
AR070000A1 (es) 2010-03-10
EP3252161A1 (en) 2017-12-06
US8796236B2 (en) 2014-08-05
EP2158322A1 (en) 2010-03-03
PL3252161T3 (pl) 2022-03-07
EP3252161B1 (en) 2021-11-17
IL202522A (en) 2017-10-31
PT2158322T (pt) 2017-08-09
IL252898A0 (en) 2017-08-31
EP2158322A4 (en) 2011-06-01
US20100173979A1 (en) 2010-07-08
ES2905616T3 (es) 2022-04-11
SI2158322T1 (sl) 2017-10-30
WO2008154198A1 (en) 2008-12-18
EP2158322B1 (en) 2017-05-03
US20150151007A1 (en) 2015-06-04

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