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

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Publication number Priority date Publication date Assignee Title
AU2007253900A1 (en) 2006-05-16 2007-11-29 Amicus Therapeutics, Inc. Assays for diagnosing and evaluating treatment options for 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
US9999618B2 (en) 2007-04-26 2018-06-19 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
EP2150254A4 (en) * 2007-04-26 2010-11-10 Amicus Therapeutics Inc DOSAGES FOR THE TREATMENT OF LYSOSOMAL STORAGE DISEASES WITH PHARMACOLOGICAL CHAPTERONES
JP5600329B2 (ja) 2009-02-23 2014-10-01 ユナイテッド セラピューティクス コーポレーション イミノ糖およびウイルス性疾患を治療する方法
ES2562635T3 (es) * 2009-02-24 2016-03-07 United Therapeutics Corporation Iminoazúcares y métodos de tratamiento de infecciones producidas por arenavirus
CN102625661B (zh) 2009-04-09 2015-05-27 阿米库斯治疗学公司 用于预防和/或治疗溶酶体贮积失调的方法
US9206457B2 (en) 2009-05-26 2015-12-08 Amicus Therapeutics, Inc. Utilization of pharmacological chaperones to improve manufacturing and purification of biologics
EP2435459B1 (en) * 2009-05-26 2016-05-04 Amicus Therapeutics, Inc. Utilization of pharmacological chaperones to improve manufacturing and purification of acid beta-glucosidase
ES2524361T3 (es) * 2009-06-12 2014-12-05 United Therapeutics Corporation Iminoazúcares para su uso en el tratamiento de enfermedades por bunyavirus y togavirus
ES2527623T3 (es) * 2009-09-04 2015-01-27 United Therapeutics Corporation Iminoazúcares para su uso en el tratamiento de enfermedades por filovirus
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
KR101497194B1 (ko) * 2009-09-04 2015-02-27 유나이티드 세러퓨틱스 코오포레이션 오르토믹소바이러스 감염의 치료 방법
DK2490532T3 (en) 2009-10-19 2017-02-27 Amicus Therapeutics Inc Hitherto UNKNOWN COMPOSITIONS FOR PREVENTION AND / OR TREATMENT OF DEGENERATIVE DISORDERS IN THE CENTRAL Nervous System
ES2555533T3 (es) 2009-10-19 2016-01-04 Amicus Therapeutics, Inc. Nuevas composiciones para prevenir y/o tratar trastornos de almacenamiento lisosomal
AR079057A1 (es) * 2009-11-17 2011-12-21 Baylor Res Inst Triaosilceramida urinaria (gb3) como marcador de enfermedad cardiaca
EP2721151B1 (en) 2011-06-20 2017-12-06 Mount Sinai School Of Medicine Anti-tnf-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
US9694056B2 (en) 2012-07-17 2017-07-04 Amicus Therapeutics, Inc. α-galactosidase A and 1-deoxygalactonojirimycin co-formulation
JP2016503405A (ja) * 2012-11-05 2016-02-04 ジェンザイム・コーポレーション タンパク質症を処置するための組成物および方法
BR112015028605A8 (pt) * 2013-05-15 2019-12-24 Univ Minnesota uso de uma composição e uso de um imunossupressor e uma composição
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
CA3031249A1 (en) 2016-07-19 2018-01-25 Amicus Therapeutics, Inc. Treatment of fabry disease in ert-naive and ert-experienced patients
US11219695B2 (en) * 2016-10-20 2022-01-11 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of Fabry disease
CA3061354A1 (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
AU2018335752A1 (en) 2017-09-22 2020-03-12 Christian HINDERER Gene therapy for treating Mucopolysaccharidosis type ii

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1993022443A1 (en) * 1992-04-24 1993-11-11 Sri International In vivo 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
EP2147681A1 (en) * 1997-10-29 2010-01-27 Genzyme Corporation Compositions and methods for treating lysosomal storage disease
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
WO2004074450A8 (en) 2004-11-04
CN1750834A (zh) 2006-03-22
US20040219132A1 (en) 2004-11-04
CA2516304A1 (en) 2004-09-02
JP2006517980A (ja) 2006-08-03
IL169633A0 (en) 2007-07-04
EP1594514A2 (en) 2005-11-16
US7446098B2 (en) 2008-11-04
MXPA05007821A (es) 2006-04-07
WO2004074450A2 (en) 2004-09-02
WO2004074450A3 (en) 2005-08-25

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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]