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)

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AU2013205532B8 (en) * 2006-05-16 2015-07-16 Amicus Therapeutics, Inc. Assays for diagnosing and evaluating treatment options for fabry disease
PL2533050T6 (pl) * 2006-05-16 2016-05-31 Amicus Therapeutics Inc Możliwości leczenia choroby Fabry'ego
US8211420B2 (en) * 2006-12-21 2012-07-03 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
MX2009011473A (es) * 2007-04-26 2010-01-18 Amicus Therapeutics Inc Regimenes de dosificacion para el tratamiento de enfermedades por almacenamiento lisosomal que utilizan chaperonas farmacologicas.
EP2398321B1 (en) 2009-02-23 2015-11-25 United Therapeutics Corporation Iminosugars and methods of treating viral diseases
EP2400843B1 (en) * 2009-02-24 2015-11-11 United Therapeutics Corporation Iminosugars and methods of treating arenaviral infections
BRPI1010517A2 (pt) 2009-04-09 2017-01-31 Amicus Therapeutics Inc métodos para prevenção e/ou tratamento de doenças de armazenamento lisossomal
US9206457B2 (en) 2009-05-26 2015-12-08 Amicus Therapeutics, Inc. Utilization of pharmacological chaperones to improve manufacturing and purification of biologics
AU2010254092B2 (en) * 2009-05-26 2015-11-12 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
CA2772813A1 (en) * 2009-09-04 2011-03-10 The Chancellor, Masters And Scholars Of The University Of Oxford Iminosugars and methods of treating filoviral diseases
JP5653438B2 (ja) * 2009-09-04 2015-01-14 ユナイテッド セラピューティクス コーポレイション オルトミクソウイルス感染の治療方法
CA2772807A1 (en) * 2009-09-04 2011-03-10 The Chancellor, Masters And Scholars Of The University Of Oxford Methods of treating poxviral infections
SI2995306T1 (sl) 2009-10-19 2019-04-30 Amicus Therapeutics, Inc. Novi sestavki za preprečevanje in/ali zdravljenje motenj lizosomskega shranjevanja
KR101880411B1 (ko) 2009-10-19 2018-07-19 아미쿠스 세라퓨틱스, 인코포레이티드 중추신경계의 퇴행성 질환을 예방 및/또는 치료하는 신규 조성물
WO2011063048A2 (en) * 2009-11-17 2011-05-26 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
WO2014014938A1 (en) * 2012-07-17 2014-01-23 Amicus Therapeutics, Inc. Alpha-galactosidase a and 1-deoxygalactonojirimycin co-formulation
AU2013337354A1 (en) * 2012-11-05 2015-05-21 Genzyme Corporation Compositions and methods for treating proteinopathies
CN115120746A (zh) * 2013-05-15 2022-09-30 明尼苏达大学董事会 腺相关病毒介导的基因向中枢神经系统转移
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
TW202408513A (zh) 2016-07-19 2024-03-01 美商阿米庫斯醫療股份有限公司 治療ert初治的和已經歷ert的患者中之法布瑞氏症
KR102712926B1 (ko) * 2016-10-20 2024-10-07 상가모 테라퓨틱스, 인코포레이티드 파브리병의 치료를 위한 방법 및 조성물
CN110573492A (zh) 2017-04-25 2019-12-13 阿米库斯治疗学公司 用于预防和/或治疗中枢神经系统退行性失调和/或溶酶体贮积失调的新颖组合物
KR102883778B1 (ko) 2017-09-22 2025-11-12 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Ii형 점액다당류증의 치료를 위한 유전자 요법

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
IL135578A0 (en) * 1997-10-29 2001-05-20 Genzyme Corp 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
US7446098B2 (en) 2008-11-04
CN1750834A (zh) 2006-03-22
MXPA05007821A (es) 2006-04-07
JP2006517980A (ja) 2006-08-03
WO2004074450A2 (en) 2004-09-02
US20040219132A1 (en) 2004-11-04
CA2516304A1 (en) 2004-09-02
WO2004074450A3 (en) 2005-08-25
IL169633A0 (en) 2007-07-04
EP1594514A2 (en) 2005-11-16

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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]