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)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Genetics & Genomics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- General Chemical & Material Sciences (AREA)
- Obesity (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Veterinary Medicine (AREA)
- Hematology (AREA)
- Diabetes (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 |
|---|---|---|---|---|
| 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 |
| EP1974752B1 (de) * | 2001-04-30 | 2012-09-26 | BioMarin Pharmaceutical Inc. | Subzelluläres Targeting von therapeutischen Proteinen |
| US20030072761A1 (en) | 2001-10-16 | 2003-04-17 | Lebowitz Jonathan | Methods and compositions for targeting proteins across the blood brain barrier |
| CA2553955C (en) * | 2004-02-10 | 2012-08-28 | 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 |
| JP2009501784A (ja) * | 2005-07-18 | 2009-01-22 | プロタリクス リミテッド | 生物学的に活性な高分子の粘膜投与または経腸投与 |
| 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 |
| CA2669347A1 (en) * | 2006-11-13 | 2008-05-29 | Zystor Therapeutics, 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 |
| ES2629853T3 (es) * | 2008-05-07 | 2017-08-16 | Biomarin Pharmaceutical Inc. | Péptidos de dirección lisosómica y usos de los mismos |
| WO2010080720A2 (en) | 2009-01-12 | 2010-07-15 | 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 |
| EP2475376B1 (de) | 2009-06-17 | 2016-03-30 | BioMarin Pharmaceutical Inc. | Formulierungen für lysosomale enzyme |
| UA115649C2 (uk) | 2010-06-25 | 2017-12-11 | Шае Хюмен Дженетік Терапіс, Інк. | Способи та композиції для доставки до цнс ідуронат-2-сульфатази |
| AU2011270670B2 (en) | 2010-06-25 | 2017-01-12 | Takeda Pharmaceutical Company Limited | Methods and compositions for CNS delivery of arylsulfatase A |
| ES2896060T3 (es) | 2010-06-25 | 2022-02-23 | Shire Human Genetic Therapies | Métodos y composiciones para la administración al SNC de arilsulfatasa A |
| CA2805413A1 (en) | 2010-06-25 | 2011-12-29 | Shire Human Genetic Therapies, Inc. | Methods and compositions for cns delivery of heparan n-sulfatase |
| NZ702801A (en) | 2010-06-25 | 2016-08-26 | Shire Human Genetic Therapies | Treatment of sanfilippo syndrome type b |
| EP2588130B1 (de) | 2010-06-25 | 2016-08-17 | Shire Human Genetic Therapies, Inc. | Abgabe therapeutischer mittel in das zns |
| AU2010366066B2 (en) | 2010-12-22 | 2016-01-14 | Fondazione Telethon | Therapeutic strategies to treat CNS pathology in mucopolysaccharidoses |
| RU2013151875A (ru) * | 2011-04-22 | 2015-05-27 | Джензим Корпорейшн | Модифицированная кислая альфа глюкозидаза с ускоренным процессингом |
| EA201892619A1 (ru) | 2011-04-29 | 2019-04-30 | Роше Гликарт Аг | Иммуноконъюгаты, содержащие мутантные полипептиды интерлейкина-2 |
| EP3741384A1 (de) | 2011-09-07 | 2020-11-25 | Mount Sinai School Of Medicine | Ceramidase und zelldifferenzierung |
| CN103958676A (zh) * | 2011-10-12 | 2014-07-30 | 辛那杰瓦生物制药股份有限公司 | 重组人naglu蛋白质及其用途 |
| WO2013096899A2 (en) | 2011-12-23 | 2013-06-27 | Shire Human Genetic Therapies, Inc. | Stable formulations for cns delivery of arylsulfatase a |
| CN109529028A (zh) | 2012-06-01 | 2019-03-29 | 西奈山伊坎医学院 | 在感染的治疗和预防中的神经酰胺水平 |
| SMT202000445T1 (it) | 2012-06-19 | 2020-09-10 | Univ Florida | Composizioni e metodi per trattare patologie |
| WO2014130723A1 (en) | 2013-02-20 | 2014-08-28 | Valerion Therapeutics, Llc | Methods and compositions for treatment of pompe disease |
| WO2014160390A1 (en) | 2013-03-14 | 2014-10-02 | Icahn School Of Medicine At Mount Sinai | Therapeutic acid ceramidase compositions and methods of making and using them |
| 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 |
| EP3293259A1 (de) | 2016-09-12 | 2018-03-14 | Genethon | Saure alpha-glucosidase-varianten und verwendungen davon |
| PE20191203A1 (es) | 2016-09-12 | 2019-09-10 | Genethon | Variantes de alfa-glucosidasa acida y usos de las mismas |
| 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 |
| ES3065816T3 (en) | 2017-06-07 | 2026-05-08 | Regeneron Pharma | 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 |
| JP7749322B2 (ja) | 2018-04-30 | 2025-10-06 | アミカス セラピューティックス インコーポレイテッド | 遺伝子治療構築物及び使用方法 |
| BR112020023145A2 (pt) | 2018-05-17 | 2021-02-02 | Regeneron Pharmaceuticals, Inc. | anticorpo anti-cd63 ou fragmento de ligação ao antígeno do mesmo, molécula de ligação ao antígeno biespecífica, proteína terapêutica de múltiplos domínios, polinucleotídeo composição farmacêutica, e, composto |
| EA202190999A1 (ru) | 2018-10-10 | 2021-10-15 | Амикус Терапьютикс, Инк. | Композиции на основе полипептидов, стабилизированных с помощью дисульфидной связи, и способы применения |
| JP2022507268A (ja) * | 2018-11-13 | 2022-01-18 | デノボ バイオファーマ エルエルシー | 結合ドメインおよび分泌可能なペプチドの遺伝子を含む組換えベクター |
| EP3880830A4 (de) * | 2018-11-16 | 2022-08-24 | Asklepios Biopharmaceutical, Inc. | Vektoren mit einer nukleinsäure codierend für lysosomale enzyme kondensiert an eine lysosomale targeting-sequenz |
| AU2019381776A1 (en) * | 2018-11-16 | 2021-07-01 | AskBio Inc. | Therapeutic adeno-associated virus for treating Pompe disease |
| CA3134485A1 (en) * | 2019-04-30 | 2020-11-05 | James M. Wilson | Compositions useful for treatment of pompe disease |
| AU2020338014A1 (en) * | 2019-08-28 | 2022-03-03 | University Of Florida Research Foundation, Incorporated | Improved production of recombinant AAV using embryonated avian eggs |
| CN114555808A (zh) | 2019-10-22 | 2022-05-27 | 吉尼松公司 | 嵌合多肽及其用途 |
| WO2021078834A1 (en) | 2019-10-22 | 2021-04-29 | Genethon | Chimeric acid-alpha glucosidase polypeptides and uses thereof |
| PH12022551229A1 (en) * | 2019-11-19 | 2023-07-31 | Asklepios Biopharmaceutical Inc | Therapeutic adeno-associated virus comprising liver-specific promoters for treating pompe disease and lysosomal disorders |
| PE20221266A1 (es) | 2019-12-20 | 2022-08-16 | Codexis Inc | Variantes de alfa-glucosidasa acida disenadas por ingenieria |
| JP2023538002A (ja) * | 2020-08-13 | 2023-09-06 | エピバックス・インコーポレーテッド | 制御性t細胞エピトープ |
| CN117042723A (zh) * | 2021-01-26 | 2023-11-10 | 西吉隆医疗股份有限公司 | 用于诱导抗原特异性免疫耐受的组合物、装置和方法 |
| WO2022164928A2 (en) * | 2021-01-26 | 2022-08-04 | Sigilon Therapeutics, Inc. | Compositions, devices and methods for treating immune-mediated inflammatory diseases |
| CN116964201A (zh) * | 2021-02-05 | 2023-10-27 | 西吉隆医疗股份有限公司 | 用于治疗mps vi疾病的组合物、装置和方法 |
| WO2023076972A1 (en) * | 2021-10-27 | 2023-05-04 | The University Of North Carolina At Chapel Hill | Aav-idua vector for treatment of mps i |
| US20250213726A1 (en) * | 2022-03-18 | 2025-07-03 | Asklepios Biopharmaceutical, Inc. | Therapeutic adeno-associated virus using codon optimized nucleic acid encoding alpha-glucosidase (gaa) for treating pompe disease, with signal peptide modifications |
| WO2024131237A1 (en) * | 2022-12-21 | 2024-06-27 | Hwu Wuh Liang | Recombinant viral vector, recombinant adeno-associated virus comprising the same, and uses thereof in treating sialidosis |
| IL327407A (en) * | 2023-09-29 | 2026-05-01 | The Board Of Regents Of The Univ Of Texas System | Endogenous nucleus targeting signal peptides and their uses |
| 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 |
| WO2025097038A1 (en) * | 2023-11-03 | 2025-05-08 | The Board Of Regents Of The University Of Texas System | Endogenous cell junction targeting signal peptides and uses thereof |
| WO2025106882A1 (en) * | 2023-11-15 | 2025-05-22 | The Board Of Regents Of The University Of Texas System | Endogenous cytoplasm targeting signal peptides and uses thereof |
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| DE3023787A1 (de) | 1980-06-25 | 1982-01-21 | Studiengesellschaft Kohle mbH, 4330 Mülheim | Verfahren zur erhoehung der inkorporation und der expression von genetischem material in die kerne von intakten zellen mit hilfe von liposomen |
| US4407956A (en) | 1981-03-13 | 1983-10-04 | The Regents Of The University Of California | Cloned cauliflower mosaic virus DNA as a plant vehicle |
| US4501729A (en) | 1982-12-13 | 1985-02-26 | Research Corporation | Aerosolized amiloride treatment of retained pulmonary secretions |
| DE3432718C1 (de) | 1984-09-06 | 1986-05-22 | Biotest Pharma GmbH, 6000 Frankfurt | Verfahren zur Herstellung einer Loesung von Milch- und/oder Kolostralimmunglobulinen |
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| JP2627899B2 (ja) | 1987-08-19 | 1997-07-09 | 株式会社 ビタミン研究所 | 遺伝子封入リポソームの製法 |
| FR2638643B1 (fr) * | 1988-11-09 | 1991-04-12 | Transgene Sa | Sequence d'adn codant pour le facteur ix humain ou une proteine analogue, vecteur d'expression, cellules transformees, procede de preparation du facteur ix et produits obtenus correspondants |
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-
2004
- 2004-01-21 JP JP2006501055A patent/JP5433133B2/ja not_active Expired - Lifetime
- 2004-01-21 EP EP04704039A patent/EP1587923B1/de not_active Expired - Lifetime
- 2004-01-21 PT PT04704039T patent/PT1587923E/pt unknown
- 2004-01-21 WO PCT/US2004/001453 patent/WO2004064750A2/en not_active Ceased
- 2004-01-21 US US10/761,530 patent/US20040248262A1/en not_active Abandoned
- 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
-
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 |
|---|---|
| JP2006517404A (ja) | 2006-07-27 |
| WO2004064750A3 (en) | 2004-11-11 |
| ES2371913T3 (es) | 2012-01-11 |
| US20180371440A1 (en) | 2018-12-27 |
| PT1587923E (pt) | 2011-12-07 |
| WO2004064750A2 (en) | 2004-08-05 |
| US9873868B2 (en) | 2018-01-23 |
| US20040248262A1 (en) | 2004-12-09 |
| EP1587923A2 (de) | 2005-10-26 |
| JP5433133B2 (ja) | 2014-03-05 |
| EP1587923B1 (de) | 2011-08-24 |
| US20120276072A1 (en) | 2012-11-01 |
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