[go: up one dir, main page]
More Web Proxy on the site http://driver.im/

WO1999061595A3 - Adeno-associated viral vector-mediated expression of factor viii activity - Google Patents

Adeno-associated viral vector-mediated expression of factor viii activity Download PDF

Info

Publication number
WO1999061595A3
WO1999061595A3 PCT/US1999/010472 US9910472W WO9961595A3 WO 1999061595 A3 WO1999061595 A3 WO 1999061595A3 US 9910472 W US9910472 W US 9910472W WO 9961595 A3 WO9961595 A3 WO 9961595A3
Authority
WO
WIPO (PCT)
Prior art keywords
factor viii
adeno
viral vector
viii activity
associated viral
Prior art date
Application number
PCT/US1999/010472
Other languages
French (fr)
Other versions
WO1999061595A2 (en
Inventor
Lawrence K Cohen
S Kaye Spratt
Linda Couto
Original Assignee
Cell Genesys Inc
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 Cell Genesys Inc filed Critical Cell Genesys Inc
Priority to CA002329143A priority Critical patent/CA2329143A1/en
Priority to JP2000550980A priority patent/JP2002516345A/en
Priority to AU41856/99A priority patent/AU4185699A/en
Priority to EP99925606A priority patent/EP1082445A2/en
Publication of WO1999061595A2 publication Critical patent/WO1999061595A2/en
Publication of WO1999061595A3 publication Critical patent/WO1999061595A3/en

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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • 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

Landscapes

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

Abstract

The instant invention provides methods and materials for expressing polypeptides with factor VIII activity comprising administering at least two rAAV vectors encoding different domains of human factor VIII and at least the heavy and light chains.
PCT/US1999/010472 1998-05-27 1999-05-27 Adeno-associated viral vector-mediated expression of factor viii activity WO1999061595A2 (en)

Priority Applications (4)

Application Number Priority Date Filing Date Title
CA002329143A CA2329143A1 (en) 1998-05-27 1999-05-27 Adeno-associated viral vector-mediated expression of factor viii activity
JP2000550980A JP2002516345A (en) 1998-05-27 1999-05-27 Adeno-associated virus vector-mediated expression of factor VIII activity
AU41856/99A AU4185699A (en) 1998-05-27 1999-05-27 Adeno-associated viral vector-mediated expression of factor viii activity
EP99925606A EP1082445A2 (en) 1998-05-27 1999-05-27 Adeno-associated viral vector-mediated expression of factor viii activity

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US8442398A 1998-05-27 1998-05-27
US09/084,423 1998-05-27

Publications (2)

Publication Number Publication Date
WO1999061595A2 WO1999061595A2 (en) 1999-12-02
WO1999061595A3 true WO1999061595A3 (en) 2000-01-27

Family

ID=22184882

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US1999/010472 WO1999061595A2 (en) 1998-05-27 1999-05-27 Adeno-associated viral vector-mediated expression of factor viii activity

Country Status (5)

Country Link
EP (1) EP1082445A2 (en)
JP (1) JP2002516345A (en)
AU (1) AU4185699A (en)
CA (1) CA2329143A1 (en)
WO (1) WO1999061595A2 (en)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12146150B2 (en) 2022-09-13 2024-11-19 Voyager Therapeutics, Inc. Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery

Families Citing this family (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6221349B1 (en) * 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
US7485291B2 (en) 2003-06-03 2009-02-03 Cell Genesys, Inc. Compositions and methods for generating multiple polypeptides from a single vector using a virus derived peptide cleavage site, and uses thereof
CA2526120A1 (en) 2003-06-03 2005-02-24 Cell Genesys, Inc. Compositions and methods for enhanced expression of recombinant polypeptides from a single vector using a peptide cleavage site
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
SG11201703148TA (en) 2014-11-05 2017-05-30 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
CN112375760A (en) 2014-11-14 2021-02-19 沃雅戈治疗公司 Regulatory polynucleotides
EP3218484A4 (en) 2014-11-14 2018-05-30 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
EP3230441A4 (en) 2014-12-12 2018-10-03 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
NZ780570A (en) 2015-09-24 2024-08-30 Biomarin Pharm Inc Adeno-associated virus factor viii vectors, associated viral particles and therapeutic formulations comprising the same
CA3006569A1 (en) 2015-12-02 2017-06-08 Voyager Therapeutics, Inc. Assays for the detection of aav neutralizing antibodies
US11299751B2 (en) 2016-04-29 2022-04-12 Voyager Therapeutics, Inc. Compositions for the treatment of disease
US11326182B2 (en) 2016-04-29 2022-05-10 Voyager Therapeutics, Inc. Compositions for the treatment of disease
AU2017267665C1 (en) 2016-05-18 2023-10-05 Voyager Therapeutics, Inc. Modulatory polynucleotides
IL297576B2 (en) 2016-05-18 2024-02-01 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
CN110650673B (en) 2016-08-30 2024-04-09 加利福尼亚大学董事会 Methods for biomedical targeting and delivery and devices and systems for practicing the methods
EP3619308A4 (en) 2017-05-05 2021-01-27 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
JP2020518258A (en) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． Amyotrophic lateral sclerosis (ALS) treatment composition and method
JOP20190269A1 (en) 2017-06-15 2019-11-20 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
AU2018302016A1 (en) 2017-07-17 2020-02-06 The Regents Of The University Of California Trajectory array guide system
MX2020001187A (en) 2017-08-03 2020-10-05 Voyager Therapeutics Inc Compositions and methods for delivery of aav.
TWI804518B (en) 2017-10-16 2023-06-11 美商航海家醫療公司 Treatment of amyotrophic lateral sclerosis (als)
US20200237799A1 (en) 2017-10-16 2020-07-30 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
JP7142815B2 (en) * 2018-06-21 2022-09-28 株式会社遺伝子治療研究所 Adeno-associated virus virions for gene transfer into human liver
AU2019377115A1 (en) * 2018-11-07 2021-05-20 Akouos, Inc. Use of adeno-associated viral vectors to correct gene defects/ express proteins in hair cells and supporting cells in the inner ear

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
FAN, D.S. ET AL.: "Behavioral recovery in 6-hydroxydopamine-lesioned rats by cotransduction of striatum with ryrosine hydroxylase and aromatic L-amino acid decarboxylase genes using two separate adeno-associated virus vectors", HUMAN GENE THERAPY, vol. 9, no. 17, 2 November 1998 (1998-11-02), pages 2527 - 2535, XP002122885 *
GNATENKO, D. ET AL.: "Recombinant adeno-associated virus as a vehicle for gene delivery of human mutant factor VIII", BLOOD, vol. 90, no. 10 Suppl., 1 Part 1, 15 November 1997 (1997-11-15), pages p119A, XP002122884 *
SNYDER, R.O. ET AL.: "Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors", NATURE GENETICS, vol. 16, 16 July 1997 (1997-07-16), pages 270 - 276, XP002122888 *
WENDTNER, C.-M. ET AL.: "Gene transfer of costimulatory molecules B7-1 and B7-2 into human multiple myeloma cells by recombinant adeno-associated virus enhances the cytolytic T cell response", GENE THERAPY, vol. 4, no. 7, July 1997 (1997-07-01), pages 726 - 735, XP002122886 *
YONEMURA, H. ET AL.: "Efficient production of recombinant human factor VIII by co-expression of the heavy and light chains", PROTEIN ENGINEERING, vol. 6, no. 6, 1993, pages 669 - 674, XP000385586 *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12146150B2 (en) 2022-09-13 2024-11-19 Voyager Therapeutics, Inc. Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery

Also Published As

Publication number Publication date
AU4185699A (en) 1999-12-13
JP2002516345A (en) 2002-06-04
CA2329143A1 (en) 1999-12-02
WO1999061595A2 (en) 1999-12-02
EP1082445A2 (en) 2001-03-14

Similar Documents

Publication Publication Date Title
WO1999061595A3 (en) Adeno-associated viral vector-mediated expression of factor viii activity
IL138212A0 (en) Chimeric proteins with cell-targeting specificity and apoptosis-inducing activities
EP1022286A4 (en) POLYPEPTIDE, cDNA ENCODING THE POLYPEPTIDE, AND USE OF THE BOTH
WO1999033982A3 (en) Human genes and gene expression products i
WO2000005367A3 (en) Human proteins having hydrophobic domains and dnas encoding these proteins
AU5671594A (en) Human tissue factor related dna segments, polypeptides and antibodies
ITMI992519A0 (en) PROTEINS (IIP) THAT INTERACT WITH THE IGF-1 RECEPTOR GENES THAT CODE THEM AND THEIR USE
HUP9802217A3 (en) Recombinant mva virus, and the use thereof
WO1999038972A8 (en) Human genes and gene expression products ii
WO1999058675A3 (en) Human genes and gene expression products v
ZA9554B (en) Ribozymes targeting the retroviral packaging sequence expression constructs and recombinant retroviruses containing such constructs
EP0973924A4 (en) Recombinant chimeric viruses and uses thereof
DE69531958D1 (en) (1-3) -BETA-D-GLUUCON-BINDING PROTEIN, ANTIBODY DETECTING THIS PROTEIN AND USE OF THE PROTEIN AND ANTIBODY
ATE321873T1 (en) RECOMBINANT AAV VECTORS FOR GENE THERAPY OF HEMOPHILIA A
WO2000029448A3 (en) Human proteins having hydrophobic domains and dnas encoding these proteins
EP0851030A4 (en) PROTEIN SPECIFIC TO HUMAN Th2, GENE (B19) ENCODING THE SAME, AND TRANSFORMANT, RECOMBINANT VECTOR AND MONOCLONAL ANTIBODY RELATING THERETO
WO2000075324A3 (en) Recombinant prion-like genes and proteins and materials and methods comprising same
DE69634489D1 (en) ADENOVIRUS DODEKAHEDRIC PROTEIN COMPLEX, THIS COMPOSITION AND USES THEREOF
AU6292694A (en) Novel polypeptide, dna coding for said polypeptide, recombinant vector containing said dna, recombinant virus prepared using said vector, and use thereof
AU3698595A (en) Dna sequence and protein of the non-structural reading frame i of the human parvovirus b19
AU4558799A (en) Methods and compositions for generating recombinant adeno-associated virus vectors
WO2000018916A3 (en) Human genes and gene expression products
AU7471098A (en) Recombinant influenza viruses expressing tumor-associated antigens as antitum or agents
IL116819A0 (en) Ribozymes targetting the retroviral packaging sequence expression constructs and recombinant retroviruses containing such constructs
AU2176997A (en) Novel fused protein, gene therefor, recombinant vector, recombinant virus, and its use

Legal Events

Date Code Title Description
AK Designated states

Kind code of ref document: A2

Designated state(s): AE AL AM AT AU AZ BA BB BG BR BY CA CH CN CU CZ DE DK EE ES FI GB GD GE GH GM HR HU ID IL IN IS JP KE KG KP KR KZ LC LK LR LS LT LU LV MD MG MK MN MW MX NO NZ PL PT RO RU SD SE SG SI SK SL TJ TM TR TT UA UG UZ VN YU ZA ZW

AL Designated countries for regional patents

Kind code of ref document: A2

Designated state(s): GH GM KE LS MW SD SL SZ UG ZW AM AZ BY KG KZ MD RU TJ TM AT BE CH CY DE DK ES FI FR GB GR IE IT LU MC NL PT SE BF BJ CF CG CI CM GA GN GW ML MR NE SN TD TG

121 Ep: the epo has been informed by wipo that ep was designated in this application
AK Designated states

Kind code of ref document: A3

Designated state(s): AE AL AM AT AU AZ BA BB BG BR BY CA CH CN CU CZ DE DK EE ES FI GB GD GE GH GM HR HU ID IL IN IS JP KE KG KP KR KZ LC LK LR LS LT LU LV MD MG MK MN MW MX NO NZ PL PT RO RU SD SE SG SI SK SL TJ TM TR TT UA UG UZ VN YU ZA ZW

AL Designated countries for regional patents

Kind code of ref document: A3

Designated state(s): GH GM KE LS MW SD SL SZ UG ZW AM AZ BY KG KZ MD RU TJ TM AT BE CH CY DE DK ES FI FR GB GR IE IT LU MC NL PT SE BF BJ CF CG CI CM GA GN GW ML MR NE SN TD TG

DFPE Request for preliminary examination filed prior to expiration of 19th month from priority date (pct application filed before 20040101)
ENP Entry into the national phase

Ref document number: 2329143

Country of ref document: CA

ENP Entry into the national phase

Ref country code: JP

Ref document number: 2000 550980

Kind code of ref document: A

Format of ref document f/p: F

NENP Non-entry into the national phase

Ref country code: KR

WWE Wipo information: entry into national phase

Ref document number: 41856/99

Country of ref document: AU

WWE Wipo information: entry into national phase

Ref document number: 1999925606

Country of ref document: EP

WWP Wipo information: published in national office

Ref document number: 1999925606

Country of ref document: EP

REG Reference to national code

Ref country code: DE

Ref legal event code: 8642

WWW Wipo information: withdrawn in national office

Ref document number: 1999925606

Country of ref document: EP