CY1119295T1 - Γονιδιακη θεραπεια για ασθενειες λυσοσωμικης αποθhκευσης - Google Patents

Γονιδιακη θεραπεια για ασθενειες λυσοσωμικης αποθhκευσης

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Publication number
CY1119295T1
CY1119295T1 CY20171100836T CY171100836T CY1119295T1 CY 1119295 T1 CY1119295 T1 CY 1119295T1 CY 20171100836 T CY20171100836 T CY 20171100836T CY 171100836 T CY171100836 T CY 171100836T CY 1119295 T1 CY1119295 T1 CY 1119295T1
Authority
CY
Cyprus
Prior art keywords
subject
transgene
brain
storage diseases
lysosomic
Prior art date
Application number
CY20171100836T
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 CY1119295T1 publication Critical patent/CY1119295T1/el

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    • 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)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (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)

Abstract

Αυτή η περιγραφή παρέχει μεθόδους και συνθέσεις για την θεραπεία ασθενειών λυσοσωμικής αποθήκευσης σε ένα υποκείμενο. Σε μία άποψη της εφεύρεσης, ένα διαγονιδιακό προϊόν χορηγείται σε ένα υποκείμενο με την χορήγηση ενός ανασυνδυασμένου νευροτροφικού ιικού φορέα που περιέχει το διαγονίδιο στον εγκέφαλο. Ο ιικός φορέας παραδίδει το διαγονίδιο σε μια περιοχή του εγκεφάλου που είναι ευάλωτη σε μόλυνση από τον ιό και η οποία εκφράζει το κωδικοποιημένο προϊόν ανασυνδυασμένου ιικού γονιδίου. Παρέχονται επίσης συνθέσεις για την παράδοση ενός διαγονιδιακού προϊόντος σε ένα υποκείμενο με την χορήγηση ενός ανασυνδυασμένου νευροτροφικού ιικού φορέα που περιέχει το διαγονίδιο στον εγκέφαλο του υποκειμένου. Το διαγονιδιακό προϊόν μπορεί να είναι οποιοδήποτε που είναι ανεπαρκές σε μία ασθένεια λυσοσωμικής αποθήκευσης.
CY20171100836T 2007-06-06 2017-08-03 Γονιδιακη θεραπεια για ασθενειες λυσοσωμικης αποθhκευσης CY1119295T1 (el)

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
CY1119295T1 true CY1119295T1 (el) 2018-02-14

Family

ID=40130114

Family Applications (1)

Application Number Title Priority Date Filing Date
CY20171100836T CY1119295T1 (el) 2007-06-06 2017-08-03 Γονιδιακη θεραπεια για ασθενειες λυσοσωμικης αποθhκευσης

Country Status (14)

Country Link
US (2) US8796236B2 (el)
EP (2) EP3252161B1 (el)
AR (1) AR070000A1 (el)
CY (1) CY1119295T1 (el)
DK (1) DK2158322T3 (el)
ES (2) ES2635726T3 (el)
HR (1) HRP20171165T1 (el)
HU (1) HUE035779T2 (el)
IL (3) IL202522A (el)
LT (1) LT2158322T (el)
PL (2) PL3252161T3 (el)
PT (2) PT3252161T (el)
SI (1) SI2158322T1 (el)
WO (1) WO2008154198A1 (el)

Families Citing this family (32)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AR059089A1 (es) * 2006-01-20 2008-03-12 Genzyme Corp Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal
PL1988823T3 (pl) 2006-02-09 2019-01-31 Genzyme Corporation Powolne dostarczanie dokomorowe
SI2158322T1 (sl) 2007-06-06 2017-10-30 Genzyme Corporation Genska terapija za bolezni lizosomskega shranjevanja
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
PT2424571T (pt) 2009-04-30 2020-05-06 Ospedale San Raffaele Srl Vetor génico
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
CA2833905C (en) 2010-04-23 2019-09-10 University Of Massachusetts Multicistronic expression constructs
CA3066596A1 (en) 2010-04-23 2011-10-27 University Of Massachusetts Cns targeting aav vectors and methods of use thereof
CA2832151C (en) 2011-03-31 2021-06-15 University Of Iowa Research Foundation Methods and compositions for treating brain diseases
WO2012145646A1 (en) * 2011-04-20 2012-10-26 Miguel Sena-Esteves Methods for the treatment of tay-sachs disease, sandhoff disease, and gmi-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
EP2751279B1 (en) 2011-08-31 2017-10-04 St. Jude Children's Research Hospital Methods and compositions to detect the level of lysosomal exocytosis activity and methods of use
CA2912678C (en) * 2013-05-15 2023-10-10 Regents Of The University Of Minnesota Adeno-associated virus mediated gene transfer to the central nervous system
WO2015031686A1 (en) 2013-08-30 2015-03-05 Amgen Inc. High titer recombinant aav vector production in adherent and suspension cells
RU2738421C2 (ru) 2014-10-21 2020-12-14 Юниверсити Оф Массачусетс Варианты рекомбинантных aav и их применения
RU2020109343A (ru) 2014-11-05 2020-03-17 Вояджер Терапьютикс, Инк. Полинуклеотиды aadc для лечения болезни паркинсона
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
CA3008268A1 (en) * 2015-12-15 2017-06-22 Genzyme Corporation Adeno-associated viral vectors for treating mucolipidosis type ii
CN109069671B (zh) * 2016-01-19 2022-10-18 加利福尼亚大学董事会 Danon病和其它自噬障碍的治疗方法
KR20230125339A (ko) 2016-04-15 2023-08-29 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Ii형 점액다당류증의 치료를 위한 유전자 요법
KR20190075964A (ko) 2016-10-13 2019-07-01 유니버시티 오브 매사추세츠 Aav 캡시드 설계
BR112019007210A2 (pt) 2016-10-20 2019-08-13 Sangamo Therapeutics Inc métodos e composições para o tratamento da doença de fabry
AU2017355502B2 (en) * 2016-11-04 2023-08-31 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
BR112020005249A2 (pt) 2017-09-22 2020-09-24 The Trustees Of The University Of Pennsylvania terapia de gene para tratar mucopolissacaridose tipo ii
EP3794131A4 (en) * 2018-05-15 2022-01-26 University of Massachusetts RECOMBINANT AAV VECTORS CODING FOR LYSOSOMIAL BETA-GALACTOSIDASE (GLB1) AND CATHEPSIN A
SG11202100022SA (en) 2018-07-12 2021-01-28 Rocket Pharmaceuticals Ltd Gene therapy vectors for treatment of danon disease
BR112021020421A2 (pt) * 2019-04-12 2021-12-21 Encoded Therapeutics Inc Composições e métodos para administração de componentes terapêuticos
KR20220112283A (ko) 2019-12-10 2022-08-10 다케다 야쿠힌 고교 가부시키가이샤 Hunter 질환 치료용 아데노-연합된 바이러스 벡터
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

Family Cites Families (26)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5672344A (en) 1987-12-30 1997-09-30 The Regents Of The University Of Michigan Viral-mediated gene transfer system
US5773278A (en) 1991-05-03 1998-06-30 Mount Sinai Medical Center Acid sphingomyelinase gene
JP3532566B2 (ja) 1993-06-24 2004-05-31 エル． グラハム，フランク 遺伝子治療のためのアデノウイルスベクター
DE69434594T2 (de) 1993-10-25 2006-09-21 Canji, Inc., San Diego Rekombinante adenoviren-vektor und verfahren zur verwendung
DK0733103T3 (da) 1993-11-09 2004-07-12 Univ Johns Hopkins Fremstilling af höje titre af rekombinante AAV vektorer
US7252989B1 (en) 1994-04-04 2007-08-07 Board Of Regents, The University Of Texas System Adenovirus supervector system
EP0755454B1 (en) 1994-04-13 2008-02-13 The Rockefeller University Aav-mediated delivery of dna to cells of the nervous system
US6274309B1 (en) 1996-07-26 2001-08-14 Richard Kolesnick Method for the modulation of acid-sphingomyelinase-related apoptosis
US20040076613A1 (en) * 2000-11-03 2004-04-22 Nicholas Mazarakis Vector system
US20050026832A1 (en) * 1997-11-25 2005-02-03 Genentech, Inc. Fibroblast growth factor-19 (FGF-19) nucleic acids and polypeptides and methods of use for the treatment of obesity and related disorders
US6544785B1 (en) 1998-09-14 2003-04-08 Mount Sinai School Of Medicine Of New York University Helper-free rescue of recombinant negative strand RNA viruses
US6730297B1 (en) * 1999-05-28 2004-05-04 Chiron Corporation Use of recombinant gene delivery vectors for treating or preventing lysosomal storage disorders
WO2001036603A2 (en) * 1999-11-17 2001-05-25 Avigen, Inc. Recombinant adeno-associated virus virions for the treatment of lysosomal disorders
WO2001060377A2 (en) * 2000-02-17 2001-08-23 Genzyme Corporation Methods for treatment of lysosomal storage diseases using biphosphonates
US20040204379A1 (en) * 2000-06-19 2004-10-14 Cheng Seng H. Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
US6667174B2 (en) 2000-09-18 2003-12-23 Genzyme Corporation Expression vectors containing hybrid ubiquitin promoters
US20040258666A1 (en) * 2003-05-01 2004-12-23 Passini Marco A. Gene therapy for neurometabolic disorders
WO2004111198A2 (en) 2003-06-12 2004-12-23 Genzyme Corporation Modified human acid sphingomyelinase having increased activity, and methods for making the same
US7442372B2 (en) * 2003-08-29 2008-10-28 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
US20050208090A1 (en) * 2004-03-18 2005-09-22 Medtronic, Inc. Methods and systems for treatment of neurological diseases of the central nervous system
US20060171926A1 (en) * 2004-04-30 2006-08-03 Passini Marco A Gene therapy for neurometabolic disorders
CN104306986A (zh) * 2005-05-02 2015-01-28 建新公司 神经代谢疾病的基因治疗
BRPI0707590A2 (pt) * 2006-02-08 2011-05-10 Genzyme Corp terapia gÊnica para a doenÇa de niemann-pick do tipo a
PL1988823T3 (pl) * 2006-02-09 2019-01-31 Genzyme Corporation Powolne dostarczanie dokomorowe
SI2158322T1 (sl) 2007-06-06 2017-10-30 Genzyme Corporation Genska terapija za bolezni lizosomskega shranjevanja
HRP20211992T1 (hr) 2009-08-28 2022-04-01 Icahn School Of Medicine At Mount Sinai Zamjenska enzimska terapija povećanjem doze za liječenje nedostatka kiseline sfingomijelinaze

Also Published As

Publication number Publication date
US20100173979A1 (en) 2010-07-08
EP2158322A1 (en) 2010-03-03
US11369693B2 (en) 2022-06-28
EP2158322B1 (en) 2017-05-03
EP3252161A1 (en) 2017-12-06
ES2905616T3 (es) 2022-04-11
IL202522A0 (en) 2011-08-01
SI2158322T1 (sl) 2017-10-30
IL252898B (en) 2019-06-30
PL3252161T3 (pl) 2022-03-07
IL202522A (en) 2017-10-31
AR070000A1 (es) 2010-03-10
HRP20171165T1 (hr) 2017-12-15
WO2008154198A1 (en) 2008-12-18
DK2158322T3 (en) 2017-08-28
EP2158322A4 (en) 2011-06-01
PT3252161T (pt) 2022-02-01
LT2158322T (lt) 2017-08-25
ES2635726T3 (es) 2017-10-04
US8796236B2 (en) 2014-08-05
HUE035779T2 (en) 2018-05-28
PT2158322T (pt) 2017-08-09
IL266734B (en) 2021-10-31
US20150151007A1 (en) 2015-06-04
EP3252161B1 (en) 2021-11-17
IL266734A (en) 2019-07-31
IL252898A0 (en) 2017-08-31
PL2158322T3 (pl) 2017-10-31

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