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)
  • Biotechnology (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (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)

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MX351004B (es) * 2006-05-16 2017-09-26 Nat Institutes Of Health Ensayos para diagnosticar y evaluar opciones de tratamiento para enfermedad de fabry.
AU2013205532B8 (en) * 2006-05-16 2015-07-16 Amicus Therapeutics, Inc. Assays for diagnosing and evaluating treatment options for fabry disease
EP2109620B1 (en) * 2006-12-21 2012-08-22 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
AU2008245578A1 (en) 2007-04-26 2008-11-06 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
JP5645918B2 (ja) 2009-04-09 2014-12-24 アミカス セラピューティックス インコーポレイテッド リソソーム蓄積障害の予防および／または治療方法
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
WO2010144759A1 (en) * 2009-06-12 2010-12-16 United Therapeutics Corporation Iminosugars and methods of treating bunyaviral and togaviral diseases
EP2473482B1 (en) * 2009-09-04 2014-03-05 United Therapeutics Corporation Methods of treating orthomyxoviral infections
CN102595895A (zh) * 2009-09-04 2012-07-18 联合治疗公司 亚氨基糖以及治疗丝状病毒性疾病的方法
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
RU2015147509A (ru) 2009-10-19 2019-01-14 Амикус Терапьютикс, Инк. Новые композиции для предупреждения и/или лечения дегенеративных расстройств центральной нервной системы
WO2011049737A1 (en) 2009-10-19 2011-04-28 Amicus Therapeutics, Inc. Novel compositions for preventing and/or treating lysosomal storage disorders
EP2502068B1 (en) * 2009-11-17 2016-02-10 Baylor Research Institute Urinary triaosylceramide (gb3) as a marker of cardiac disease
ES2664844T3 (es) 2011-06-20 2018-04-23 Mount Sinai School Of Medicine Tratamiento dirigido contra TNF para las mucopolisacaridosis y otros trastornos lisosómicos
EP2874648A4 (en) 2012-07-17 2015-12-30 Amicus Therapeutics Inc ALPHA GALACTOSIDASE A AND 1-DEOXYGALACTONO JIRIMYCIN CO FORMULATION
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
HK1214521A1 (zh) * 2012-11-05 2016-07-29 Genzyme Corporation 用於治療蛋白質病的組合物和方法
JP2016523835A (ja) * 2013-05-15 2016-08-12 リージェンツ オブ ザ ユニバーシティ オブ ミネソタ 中枢神経系へのアデノ随伴ウイルスを介した遺伝子導入
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
WO2018017721A1 (en) 2016-07-19 2018-01-25 Amicus Therapeutics, Inc. Treatment of fabry disease in ert-naïve and ert-experienced patients
KR102712926B1 (ko) * 2016-10-20 2024-10-07 상가모 테라퓨틱스, 인코포레이티드 파브리병의 치료를 위한 방법 및 조성물
KR20190134807A (ko) 2017-04-25 2019-12-04 아미쿠스 세라퓨틱스, 인코포레이티드 중추 신경계의 퇴행성 질환 및/또는 리소솜 저장병을 예방 및/또는 치료하기 위한 신규한 조성물
EP3684938A1 (en) 2017-09-22 2020-07-29 The Trustees of the University of Pennsylvania Gene therapy for treating mucopolysaccharidosis type ii
CN113423419A (zh) 2019-01-04 2021-09-21 桑格摩生物治疗股份有限公司 用于治疗法布里病的方法和组合物

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AU4115693A (en) * 1992-04-24 1993-11-29 Sri International In vivo homologous sequence targeting in eukaryotic cells
US5916911A (en) * 1995-09-20 1999-06-29 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
EP1027069B1 (en) * 1997-10-29 2006-07-26 Genzyme Corporation Gene therapy for gaucher 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
JP2006517980A (ja) 2006-08-03
CA2516304A1 (en) 2004-09-02
WO2004074450A8 (en) 2004-11-04
MXPA05007821A (es) 2006-04-07
EP1594514A2 (en) 2005-11-16
CN1750834A (zh) 2006-03-22
WO2004074450A2 (en) 2004-09-02
IL169633A0 (en) 2007-07-04
WO2004074450A3 (en) 2005-08-25
US7446098B2 (en) 2008-11-04
US20040219132A1 (en) 2004-11-04

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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]