HRP20171165T1 - Genska terapija za lizosomske bolesti nakupljanja - Google Patents

Genska terapija za lizosomske bolesti nakupljanja Download PDF

Info

Publication number
HRP20171165T1
HRP20171165T1 HRP20171165TT HRP20171165T HRP20171165T1 HR P20171165 T1 HRP20171165 T1 HR P20171165T1 HR P20171165T T HRP20171165T T HR P20171165TT HR P20171165 T HRP20171165 T HR P20171165T HR P20171165 T1 HRP20171165 T1 HR P20171165T1
Authority
HR
Croatia
Prior art keywords
aav vector
recombinant aav
use according
vector
brain
Prior art date
Application number
HRP20171165TT
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 HRP20171165T1 publication Critical patent/HRP20171165T1/hr

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/47Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01045Glucosylceramidase (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)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Virology (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)

Claims (6)

1. Rekombinantni AAV vektor koji sadrži transgen kodira kiselinu sfingomijelinazu za upotrebu u postupku liječenja Niemann–Pickove bolesti tip A ili Niemann–Pickove bolesti tip B kod subjekta, naznačen time da navedeni rekombinantni AAV vektor je AAV5 vektor i pri čemu postupak sadrži davanje vektora u barem jednu ventrikulu mozga od subjekta, pri čemu se navedeni transgen eksprimira i eksprimirani proizvod proteina se dovodi u središnji živčani sustav, te proizvoljno, u visceralne organe.
2. Rekombinantni AAV vektor za uporabu prema zahtjevu 1, naznačen time da navedeni rekombinantni AAV vektor je AAV vektor koji sadrži AAV5 ovojnicu i AAV2 genom.
3. Rekombinantni AAV vektor za uporabu prema zahtjevu 1 ili zahtjevu 2, naznačen time da postupak sadrži davanje vektora izravnim ubrizgavanjem u: (i) ventrikulu mozga; (ii) lateralnu ventrikulu mozga; ili (iii) četvrtu ventrikulu mozga.
4. Rekombinantni AAV vektor za uporabu prema bilo kojem od zahtjeva 1 do 3, naznačen time da subjekt je sisavac.
5. Rekombinantni AAV vektor za uporabu prema zahtjevu 4, naznačen time da subjekt je ljudski pacijent.
6. Rekombinantni AAV vektor za uporabu prema bilo kojem od zahtjeva 1 do 5, naznačen time da navedeni postupak je postupak liječenja Niemann–Pickove bolesti tip A.
HRP20171165TT 2007-06-06 2017-07-31 Genska terapija za lizosomske bolesti nakupljanja HRP20171165T1 (hr)

Applications Claiming Priority (3)

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
EP08756596.6A EP2158322B1 (en) 2007-06-06 2008-06-02 Gene therapy for lysosomal storage diseases

Publications (1)

Publication Number Publication Date
HRP20171165T1 true HRP20171165T1 (hr) 2017-12-15

Family

ID=40130114

Family Applications (1)

Application Number Title Priority Date Filing Date
HRP20171165TT HRP20171165T1 (hr) 2007-06-06 2017-07-31 Genska terapija za lizosomske bolesti nakupljanja

Country Status (14)

Country Link
US (2) US8796236B2 (hr)
EP (2) EP2158322B1 (hr)
AR (1) AR070000A1 (hr)
CY (1) CY1119295T1 (hr)
DK (1) DK2158322T3 (hr)
ES (2) ES2905616T3 (hr)
HR (1) HRP20171165T1 (hr)
HU (1) HUE035779T2 (hr)
IL (3) IL202522A (hr)
LT (1) LT2158322T (hr)
PL (2) PL3252161T3 (hr)
PT (2) PT3252161T (hr)
SI (1) SI2158322T1 (hr)
WO (1) WO2008154198A1 (hr)

Families Citing this family (34)

* 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
DK1988823T3 (en) 2006-02-09 2018-12-03 Genzyme Corp SLOW INTRAVENTRICULAR ADMINISTRATION
PL3252161T3 (pl) 2007-06-06 2022-03-07 Genzyme Corporation 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
DK2424571T3 (da) 2009-04-30 2020-05-04 Ospedale San Raffaele Srl Genvektor
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
CA3049237C (en) 2010-04-23 2024-06-11 University Of Massachusetts Cns targeting aav vectors and methods of use thereof
CA3050894C (en) 2010-04-23 2022-10-18 University Of Massachusetts Multicistronic expression constructs
EP3546584B1 (en) 2011-03-31 2024-02-14 University of Iowa Research Foundation Compositions for treating lysosmal storage disease (lsd)
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
PL2751279T3 (pl) 2011-08-31 2018-03-30 St. Jude Children's Research Hospital Sposoby i kompozycje do wykrywania poziomu aktywności lizosomalnej egzocytozy i sposoby zastosowania
BR112015028605A8 (pt) * 2013-05-15 2019-12-24 Univ Minnesota uso de uma composição e uso de um imunossupressor e uma composição
EP3039129B1 (en) * 2013-08-30 2018-06-27 Amgen Inc. High titer recombinant aav vector production in adherent and suspension cells
AU2015335923B2 (en) 2014-10-21 2021-04-29 University Of Massachusetts Recombinant AAV variants and uses thereof
BR112017009497A2 (pt) 2014-11-05 2018-02-06 Voyager Therapeutics, Inc. polinucleotídeos de aadc para o tratamento da doença de parkinson
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
AU2016372035B2 (en) * 2015-12-15 2023-03-30 Genzyme Corporation Adeno-associated viral vectors for treating mucolipidosis type ii
PT3405215T (pt) * 2016-01-19 2022-09-20 Univ California Métodos para o tratamento da doença de danon e outros transtornos da autofagia
AU2017250298B2 (en) 2016-04-15 2024-10-03 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type II
WO2018071831A1 (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
EP3534892A4 (en) * 2016-11-04 2020-05-27 The Children's Hospital of Philadelphia GENE TRANSFER COMPOSITIONS, METHODS AND USE FOR TREATING NEURODEGENERATIVE DISEASES
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
EP3642345A1 (en) 2017-06-20 2020-04-29 The U.S.A. 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
AU2018335752A1 (en) 2017-09-22 2020-03-12 Christian HINDERER Gene therapy for treating Mucopolysaccharidosis type ii
US20210228739A1 (en) * 2018-05-15 2021-07-29 University Of Massachusetts Raav vectors encoding of lysosomal beta-galactosidase (glb1) and cathepsin a
EP3820537A4 (en) 2018-07-12 2022-04-13 Spacecraft Seven, LLC GENE THERAPY VECTORS FOR TREATMENT OF DANON DISEASE
MX2021010266A (es) * 2019-02-26 2021-09-23 Univ Pennsylvania Composiciones utiles en el tratamiento de la enfermedad de krabbe.
BR112021020421A2 (pt) * 2019-04-12 2021-12-21 Encoded Therapeutics Inc Composições e métodos para administração de componentes terapêuticos
US11981912B2 (en) 2019-12-10 2024-05-14 Takeda Pharma ceutical Company Limited Adeno associated virus vectors for the treatment of hunter disease
CA3174863A1 (en) * 2020-04-14 2021-10-21 Ana BUJ BELLO Vectors for the treatment of acid ceramidase deficiency

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
CA2166118C (en) 1993-06-24 2007-04-17 Frank L. Graham Adenovirus vectors for gene therapy
HU223733B1 (hu) 1993-10-25 2004-12-28 Canji, Inc. Rekombináns adenovírus vektor és eljárás alkalmazására
WO1995013365A1 (en) 1993-11-09 1995-05-18 Targeted Genetics Corporation Generation of high titers of recombinant aav vectors
US7252989B1 (en) 1994-04-04 2007-08-07 Board Of Regents, The University Of Texas System Adenovirus supervector system
ATE386131T1 (de) 1994-04-13 2008-03-15 Univ Rockefeller Aav-vermittelte überbringung von dna in zellen des nervensystems
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
AU1775901A (en) * 1999-11-17 2001-05-30 Avigen, Inc. Recombinant adeno-associated virus virions for the treatment of lysosomal disorders
US20010031741A1 (en) * 2000-02-17 2001-10-18 Robin Ziegler Methods for treatment of lysosomal storage diseases
US20040204379A1 (en) * 2000-06-19 2004-10-14 Cheng Seng H. Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
WO2002024932A2 (en) 2000-09-18 2002-03-28 Genzyme Corporation Expression vectors containing hybrid ubiquitin promoters
US20040258666A1 (en) 2003-05-01 2004-12-23 Passini Marco A. Gene therapy for neurometabolic disorders
CA2528427A1 (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
CN101212988A (zh) * 2005-05-02 2008-07-02 建新公司 神经代谢疾病的基因治疗
JP5706602B2 (ja) * 2006-02-08 2015-04-22 ジェンザイム・コーポレーション ニーマンピック病a型に対する遺伝子治療
DK1988823T3 (en) * 2006-02-09 2018-12-03 Genzyme Corp SLOW INTRAVENTRICULAR ADMINISTRATION
PL3252161T3 (pl) 2007-06-06 2022-03-07 Genzyme Corporation Terapia genowa dla lizosomalnych chorób spichrzeniowych
KR20230164773A (ko) 2009-08-28 2023-12-04 이칸 스쿨 오브 메디슨 엣 마운트 시나이 산성 스핑고미엘리나제 결핍을 치료하기 위한 투여량 단계적 증가 효소 대체 요법

Also Published As

Publication number Publication date
IL252898A0 (en) 2017-08-31
DK2158322T3 (en) 2017-08-28
IL266734B (en) 2021-10-31
PT2158322T (pt) 2017-08-09
PL2158322T3 (pl) 2017-10-31
CY1119295T1 (el) 2018-02-14
US11369693B2 (en) 2022-06-28
EP3252161A1 (en) 2017-12-06
IL266734A (en) 2019-07-31
PL3252161T3 (pl) 2022-03-07
US20100173979A1 (en) 2010-07-08
EP2158322A4 (en) 2011-06-01
US8796236B2 (en) 2014-08-05
EP3252161B1 (en) 2021-11-17
LT2158322T (lt) 2017-08-25
IL202522A0 (en) 2011-08-01
EP2158322A1 (en) 2010-03-03
PT3252161T (pt) 2022-02-01
HUE035779T2 (en) 2018-05-28
EP2158322B1 (en) 2017-05-03
ES2905616T3 (es) 2022-04-11
ES2635726T3 (es) 2017-10-04
AR070000A1 (es) 2010-03-10
IL252898B (en) 2019-06-30
US20150151007A1 (en) 2015-06-04
WO2008154198A1 (en) 2008-12-18
IL202522A (en) 2017-10-31
SI2158322T1 (sl) 2017-10-30

Similar Documents

Publication Publication Date Title
HRP20171165T1 (hr) Genska terapija za lizosomske bolesti nakupljanja
US20240285667A1 (en) Central nervous system targeting polynucleotides
RU2018120736A (ru) Нацеливающие пептиды для направленной доставки аденоассоциированных вирусов (aav)
RU2653444C2 (ru) Композиции вектора aav и способы переноса генов в клетки, органы и ткани
ES2814901T3 (es) Vectores de AAV dirigidos a oligodendrocitos
JP2020527333A (ja) パーキンソン病を治療するためのaadcポリヌクレオチド
JP2020533959A (ja) Aavを送達するための組成物および方法
JP2022525955A (ja) 組換えアデノ随伴ウイルスベクター
JP2020019796A (ja) 脊髄軟膜下遺伝子送達システム
JP2021519581A (ja) 抗体を回避するウイルスベクター
RU2011149094A (ru) Генная терапия нейродегенеративных нарушений
JP2018506530A5 (hr)
KR20210006357A (ko) 항체-회피 바이러스 벡터
UA112841C2 (uk) Вектор та послідовність для лікування мукополісахаридозів
WO2007092563A3 (en) Gene therapy for niemann-pick disease type a
JP2017518271A5 (hr)
WO2007127428A8 (en) Double-stranded/self-complementary vectors with a truncated cba promoter and methods of gene delivery
CA3002406A1 (en) Delivery of central nervous system targeting polynucleotides
CN107427557A (zh) 用于包装和表达变体因子viii以治疗血友病的改良表达组件
WO2012159006A8 (en) Polypeptides and vectors for targeting her2/neu expressing cells and uses thereof
JP2021523914A (ja) パーキンソン病を治療するための組成物および方法
TW202014210A (zh) 具增強趨性之經工程化aav蛋白質膜及包含該經工程化蛋白質膜之aav載體以及其製造及使用方法
HRP20180221T1 (hr) Novi sustav prijenosa lijeka zasnovan za jcv-vlp
Bish et al. Adeno-associated virus vector delivery to the heart
Foust et al. Recombinant adeno-associated virus-mediated global anterograde delivery of glial cell line-derived neurotrophic factor to the spinal cord: comparison of rubrospinal and corticospinal tracts in the rat