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WO1999061595A3 - Expression de l'activite du facteur viii par un vecteur viral adeno-associe - Google Patents

Expression de l'activite du facteur viii par un vecteur viral adeno-associe Download PDF

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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
Legal status (The legal status 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 status listed.)
Ceased
Application number
PCT/US1999/010472
Other languages
English (en)
Other versions
WO1999061595A2 (fr
Inventor
Lawrence K Cohen
S Kaye Spratt
Linda Couto
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Cell Genesys Inc
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 EP99925606A priority Critical patent/EP1082445A2/fr
Priority to AU41856/99A priority patent/AU4185699A/en
Priority to JP2000550980A priority patent/JP2002516345A/ja
Priority to CA002329143A priority patent/CA2329143A1/fr
Publication of WO1999061595A2 publication Critical patent/WO1999061595A2/fr
Publication of WO1999061595A3 publication Critical patent/WO1999061595A3/fr
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

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

Abstract

L'invention concerne des procédés et des matériaux permettant l'expression de polypeptides comprenant une activité facteur VIII. Ces procédés comprennent l'administration d'au moins deux vecteur rAAV codant pour différents domaines du facteur VIII humain et au moins pour les chaînes lourde et légère.
PCT/US1999/010472 1998-05-27 1999-05-27 Expression de l'activite du facteur viii par un vecteur viral adeno-associe Ceased WO1999061595A2 (fr)

Priority Applications (4)

Application Number Priority Date Filing Date Title
EP99925606A EP1082445A2 (fr) 1998-05-27 1999-05-27 Expression de l'activite du facteur viii par un vecteur viral adeno-associe
AU41856/99A AU4185699A (en) 1998-05-27 1999-05-27 Adeno-associated viral vector-mediated expression of factor viii activity
JP2000550980A JP2002516345A (ja) 1998-05-27 1999-05-27 第ｖｉｉｉ因子活性のアデノ随伴ウイルスベクター媒介性発現
CA002329143A CA2329143A1 (fr) 1998-05-27 1999-05-27 Expression de l'activite du facteur viii par un vecteur viral adeno-associe

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 (fr) 1999-12-02
WO1999061595A3 true WO1999061595A3 (fr) 2000-01-27

Family

ID=22184882

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US1999/010472 Ceased WO1999061595A2 (fr) 1998-05-27 1999-05-27 Expression de l'activite du facteur viii par un vecteur viral adeno-associe

Country Status (5)

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

Families Citing this family (27)

* 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
US7498024B2 (en) 2003-06-03 2009-03-03 Cell Genesys, Inc. Compositions and methods for enhanced expression of immunoglobulins from a single vector using a peptide cleavage site
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
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
MX2017005834A (es) 2014-11-05 2017-11-17 Voyager Therapeutics Inc Polinucleotidos aad para el tratamiento de la enfermedad de parkinson.
RU2020108189A (ru) 2014-11-14 2020-03-11 Вояджер Терапьютикс, Инк. Композиции и способы лечения бокового амиотрофического склероза (als)
MX2017006217A (es) 2014-11-14 2018-05-02 Voyager Therapeutics Inc Polinucleotidos moduladores.
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
RU2742352C2 (ru) * 2015-09-24 2021-02-05 Байомарин Фармасьютикал Инк. Векторы фактора fviii на основе аденоассоциированных вирусов, соответствующие вирусные частицы и содержащие их терапевтические композиции
US10983110B2 (en) 2015-12-02 2021-04-20 Voyager Therapeutics, Inc. Assays for the detection of AAV neutralizing antibodies
EP3448987A4 (fr) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. Compositions pour le traitement de maladies
EP3448874A4 (fr) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. Compositions pour le traitement de maladies
SG11201809699XA (en) 2016-05-18 2018-12-28 Voyager Therapeutics Inc Modulatory polynucleotides
SG11201809643UA (en) 2016-05-18 2018-12-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
EP3831281A1 (fr) 2016-08-30 2021-06-09 The Regents of The University of California Procédés de ciblage et d'administration biomédicaux, et dispositifs et systèmes pour leur mise en uvre
EP3619308A4 (fr) 2017-05-05 2021-01-27 Voyager Therapeutics, Inc. Compositions et méthodes de traitement de la maladie de huntington
CN110913866A (zh) 2017-05-05 2020-03-24 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
AU2018302016A1 (en) 2017-07-17 2020-02-06 The Regents Of The University Of California Trajectory array guide system
JP7221275B2 (ja) 2017-08-03 2023-02-13 ボイジャー セラピューティクス インコーポレイテッド Ａａｖを送達するための組成物および方法
AU2018338728B2 (en) 2017-09-29 2025-01-02 Centre National De La Recherche Scientifique (Cnrs) Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery
EP4124658A3 (fr) 2017-10-16 2023-04-19 Voyager Therapeutics, Inc. Traitement de la sclérose latérale amyotrophique (sla)
US20200237799A1 (en) 2017-10-16 2020-07-30 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
TW202012628A (zh) 2018-05-15 2020-04-01 美商航海家醫療公司 用於帕金森氏症治療的組成物及方法
JP7142815B2 (ja) * 2018-06-21 2022-09-28 株式会社遺伝子治療研究所 ヒト肝臓への遺伝子導入のためのアデノ随伴ウイルスビリオン
WO2020069461A1 (fr) 2018-09-28 2020-04-02 Voyager Therapeutics, Inc. Constructions d'expression de frataxine comprenant des promoteurs modifiés et leurs méthodes d'utilisation
CN113853437A (zh) * 2018-11-07 2021-12-28 阿库斯股份有限公司 腺相关病毒载体用于在内耳中的毛细胞和支持细胞中校正基因缺陷/表达蛋白质的用途

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 *

Also Published As

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

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