BRPI0407648A - uso de uma proteìna ligante sìtio-especìfica ativa juntamente com um vetor terapêutico codificando uma proteìna para manufatura de um medicamente - Google Patents

uso de uma proteìna ligante sìtio-especìfica ativa juntamente com um vetor terapêutico codificando uma proteìna para manufatura de um medicamente

Info

Publication number
BRPI0407648A
BRPI0407648A BRPI0407648-6A BRPI0407648A BRPI0407648A BR PI0407648 A BRPI0407648 A BR PI0407648A BR PI0407648 A BRPI0407648 A BR PI0407648A BR PI0407648 A BRPI0407648 A BR PI0407648A
Authority
BR
Brazil
Prior art keywords
protein
vector encoding
site
manufacture
therapeutic vector
Prior art date
Application number
BRPI0407648-6A
Other languages
English (en)
Inventor
Jian-Qiang Fan
Original Assignee
Sinai School Medicine
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 Sinai School Medicine filed Critical Sinai School Medicine
Publication of BRPI0407648A publication Critical patent/BRPI0407648A/pt

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
    • A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

"USO DE UMA PROTEìNA LIGANTE SìTIO-ESPECìFICA ATIVA JUNTAMENTE COM UM VETOR TERAPêUTICO CODIFICANDO UMA PROTEìNA PARA MANUFATURA DE UM MEDICAMENTO". A patente provê métodos para melhorar terapia gênica, combinando terapia gênica com proteínas ligantes sítio-específicas ativas (ASSCs). A ASSC aumenta a estabilidade e eficiência da proteína codificada pelo gene recombinante que é administrado.
BRPI0407648-6A 2003-02-18 2004-02-18 uso de uma proteìna ligante sìtio-especìfica ativa juntamente com um vetor terapêutico codificando uma proteìna para manufatura de um medicamente BRPI0407648A (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US44807303P 2003-02-18 2003-02-18
PCT/US2004/004909 WO2004074450A2 (en) 2003-02-18 2004-02-18 Combination therapy for treating protein deficiencies

Publications (1)

Publication Number Publication Date
BRPI0407648A true BRPI0407648A (pt) 2006-02-21

Family

ID=32908532

Family Applications (1)

Application Number Title Priority Date Filing Date
BRPI0407648-6A BRPI0407648A (pt) 2003-02-18 2004-02-18 uso de uma proteìna ligante sìtio-especìfica ativa juntamente com um vetor terapêutico codificando uma proteìna para manufatura de um medicamente

Country Status (9)

Country Link
US (1) US7446098B2 (pt)
EP (1) EP1594514A2 (pt)
JP (1) JP2006517980A (pt)
CN (1) CN1750834A (pt)
BR (1) BRPI0407648A (pt)
CA (1) CA2516304A1 (pt)
IL (1) IL169633A0 (pt)
MX (1) MXPA05007821A (pt)
WO (1) WO2004074450A2 (pt)

Families Citing this family (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HUE027569T2 (en) 2006-05-16 2016-10-28 Amicus Therapeutics Inc Preparation for the treatment of Fabry disease
AU2013205532B8 (en) * 2006-05-16 2015-07-16 Amicus Therapeutics, Inc. Assays for diagnosing and evaluating treatment options for fabry disease
AU2007336184A1 (en) * 2006-12-21 2008-06-26 Novo Nordisk A/S Interleukin-21 variants with altered binding to the IL-21 receptor
JP2010525084A (ja) * 2007-04-26 2010-07-22 アミカス セラピューティックス インコーポレイテッド 薬理シャペロンを用いたリソソーム蓄積症治療のための投薬計画
US9999618B2 (en) 2007-04-26 2018-06-19 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
ES2579628T3 (es) 2009-02-23 2016-08-12 Emergent Virology Llc Iminoazúcares y métodos de tratamiento de enfermedades virales
EP2400843B1 (en) * 2009-02-24 2015-11-11 United Therapeutics Corporation Iminosugars and methods of treating arenaviral infections
CA2758271C (en) 2009-04-09 2018-06-05 Robert Boyd Methods for preventing and/or treating lysosomal storage disorders
US20110070643A1 (en) * 2009-05-26 2011-03-24 Do Hung V Utilization of Pharmacological Chaperones to Improve Manufacturing and Purification of Biologics
US9206457B2 (en) 2009-05-26 2015-12-08 Amicus Therapeutics, Inc. Utilization of pharmacological chaperones to improve manufacturing and purification of biologics
ES2524361T3 (es) 2009-06-12 2014-12-05 United Therapeutics Corporation Iminoazúcares para su uso en el tratamiento de enfermedades por bunyavirus y togavirus
EP2473046B1 (en) * 2009-09-04 2014-10-22 United Therapeutics Corporation Iminosugars for their use in the treatment of filoviral diseases
JP5575246B2 (ja) * 2009-09-04 2014-08-20 ユナイテッド セラピューティクス コーポレイション ポックスウイルス感染の治療方法
CA2772875A1 (en) * 2009-09-04 2011-03-10 United Therapeutics Corporation Methods of treating orthomyxoviral infections
EP3143875B1 (en) 2009-10-19 2020-05-27 Amicus Therapeutics, Inc. Novel compositions for preventing and/or treating degenerative disorders of the central nervous system
CA2778349C (en) 2009-10-19 2018-12-04 Amicus Therapeutics, Inc. Novel compositions for preventing and/or treating lysosomal storage disorders
CA2781277C (en) * 2009-11-17 2018-01-09 Baylor Research Institute Urinary triaosylceramide (gb3) as a marker of cardiac disease
US9155784B2 (en) 2011-06-20 2015-10-13 Icahn School Of Medicine At Mount Sinai Anti-TNF-α therapy for the mucopolysaccharidoses and other lysosomal disorders
US10155027B2 (en) 2012-07-17 2018-12-18 Amicus Therapeutics, Inc. Alpha-galactosidase A and 1-deoxygalactonojirimycin co-formulation for the treatment of fabry disease
CA2917995C (en) 2012-07-17 2021-01-26 Amicus Therapeutics, Inc. Alpha-galactosidase a and 1-deoxygalactonojirimycin co-formulation
WO2014071282A1 (en) * 2012-11-05 2014-05-08 Genzyme Corporation Compositions and methods for treating proteinopathies
CA3209821A1 (en) * 2013-05-15 2014-11-20 Regents Of The University Of Minnesota Adeno-associated virus mediated gene transfer to the central nervous system
US9981021B1 (en) 2015-04-09 2018-05-29 Kinetiq, Inc. Subcutaneous therapeutic enzyme formulations, uses, and methods for generating thereof
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
CN105622459B (zh) * 2016-01-11 2017-10-13 中国科学院化学研究所 一种制备打碗花精及其中间体的方法
US12070453B2 (en) 2016-07-19 2024-08-27 Amicus Therapeutics, Inc. Treatment of Fabry disease in ERT-naïve and ERT-experienced patients
IL266047B2 (en) * 2016-10-20 2025-02-01 Sangamo Therapeutics Inc Methods and compositions for the treatment of fabry disease
WO2018200618A1 (en) 2017-04-25 2018-11-01 Amicus Therapeutics, Inc. Novel compositions for preventing and/or treating degenerative disorders of the central nervous system and/or lysosomal storage disorders
BR112020005249A2 (pt) 2017-09-22 2020-09-24 The Trustees Of The University Of Pennsylvania terapia de gene para tratar mucopolissacaridose tipo ii

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0672159B1 (en) * 1992-04-24 2005-12-28 Sri International Homologous sequence targeting in eukaryotic cells
WO1997010817A1 (en) * 1995-09-20 1997-03-27 The Regents Of The University Of Michigan Amino ceramide-like compounds and therapeutic methods of use
US5948653A (en) * 1997-03-21 1999-09-07 Pati; Sushma Sequence alterations using homologous recombination
JP2002522509A (ja) * 1997-10-29 2002-07-23 ジェンザイム・コーポレイション リソソーム貯蔵疾患治療組成物および方法
US6274597B1 (en) * 1998-06-01 2001-08-14 Mount Sinai School Of Medicine Of New York University Method of enhancing lysosomal α-Galactosidase A
US20040204379A1 (en) 2000-06-19 2004-10-14 Cheng Seng H. Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases

Also Published As

Publication number Publication date
JP2006517980A (ja) 2006-08-03
WO2004074450A8 (en) 2004-11-04
WO2004074450A3 (en) 2005-08-25
MXPA05007821A (es) 2006-04-07
EP1594514A2 (en) 2005-11-16
CN1750834A (zh) 2006-03-22
WO2004074450A2 (en) 2004-09-02
US20040219132A1 (en) 2004-11-04
US7446098B2 (en) 2008-11-04
IL169633A0 (en) 2007-07-04
CA2516304A1 (en) 2004-09-02

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Legal Events

Date Code Title Description
B11A Dismissal acc. art.33 of ipl - examination not requested within 36 months of filing
B11Y Definitive dismissal - extension of time limit for request of examination expired [chapter 11.1.1 patent gazette]