ATE521701T1 - Verbesserte konstrukte zur expression lysosomaler polypeptide - Google Patents
Verbesserte konstrukte zur expression lysosomaler polypeptideInfo
- Publication number
- ATE521701T1 ATE521701T1 AT04704039T AT04704039T ATE521701T1 AT E521701 T1 ATE521701 T1 AT E521701T1 AT 04704039 T AT04704039 T AT 04704039T AT 04704039 T AT04704039 T AT 04704039T AT E521701 T1 ATE521701 T1 AT E521701T1
- Authority
- AT
- Austria
- Prior art keywords
- gaa
- lysosomal
- utr
- isolated nucleic
- polypeptide
- Prior art date
Links
Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
- C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
- C12N9/2405—Glucanases
- C12N9/2408—Glucanases acting on alpha -1,4-glucosidic bonds
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/036—Fusion polypeptide containing a localisation/targetting motif targeting to the medium outside of the cell, e.g. type III secretion
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Genetics & Genomics (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Animal Behavior & Ethology (AREA)
- Obesity (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Diabetes (AREA)
- Pharmacology & Pharmacy (AREA)
- Hematology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US44178903P | 2003-01-22 | 2003-01-22 | |
PCT/US2004/001453 WO2004064750A2 (en) | 2003-01-22 | 2004-01-21 | Improved constructs for expressing lysosomal polypeptides |
Publications (1)
Publication Number | Publication Date |
---|---|
ATE521701T1 true ATE521701T1 (de) | 2011-09-15 |
Family
ID=32771976
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
AT04704039T ATE521701T1 (de) | 2003-01-22 | 2004-01-21 | Verbesserte konstrukte zur expression lysosomaler polypeptide |
Country Status (7)
Country | Link |
---|---|
US (3) | US20040248262A1 (de) |
EP (1) | EP1587923B1 (de) |
JP (1) | JP5433133B2 (de) |
AT (1) | ATE521701T1 (de) |
ES (1) | ES2371913T3 (de) |
PT (1) | PT1587923E (de) |
WO (1) | WO2004064750A2 (de) |
Families Citing this family (52)
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US7629309B2 (en) * | 2002-05-29 | 2009-12-08 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
US7560424B2 (en) | 2001-04-30 | 2009-07-14 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
CA2445577C (en) * | 2001-04-30 | 2012-07-03 | Symbiontics, Inc. | Subcellular targeting of therapeutic proteins |
US20030072761A1 (en) | 2001-10-16 | 2003-04-17 | Lebowitz Jonathan | Methods and compositions for targeting proteins across the blood brain barrier |
WO2005078077A2 (en) * | 2004-02-10 | 2005-08-25 | Zystor Therapeutics, Inc. | Acid alpha-glucosidase and fragments thereof |
US8889127B2 (en) | 2004-07-01 | 2014-11-18 | Icahn School Of Medicine At Mount Sinai | Targeted protein replacement for the treatment of lysosomal storage disorders |
EP1904638A2 (de) * | 2005-07-18 | 2008-04-02 | Metabogal Ltd. | Mukosale oder enterale verabreichung biologisch aktiver makromoleküle |
FR2889066B1 (fr) * | 2005-07-28 | 2007-11-09 | Centre Nat Rech Scient | Procede d'immunisation genetique par electrotransfert contre une toxine et antiserum susceptible d'etre obtenu par ledit procede |
JP2010509344A (ja) * | 2006-11-13 | 2010-03-25 | ザイストール セラピューティクス, インコーポレイテッド | ポンペ病を治療するための方法 |
US20120322861A1 (en) * | 2007-02-23 | 2012-12-20 | Barry John Byrne | Compositions and Methods for Treating Diseases |
WO2009075815A1 (en) | 2007-12-07 | 2009-06-18 | Duke University | Immunomodulating gene therapy |
DK2279210T3 (en) | 2008-05-07 | 2017-07-24 | Biomarin Pharm Inc | Lysosomal targeting peptides and uses thereof |
US20110318322A1 (en) | 2009-01-12 | 2011-12-29 | Nektar Therapeutics | Conjugates of a Lysosomal Enzyme Moiety and a Water Soluble Polymer |
ES2398006T3 (es) * | 2009-02-04 | 2013-03-13 | Stryker Leibinger Gmbh & Co. Kg | Herramienta quirúrgica eléctrica y conjunto de accionamiento para la misma |
ES2405550T3 (es) * | 2009-02-04 | 2013-05-31 | Stryker Leibinger Gmbh & Co. Kg | Herramienta eléctrica quirúrgica, procedimiento de funcionamiento y grupo constructivo de accionamiento correspondientes |
ES2569514T3 (es) | 2009-06-17 | 2016-05-11 | Biomarin Pharmaceutical Inc. | Formulaciones para enzimas lisosómicas |
JP6045492B2 (ja) | 2010-06-25 | 2016-12-14 | シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド | イズロン酸−2−スルファターゼのcns送達のための方法および組成物 |
NZ605871A (en) | 2010-06-25 | 2015-02-27 | Shire Human Genetic Therapies | Treatment of sanfilippo syndrome type b |
DK2585104T3 (da) | 2010-06-25 | 2019-10-14 | Shire Human Genetic Therapies | Fremgangsmåder og sammensætninger tilcns-levering af arylsulfatase a |
DK3103469T3 (en) | 2010-06-25 | 2021-02-22 | Shire Human Genetic Therapies | Indgivelse af terapeutiske midler til centralnervesystemet |
JP6073783B2 (ja) | 2010-06-25 | 2017-02-01 | シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド | ヘパランn−スルファターゼのcns送達のための方法および組成物 |
IL291554B2 (en) | 2010-06-25 | 2024-02-01 | Shire Human Genetic Therapies | Administering therapeutic agents to the central nervous system |
WO2012085622A1 (en) | 2010-12-22 | 2012-06-28 | Fondazione Telethon | Therapeutic strategies to treat cns pathology in mucopolysaccharidoses |
WO2012145644A1 (en) * | 2011-04-22 | 2012-10-26 | Genzyme Corporation | Modified acid alpha glucosidase with accelerated processing |
EA201892619A1 (ru) | 2011-04-29 | 2019-04-30 | Роше Гликарт Аг | Иммуноконъюгаты, содержащие мутантные полипептиды интерлейкина-2 |
DK2753346T3 (da) | 2011-09-07 | 2020-05-25 | Sinai School Medicine | Ceramidase og celledifferentiering |
JP6329483B2 (ja) | 2011-10-12 | 2018-05-23 | シナジェバ・バイオファーマ・コーポレイションSynageva Biopharma Corp. | 組換えヒトnagluタンパク質およびその利用 |
EP2793922B1 (de) | 2011-12-23 | 2019-10-30 | Shire Human Genetic Therapies, Inc. | Stabile formulierungen zur verabreichung von arylsulfatase a in das zns |
HUE051020T2 (hu) | 2012-06-01 | 2021-01-28 | Icahn School Med Mount Sinai | A ceramid szintjei a fertõzések kezelésében és megelõzésében |
MX2014015985A (es) | 2012-06-19 | 2015-07-21 | Univ Florida | Composiciones y metodos para tratar enfermedades. |
AU2014218854B2 (en) | 2013-02-20 | 2019-01-24 | Valerion Therapeutics, Llc | Methods and compositions for treatment of Pompe disease |
ES2739811T3 (es) | 2013-03-14 | 2020-02-04 | Icahn School Med Mount Sinai | Composiciones terapéuticas de ceramidasa ácida y métodos de fabricación y uso de ellas |
WO2016149508A1 (en) | 2015-03-19 | 2016-09-22 | Shire Human Genetic Therapies, Inc. | Mrna therapy for pompe disease |
GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
EP3293260A1 (de) | 2016-09-12 | 2018-03-14 | Genethon | Saure alpha-glucosidase-varianten und verwendungen davon |
EP3510149A1 (de) * | 2016-09-12 | 2019-07-17 | Genethon | Saure alpha-glucosidase-varianten und verwendungen davon |
EP3293203A1 (de) | 2016-09-12 | 2018-03-14 | Genethon | Saure alpha-glucosidase-varianten und verwendungen davon |
EP3293259A1 (de) * | 2016-09-12 | 2018-03-14 | Genethon | Saure alpha-glucosidase-varianten und verwendungen davon |
US11160493B2 (en) * | 2017-03-03 | 2021-11-02 | Musclesound, Inc. | System and method for determining a subject's muscle fuel level, muscle fuel rating, and muscle energy status |
BR112019025888A2 (pt) | 2017-06-07 | 2020-06-30 | Regeneron Pharmaceuticals, Inc. | polinucleotídeo que codifica uma proteína terapêutica multidomínio, vetor de terapia gênica, proteína terapêutica multidomínio recombinante, método de expressão, métodos para reduzir o acúmulo de glicogênio em um tecido em um paciente em necessidade, para reduzir o acúmulo de glicogênio em um tecido em um paciente em necessidade epara tratar a deficiência enzimática em um paciente em necessidade e/ou tolerar o paciente à enzima para a qual é deficiente, anticorpo anti-cd63 ou fragmento de ligação a antígeno, e, composição farmacêutica |
BR112020021962A2 (pt) | 2018-04-30 | 2021-01-26 | Amicus Therapeutics, Inc. | construtos para terapia gênica e métodos de uso |
CA3115944A1 (en) | 2018-10-10 | 2020-04-16 | Amicus Therapeutics, Inc. | Disulfide bond stabilized polypeptide compositions and methods of use |
CN113677800A (zh) * | 2018-11-13 | 2021-11-19 | 索元生物医药(美国)有限公司 | 包含用于结合结构域和可分泌肽的基因的重组载体 |
WO2020102645A1 (en) * | 2018-11-16 | 2020-05-22 | Asklepios Biopharmaceutical, Inc. | Therapeutic adeno-associated virus for treating pompe disease |
WO2020102667A2 (en) * | 2018-11-16 | 2020-05-22 | Asklepios Biopharmaceutical, Inc. | Vectors comprising a nucleic acid encoding lysosomal enzymes fused to a lysosomal targeting sequence |
BR112021021792A2 (pt) * | 2019-04-30 | 2022-01-04 | Amicus Therapeutics Inc | Composições úteis para tratamento de doença de pompe |
WO2021078834A1 (en) | 2019-10-22 | 2021-04-29 | Genethon | Chimeric acid-alpha glucosidase polypeptides and uses thereof |
WO2021078833A1 (en) | 2019-10-22 | 2021-04-29 | Genethon | Chimeric polypeptides and uses thereof |
WO2021102107A1 (en) * | 2019-11-19 | 2021-05-27 | Asklepios Biopharmaceutical, Inc. | Therapeutic adeno-associated virus comprising liver-specific promoters for treating pompe disease and lysosomal disorders |
US20230321237A1 (en) * | 2020-08-13 | 2023-10-12 | Epivax, Inc. | Regulatory t cell epitopes |
CA3236188A1 (en) * | 2021-10-27 | 2023-05-04 | The University Of North Carolina At Chapel Hill | Aav-idua vector for treatment of mps i |
TW202403048A (zh) * | 2022-03-18 | 2024-01-16 | 美商艾司科皮歐斯生物製藥有限公司 | 用於治療龐貝症之具有訊號肽修飾之以密碼子最佳化核酸編碼α-葡萄糖苷酶(GAA)之治療性腺相關病毒 |
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US7858367B2 (en) | 2002-04-30 | 2010-12-28 | Duke University | Viral vectors and methods for producing and using the same |
EP1594530A4 (de) | 2003-01-22 | 2006-10-11 | Human Genome Sciences Inc | Albuminfusionsproteine |
-
2004
- 2004-01-21 AT AT04704039T patent/ATE521701T1/de not_active IP Right Cessation
- 2004-01-21 ES ES04704039T patent/ES2371913T3/es not_active Expired - Lifetime
- 2004-01-21 PT PT04704039T patent/PT1587923E/pt unknown
- 2004-01-21 US US10/761,530 patent/US20040248262A1/en not_active Abandoned
- 2004-01-21 EP EP04704039A patent/EP1587923B1/de not_active Expired - Lifetime
- 2004-01-21 JP JP2006501055A patent/JP5433133B2/ja not_active Expired - Lifetime
- 2004-01-21 WO PCT/US2004/001453 patent/WO2004064750A2/en active Application Filing
-
2012
- 2012-02-23 US US13/403,783 patent/US9873868B2/en not_active Expired - Lifetime
-
2018
- 2018-01-22 US US15/877,386 patent/US20180371440A1/en not_active Abandoned
Also Published As
Publication number | Publication date |
---|---|
EP1587923B1 (de) | 2011-08-24 |
WO2004064750A3 (en) | 2004-11-11 |
ES2371913T3 (es) | 2012-01-11 |
JP2006517404A (ja) | 2006-07-27 |
US9873868B2 (en) | 2018-01-23 |
US20180371440A1 (en) | 2018-12-27 |
US20040248262A1 (en) | 2004-12-09 |
WO2004064750A2 (en) | 2004-08-05 |
JP5433133B2 (ja) | 2014-03-05 |
EP1587923A2 (de) | 2005-10-26 |
PT1587923E (pt) | 2011-12-07 |
US20120276072A1 (en) | 2012-11-01 |
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RER | Ceased as to paragraph 5 lit. 3 law introducing patent treaties |