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
- 229920001184 polypeptide Polymers 0.000 title abstract 7
- 108090000765 processed proteins & peptides Proteins 0.000 title abstract 7
- 102000004196 processed proteins & peptides Human genes 0.000 title abstract 7
- 230000002132 lysosomal effect Effects 0.000 title abstract 6
- 108020004707 nucleic acids Proteins 0.000 abstract 4
- 102000039446 nucleic acids Human genes 0.000 abstract 4
- 150000007523 nucleic acids Chemical class 0.000 abstract 4
- 108020005345 3' Untranslated Regions Proteins 0.000 abstract 3
- 108091026890 Coding region Proteins 0.000 abstract 3
- 239000013598 vector Substances 0.000 abstract 2
- 101710096786 Lysosomal acid alpha-glucosidase Proteins 0.000 abstract 1
- 108010076504 Protein Sorting Signals Proteins 0.000 abstract 1
- 108091036066 Three prime untranslated region Proteins 0.000 abstract 1
- 108091023045 Untranslated Region Proteins 0.000 abstract 1
- 230000007812 deficiency Effects 0.000 abstract 1
- 238000012217 deletion Methods 0.000 abstract 1
- 230000037430 deletion Effects 0.000 abstract 1
- 238000000034 method Methods 0.000 abstract 1
- 239000002773 nucleotide Substances 0.000 abstract 1
- 125000003729 nucleotide group Chemical group 0.000 abstract 1
- 230000003248 secreting effect Effects 0.000 abstract 1
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 (64)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP1436316B1 (de) * | 2001-04-30 | 2008-01-23 | ZyStor Therapeutics , Inc. | Subzelluläres targeting von therapeutischen proteinen |
| 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 |
| US20030072761A1 (en) | 2001-10-16 | 2003-04-17 | Lebowitz Jonathan | Methods and compositions for targeting proteins across the blood brain barrier |
| DE602005020745D1 (de) * | 2004-02-10 | 2010-06-02 | Zystor Therapeutics Inc | Saure alpha-glukosidase und fragmente davon |
| WO2006007560A2 (en) | 2004-07-01 | 2006-01-19 | University Of Pennsylvania | Targeted protein replacement for the treatment of lysosomal storage disorders |
| KR20080038131A (ko) * | 2005-07-18 | 2008-05-02 | 프로탈릭스 리미티드 | 생물학적으로 활성인 거대분자의 점막 또는 장관 투여 |
| 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 |
| AU2007322123A1 (en) * | 2006-11-13 | 2008-05-29 | Biomarin Pharmaceutical Inc. | Methods for treating Pompe disease |
| 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 |
| WO2009137721A2 (en) | 2008-05-07 | 2009-11-12 | Zystor Therapeutics, 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 |
| EP2215981B1 (de) * | 2009-02-04 | 2013-04-03 | Stryker Leibinger GmbH & Co. KG | Chirurgisches Elektrowerkzeug, Betriebsverfahren und Betätigungsbaugruppe hierfür |
| ES2398006T3 (es) * | 2009-02-04 | 2013-03-13 | Stryker Leibinger Gmbh & Co. Kg | Herramienta quirúrgica eléctrica y conjunto de accionamiento para la misma |
| US8785168B2 (en) | 2009-06-17 | 2014-07-22 | Biomarin Pharmaceutical Inc. | Formulations for lysosomal enzymes |
| CA2803166C (en) | 2010-06-25 | 2023-03-07 | Shire Human Genetic Therapies, Inc. | Cns delivery of therapeutic agents |
| KR20130043165A (ko) | 2010-06-25 | 2013-04-29 | 샤이어 휴먼 지네틱 테라피즈 인크. | 이듀로네이트-2-설파타제의 cns 전달을 위한 방법들 및 조성물들 |
| 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 |
| CN103269709A (zh) | 2010-06-25 | 2013-08-28 | 夏尔人类遗传性治疗公司 | Sanfilippo综合症类型B的治疗 |
| BR112012033214B1 (pt) | 2010-06-25 | 2020-11-03 | Shire Human Genetic Therapies, Inc. | uso de uma formulação estável compreendendo uma proteína de iduronato-2-sulfatase (i2s), formulação compreendendo a mesma e recipiente |
| JP6045493B2 (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 | アリールスルファターゼaのcns送達の方法および組成物 |
| US9206401B2 (en) * | 2010-12-22 | 2015-12-08 | Fondazione Telethon | Therapeutic strategies to treat CNS pathology in mucopolysaccharidoses |
| SG10201605874TA (en) * | 2011-04-22 | 2016-09-29 | Genzyme Corp | Modified acid alpha glucosidase with accelerated processing |
| EA201892619A1 (ru) | 2011-04-29 | 2019-04-30 | Роше Гликарт Аг | Иммуноконъюгаты, содержащие мутантные полипептиды интерлейкина-2 |
| US10350277B2 (en) | 2011-09-07 | 2019-07-16 | Icahn School Of Medicine At Mount Sinai | Ceramidase and cell differentiation |
| WO2013055888A2 (en) * | 2011-10-12 | 2013-04-18 | Synageva Biopharma Corp. | Recombinant human naglu protein and uses thereof |
| US20140377244A1 (en) | 2011-12-23 | 2014-12-25 | Shire Human Genetic Therapies, Inc. | Stable formulations for cns delivery of arylsulfatase a |
| ES2786128T3 (es) | 2012-06-01 | 2020-10-08 | Icahn School Med Mount Sinai | Niveles de ceramida en el tratamiento y prevención de infecciones |
| EP4124346A3 (de) | 2012-06-19 | 2023-04-19 | University of Florida Research Foundation, Inc. | Zusammensetzungen und verfahren zur behandlung von krankheiten |
| JP2016514096A (ja) | 2013-02-20 | 2016-05-19 | バレリオン セラピューティクス, エルエルシー | ポンぺ病の処置のための方法および組成物 |
| JP6832158B2 (ja) | 2013-03-14 | 2021-02-24 | アイカーン スクール オブ メディシン アット マウント サイナイ | 治療用酸性セラミダーゼ組成物ならびにそれを作製および使用する方法 |
| EP3270894B1 (de) * | 2015-03-19 | 2021-02-24 | Translate Bio, Inc. | Mrna-therapie für pompe-krankheit |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| EP3293259A1 (de) | 2016-09-12 | 2018-03-14 | Genethon | Saure alpha-glucosidase-varianten und verwendungen davon |
| KR102617502B1 (ko) * | 2016-09-12 | 2023-12-26 | 제네똥 | 산-알파 글루코시다제 변이체 및 이의 용도 |
| EP3293203A1 (de) | 2016-09-12 | 2018-03-14 | Genethon | Saure alpha-glucosidase-varianten und verwendungen davon |
| EP3293260A1 (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 |
| AU2018281280B2 (en) | 2017-06-07 | 2024-12-19 | Regeneron Pharmaceuticals, Inc. | Compositions and methods for internalizing enzymes |
| CN112040985A (zh) | 2018-02-07 | 2020-12-04 | 瑞泽恩制药公司 | 用于递送治疗性蛋白质的方法和组合物 |
| TW202344691A (zh) | 2018-04-30 | 2023-11-16 | 美商阿米庫斯醫療股份有限公司 | 基因治療構築體及使用方法 |
| EP3793591A1 (de) | 2018-05-17 | 2021-03-24 | Regeneron Pharmaceuticals, Inc. | Anti-cd63-antikörper, konjugate und verwendungen davon |
| JP7577650B2 (ja) | 2018-10-10 | 2024-11-05 | アミカス セラピューティックス インコーポレイテッド | ジスルフィド結合により安定したポリペプチド組成物及びその使用方法 |
| US20220002752A1 (en) * | 2018-11-13 | 2022-01-06 | Denovo Biopharma Llc | Recombinant vectors comprising genes for binding domains and secretable peptides |
| WO2020102645A1 (en) * | 2018-11-16 | 2020-05-22 | Asklepios Biopharmaceutical, Inc. | Therapeutic adeno-associated virus for treating pompe disease |
| CA3120087A1 (en) * | 2018-11-16 | 2020-05-22 | Asklepios Biopharmaceutical, Inc. | Vectors comprising a nucleic acid encoding lysosomal enzymes fused to a lysosomal targeting sequence |
| US20220193261A1 (en) * | 2019-04-30 | 2022-06-23 | The Trustees Of The University Of Pennsylvania | Compositions useful for treatment of pompe disease |
| WO2021041986A1 (en) * | 2019-08-28 | 2021-03-04 | University Of Florida Research Foundation, Incorporated | Improved production of recombinant aav using embryonated avian eggs |
| WO2021078834A1 (en) | 2019-10-22 | 2021-04-29 | Genethon | Chimeric acid-alpha glucosidase polypeptides and uses thereof |
| US12662512B2 (en) * | 2019-10-22 | 2026-06-23 | 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 |
| MX2022007399A (es) | 2019-12-20 | 2022-09-19 | Codexis Inc | Variantes de alfa-glucosidasa ácida diseñadas. |
| WO2022036193A2 (en) * | 2020-08-13 | 2022-02-17 | Epivax, Inc. | Regulatory t cell epitopes |
| US20240374507A1 (en) * | 2021-01-26 | 2024-11-14 | Sigilon Therapeutics, Inc. | Compositions, devices and methods for inducing antigen-specific immune tolerance |
| CN116981473A (zh) * | 2021-01-26 | 2023-10-31 | 西吉隆医疗股份有限公司 | 用于治疗免疫介导的炎性疾病的组合物、装置和方法 |
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| AU2022379625A1 (en) * | 2021-10-27 | 2024-05-16 | The University Of North Carolina At Chapel Hill | Aav-idua vector for treatment of mps i |
| CA3254578A1 (en) * | 2022-03-18 | 2023-09-21 | AskBio Inc. | Adeno-associated virus therapy involving an alpha-glucosidase (GAA) codon-optimized nucleic acid to treat Pompe disease, with signal peptide modifications |
| US20260092290A1 (en) * | 2022-12-21 | 2026-04-02 | National Taiwan University | Recombinant viral vector, recombinant adeno-associated virus comprising the same, and uses thereof in treating sialidosis |
| WO2025072816A2 (en) * | 2023-09-29 | 2025-04-03 | The Board Of Regents Of The University Of Texas System | Endogenous nucleus targeting signal peptides and uses thereof |
| AU2024359822A1 (en) * | 2023-10-09 | 2026-04-30 | The Board Of Regents Of The University Of Texas System | Endogenous cell membrane targeting signal peptides and uses thereof |
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-
2004
- 2004-01-21 PT PT04704039T patent/PT1587923E/pt unknown
- 2004-01-21 ES ES04704039T patent/ES2371913T3/es not_active Expired - Lifetime
- 2004-01-21 JP JP2006501055A patent/JP5433133B2/ja not_active Expired - Lifetime
- 2004-01-21 AT AT04704039T patent/ATE521701T1/de not_active IP Right Cessation
- 2004-01-21 WO PCT/US2004/001453 patent/WO2004064750A2/en not_active Ceased
- 2004-01-21 EP EP04704039A patent/EP1587923B1/de not_active Expired - Lifetime
- 2004-01-21 US US10/761,530 patent/US20040248262A1/en not_active Abandoned
-
2012
- 2012-02-23 US US13/403,783 patent/US9873868B2/en not_active Expired - Fee Related
-
2018
- 2018-01-22 US US15/877,386 patent/US20180371440A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| EP1587923A2 (de) | 2005-10-26 |
| US20180371440A1 (en) | 2018-12-27 |
| WO2004064750A2 (en) | 2004-08-05 |
| WO2004064750A3 (en) | 2004-11-11 |
| US9873868B2 (en) | 2018-01-23 |
| EP1587923B1 (de) | 2011-08-24 |
| US20120276072A1 (en) | 2012-11-01 |
| ES2371913T3 (es) | 2012-01-11 |
| JP5433133B2 (ja) | 2014-03-05 |
| PT1587923E (pt) | 2011-12-07 |
| JP2006517404A (ja) | 2006-07-27 |
| US20040248262A1 (en) | 2004-12-09 |
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