HK1035205A1 - Recombinant adeno-associated viral vector encodingalpha-1-antitrypsin for gene therapy. - Google Patents

Recombinant adeno-associated viral vector encodingalpha-1-antitrypsin for gene therapy.

Info

Publication number
HK1035205A1
HK1035205A1 HK01103975A HK01103975A HK1035205A1 HK 1035205 A1 HK1035205 A1 HK 1035205A1 HK 01103975 A HK01103975 A HK 01103975A HK 01103975 A HK01103975 A HK 01103975A HK 1035205 A1 HK1035205 A1 HK 1035205A1
Authority
HK
Hong Kong
Prior art keywords
gene therapy
methods
antitrypsin
encodingalpha
viral vector
Prior art date
Application number
HK01103975A
Other languages
English (en)
Inventor
Terence R Flotte
Sihong Song
Barry J Byrne
Michael Morgan
Original Assignee
Univ Florida
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 Univ Florida filed Critical Univ Florida
Publication of HK1035205A1 publication Critical patent/HK1035205A1/xx

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/81—Protease inhibitors
    • C07K14/8107—Endopeptidase (E.C. 3.4.21-99) inhibitors
    • C07K14/811—Serine protease (E.C. 3.4.21) inhibitors
    • C07K14/8121—Serpins
    • C07K14/8125—Alpha-1-antitrypsin
    • 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
    • A61K38/55—Protease inhibitors
    • A61K38/57—Protease inhibitors from animals; from humans
    • 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
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • 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
    • 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
    • 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)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Diabetes (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Immunology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Endocrinology (AREA)
  • Emergency Medicine (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (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)
HK01103975A 1998-04-24 2001-06-11 Recombinant adeno-associated viral vector encodingalpha-1-antitrypsin for gene therapy. HK1035205A1 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US8302598P 1998-04-24 1998-04-24
PCT/US1999/008921 WO1999055564A1 (en) 1998-04-24 1999-04-23 Materials and methods for gene therapy

Publications (1)

Publication Number Publication Date
HK1035205A1 true HK1035205A1 (en) 2001-11-16

Family

ID=22175690

Family Applications (1)

Application Number Title Priority Date Filing Date
HK01103975A HK1035205A1 (en) 1998-04-24 2001-06-11 Recombinant adeno-associated viral vector encodingalpha-1-antitrypsin for gene therapy.

Country Status (12)

Country Link
US (4) US6461606B1 (xx)
EP (1) EP1071806B1 (xx)
AT (1) ATE269412T1 (xx)
AU (1) AU3663699A (xx)
CA (1) CA2326327C (xx)
DE (1) DE69918090T2 (xx)
DK (1) DK1071806T3 (xx)
ES (1) ES2219011T3 (xx)
HK (1) HK1035205A1 (xx)
NZ (1) NZ507308A (xx)
PT (1) PT1071806E (xx)
WO (1) WO1999055564A1 (xx)

Families Citing this family (25)

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Publication number Priority date Publication date Assignee Title
US6759237B1 (en) * 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
JP4851041B2 (ja) * 1999-11-05 2012-01-11 協和発酵キリン株式会社 細胞内のカルシウムイオンの濃度変化をモニターするトランスジェニック非ヒト哺乳動物
US6821775B1 (en) * 2000-02-11 2004-11-23 Genvec, Inc. Viral vector encoding pigment epithelium-derived factor
KR101250021B1 (ko) 2000-05-26 2013-04-03 다이닛본 스미토모 세이야꾸 가부시끼가이샤 완화된 부작용을 갖는 신규 재조합 아데노바이러스 벡터
EP2017338A1 (en) * 2001-05-24 2009-01-21 Genzyme Corporation Muscle-specific expression vectors
WO2003089011A1 (en) * 2002-04-19 2003-10-30 University Of Florida rAAV VECTOR-BASED COMPOSITIONS AND METHODS FOR THE PREVENTION AND TREATMENT OF MAMMALIAN DISEASES
US7312202B2 (en) * 2003-02-18 2007-12-25 Board Of Regents, The University Of Texas System Rationally designed and chemically synthesized promoter for genetic vaccine and gene therapy
ES2247942B1 (es) * 2004-08-27 2006-10-01 Instituto Nacional De Investigacion Y Tecnologia Agraria Y Alimentaria (Inia) Construccion genica, vector y vacuna adn para la vacunacion de animales acuaticos.
US7479550B2 (en) * 2006-06-02 2009-01-20 The Board Of Regents Of The University Of Texas System Amyloid β gene vaccines
WO2009091912A2 (en) 2008-01-15 2009-07-23 Abbott Laboratories Improved mammalian expression vectors and uses thereof
AU2012327236B2 (en) 2011-10-28 2016-11-10 University Of Florida Research Foundation, Inc. Chimeric promoter for cone photoreceptor targeted gene therapy
WO2013106273A2 (en) 2012-01-09 2013-07-18 Serpin Pharma, Llc Peptides and methods of using same
EP3253406B1 (en) * 2015-02-05 2023-11-01 Canem Holdings, LLC Compositions for treating granulomatosis with polyangiitis
EP4089172A3 (en) 2015-03-03 2023-03-01 Fondazione Telethon Multiple vector system and uses thereof
CA2996975C (en) 2015-08-28 2022-02-15 Serpin Pharma, Llc Methods for treatment of diseases
EP3356397A1 (en) 2015-09-28 2018-08-08 Fondazione Telethon Treatment of bone growth disorders
EP3723790B1 (en) * 2017-12-12 2024-03-13 Kamada Ltd Methods of inducing immune tolerance and reducing anti-drug antibody response
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
CN111886021A (zh) * 2018-02-01 2020-11-03 同源药物公司 用于pah基因转移的腺伴随病毒组合物及其使用方法
US11306329B2 (en) 2018-02-19 2022-04-19 City Of Hope Adeno-associated virus compositions for restoring F8 gene function and methods of use thereof
EP3923995A4 (en) * 2019-02-15 2023-05-17 Allen Institute ARTIFICIAL EXPRESSION RECOMBINANTS FOR THE SELECTIVE MODULATION OF GENE EXPRESSION IN SELECTED NEURON CELL POPULATIONS
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
AU2021252515A1 (en) * 2020-04-06 2022-10-27 Homology Medicines, Inc. Adeno-associated virus compositions for IDS gene transfer and methods of use thereof
WO2023213817A1 (en) 2022-05-02 2023-11-09 Fondazione Telethon Ets Gene therapy for gyrate atrophy of the choroid and retina
CN117223676A (zh) * 2023-09-25 2023-12-15 武汉大学 面中部发育畸形动物选育方法、辅助选育试剂和预防药物

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5399346A (en) 1989-06-14 1995-03-21 The United States Of America As Represented By The Department Of Health And Human Services Gene therapy
US6261834B1 (en) * 1991-11-08 2001-07-17 Research Corporation Technologies, Inc. Vector for gene therapy
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
US5439824A (en) * 1993-05-06 1995-08-08 The United States Of America Increased expression of α-1-antitrypsin in expression vectors through the inclusion of intron II
ES2216005T3 (es) 1993-11-09 2004-10-16 Targeted Genetics Corporation Produccion de titulos elevados de vectores de aav recombinantes.
US5693531A (en) * 1993-11-24 1997-12-02 The United States Of America As Represented By The Department Of Health And Human Services Vector systems for the generation of adeno-associated virus particles
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US6204059B1 (en) * 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US5652224A (en) * 1995-02-24 1997-07-29 The Trustees Of The University Of Pennsylvania Methods and compositions for gene therapy for the treatment of defects in lipoprotein metabolism
US5846528A (en) 1996-01-18 1998-12-08 Avigen, Inc. Treating anemia using recombinant adeno-associated virus virions comprising an EPO DNA sequence
US5858351A (en) 1996-01-18 1999-01-12 Avigen, Inc. Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors
US5780447A (en) * 1996-06-14 1998-07-14 St. Jude Children's Research Hospital Recombinant adeno-associated viral vectors
AU5070298A (en) * 1996-12-05 1998-06-29 Introgene B.V. Genetic modification of primate hemopoietic repopulating stem cells
US6723707B1 (en) * 1997-08-25 2004-04-20 The Trustees Of The University Of Pennsylvania Use of insulin-like growth factor-I in muscle

Also Published As

Publication number Publication date
AU3663699A (en) 1999-11-16
DK1071806T3 (da) 2004-07-19
ES2219011T3 (es) 2004-11-16
CA2326327C (en) 2009-06-16
US6461606B1 (en) 2002-10-08
PT1071806E (pt) 2004-09-30
DE69918090D1 (de) 2004-07-22
WO1999055564A8 (en) 1999-12-02
CA2326327A1 (en) 1999-11-04
US20090186002A1 (en) 2009-07-23
EP1071806B1 (en) 2004-06-16
ATE269412T1 (de) 2004-07-15
US20030095949A1 (en) 2003-05-22
NZ507308A (en) 2003-01-31
WO1999055564A9 (en) 2000-04-06
WO1999055564A1 (en) 1999-11-04
DE69918090T2 (de) 2005-06-16
EP1071806A2 (en) 2001-01-31
US20030082162A1 (en) 2003-05-01

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Legal Events

Date Code Title Description
AM Amended specification (according sect 146 of patent law)

Free format text: CORRECTION OF THE NAME OF INVENTOR FROM BARRY J. BYRNES TO BARRY J. BYRNE.

Effective date: 20050304

PC Patent ceased (i.e. patent has lapsed due to the failure to pay the renewal fee)

Effective date: 20170423