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)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (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)
  • 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)

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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
JP2009537149A (ja) 2006-05-16 2009-10-29 アミークス セラピューティックス インコーポレイテッド ファブリー病に対する治療の選択を診断し、評価するためのアッセイ
WO2008074863A1 (en) * 2006-12-21 2008-06-26 Novo Nordisk A/S Interleukin-21 variants with altered binding to the il-21 receptor
WO2008134628A2 (en) 2007-04-26 2008-11-06 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
US9999618B2 (en) 2007-04-26 2018-06-19 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
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
KR101787434B1 (ko) * 2009-02-24 2017-11-15 유나이티드 세러퓨틱스 코오포레이션 이미노슈가 및 아레나바이러스성 감염을 치료하는 방법
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
WO2010138608A1 (en) * 2009-05-26 2010-12-02 Amicus Therapeutics, Inc. Utilization of pharmacological chaperones to improve manufacturing and purification of biologics
JP5634510B2 (ja) 2009-06-12 2014-12-03 ユナイテッド セラピューティクス コーポレイション イミノ糖ならびにブニヤウイルスおよびトガウイルス疾患を治療する方法
JP5752689B2 (ja) * 2009-09-04 2015-07-22 ユナイテッド セラピューティクス コーポレイション フィロウイルス疾患を治療するイミノ糖および方法
KR101459530B1 (ko) * 2009-09-04 2014-11-07 유나이티드 세러퓨틱스 코오포레이션 폭스바이러스 감염의 치료 방법
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
PT2995306T (pt) 2009-10-19 2019-03-29 Amicus Therapeutics Inc Novas composições para prevenção e/ou tratamento de disfunções de armazenamento lisossomal
AR079057A1 (es) * 2009-11-17 2011-12-21 Baylor Res Inst Triaosilceramida urinaria (gb3) como marcador de enfermedad cardiaca
WO2012177778A1 (en) 2011-06-20 2012-12-27 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
HK1209625A1 (en) * 2012-07-17 2016-04-08 Amicus Therapeutics, Inc. Alpha-galactosidase a and 1-deoxygalactonojirimycin co-formulation
BR112015009746A2 (pt) * 2012-11-05 2017-08-15 Genzyme Corp Agente que aumenta atividade de glicocerebrosidase em mamíferos
KR20160010526A (ko) * 2013-05-15 2016-01-27 리젠츠 오브 더 유니버시티 오브 미네소타 중추 신경계로의 아데노-연관 바이러스 매개 유전자 전달
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
AU2017298376A1 (en) 2016-07-19 2019-03-07 Amicus Therapeutics, Inc. Treatment of Fabry disease in ERT-naive and ERT-experienced patients
CN110022904B (zh) * 2016-10-20 2024-04-19 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
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
JP7449223B2 (ja) 2017-09-22 2024-03-13 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ムコ多糖症ｉｉ型を治療するための遺伝子療法

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
US6066626A (en) * 1997-10-29 2000-05-23 Genzyme Corporation Compositions and method 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
EP1594514A2 (en) 2005-11-16
US7446098B2 (en) 2008-11-04
WO2004074450A2 (en) 2004-09-02
CA2516304A1 (en) 2004-09-02
WO2004074450A3 (en) 2005-08-25
IL169633A0 (en) 2007-07-04
CN1750834A (zh) 2006-03-22
WO2004074450A8 (en) 2004-11-04
JP2006517980A (ja) 2006-08-03
US20040219132A1 (en) 2004-11-04
MXPA05007821A (es) 2006-04-07

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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]