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 |
|---|---|---|---|---|
| WO2002087510A2 (en) * | 2001-04-30 | 2002-11-07 | Symbiontics, Inc. | Subcellular targeting of therapeutic proteins |
| US7560424B2 (en) | 2001-04-30 | 2009-07-14 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
| US7629309B2 (en) | 2002-05-29 | 2009-12-08 | 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 |
| 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 |
| EP2484768A3 (de) * | 2005-07-18 | 2012-11-21 | Protalix 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 |
| EP2099523A2 (de) * | 2006-11-13 | 2009-09-16 | ZyStor Therapeutics , Inc. | Verfahren zur behandlung von pompe-krankheit |
| 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 |
| JP5627571B2 (ja) | 2008-05-07 | 2014-11-19 | バイオマリン ファーマシューティカル インコーポレイテッド | リソソーム標的化ペプチドおよびその使用 |
| US20110318322A1 (en) | 2009-01-12 | 2011-12-29 | Nektar Therapeutics | Conjugates of a Lysosomal Enzyme Moiety and a Water Soluble Polymer |
| EP2215980B1 (de) * | 2009-02-04 | 2012-12-19 | Stryker Leibinger GmbH & Co. KG | Chirurgisches Elektrowerkzeug und Betätigungsbaugruppe hierfür |
| EP2215981B1 (de) * | 2009-02-04 | 2013-04-03 | Stryker Leibinger GmbH & Co. KG | Chirurgisches Elektrowerkzeug, Betriebsverfahren und Betätigungsbaugruppe hierfür |
| EP3679942A1 (de) | 2009-06-17 | 2020-07-15 | BioMarin Pharmaceutical Inc. | Formulierungen für lysosomale enzyme |
| HRP20191923T1 (hr) | 2010-06-25 | 2020-01-10 | Shire Human Genetic Therapies, Inc. | Postupci i sastavi za isporuku arilsulfataze a u cns |
| 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 |
| KR20230159646A (ko) | 2010-06-25 | 2023-11-21 | 샤이어 휴먼 지네틱 테라피즈 인크. | 이듀로네이트-2-설파타제의 cns 전달을 위한 방법들 및 조성물들 |
| LT2588130T (lt) | 2010-06-25 | 2016-12-12 | Shire Human Genetic Therapies, Inc. | Terapinės priemonės pristatymas cns |
| PT3103469T (pt) | 2010-06-25 | 2021-03-04 | Shire Human Genetic Therapies | Administração de agentes terapêuticos no snc |
| AU2011270672B2 (en) | 2010-06-25 | 2017-01-12 | Shire Human Genetic Therapies, Inc. | Treatment of Sanfilippo Syndrome type B |
| AU2010366066B2 (en) * | 2010-12-22 | 2016-01-14 | Fondazione Telethon | Therapeutic strategies to treat CNS pathology in mucopolysaccharidoses |
| MX2013012345A (es) * | 2011-04-22 | 2015-05-07 | Genzyme Corp | Alfa glucosidasa acida modificada con procesamiento acelerado. |
| EA201892619A1 (ru) | 2011-04-29 | 2019-04-30 | Роше Гликарт Аг | Иммуноконъюгаты, содержащие мутантные полипептиды интерлейкина-2 |
| LT2753346T (lt) | 2011-09-07 | 2020-08-10 | Mount Sinai School Of Medicine | Ceramidazė ir ląstelių diferenciacija |
| SG10201510106PA (en) * | 2011-10-12 | 2016-01-28 | 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 |
| ES2897825T3 (es) | 2012-06-01 | 2022-03-02 | Icahn School Med Mount Sinai | Niveles de ceramida en el tratamiento y prevención de infecciones |
| KR20150020250A (ko) | 2012-06-19 | 2015-02-25 | 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 | 당뇨병을 치료하기 위한 조성물 및 방법 |
| AU2014218854B2 (en) | 2013-02-20 | 2019-01-24 | Valerion Therapeutics, Llc | Methods and compositions for treatment of Pompe disease |
| PT2968479T (pt) | 2013-03-14 | 2019-08-07 | Icahn School Med Mount Sinai | Composições terapêuticas de ceramidase ácida e métodos de fabrico e utilização |
| 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 |
| WO2018046774A1 (en) * | 2016-09-12 | 2018-03-15 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| EP3293259A1 (de) * | 2016-09-12 | 2018-03-14 | Genethon | Saure alpha-glucosidase-varianten und verwendungen davon |
| 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 |
| DK3635009T3 (da) | 2017-06-07 | 2026-03-30 | Regeneron Pharma | Sammensætninger og fremgangsmåder til internalisering af enzymer |
| US12258597B2 (en) | 2018-02-07 | 2025-03-25 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for therapeutic protein delivery |
| BR112020021962A2 (pt) | 2018-04-30 | 2021-01-26 | Amicus Therapeutics, Inc. | construtos para terapia gênica e métodos de uso |
| 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 |
| CN113631182B (zh) | 2018-10-10 | 2025-03-21 | 阿米库斯治疗学公司 | 二硫键稳定的多肽组合物和使用方法 |
| US20220002752A1 (en) * | 2018-11-13 | 2022-01-06 | Denovo Biopharma Llc | Recombinant vectors comprising genes for binding domains and secretable peptides |
| JP2022513067A (ja) * | 2018-11-16 | 2022-02-07 | アスクレピオス バイオファーマシューティカル, インコーポレイテッド | ポンペ病を処置するための治療用アデノ随伴ウイルス |
| AU2019381803A1 (en) * | 2018-11-16 | 2021-06-10 | AskBio Inc. | Vectors comprising a nucleic acid encoding lysosomal enzymes fused to a lysosomal targeting sequence |
| JP7585233B2 (ja) * | 2019-04-30 | 2024-11-18 | 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 | ポンペ病の治療のために有用な組成物 |
| EP4021469A4 (de) * | 2019-08-28 | 2023-10-04 | University Of Florida Research Foundation, Incorporated | Verbesserte herstellung von rekombinantem aav unter verwendung von embryonierten vogeleiern |
| WO2021078834A1 (en) | 2019-10-22 | 2021-04-29 | Genethon | Chimeric acid-alpha glucosidase polypeptides and uses thereof |
| JP7719060B2 (ja) | 2019-10-22 | 2025-08-05 | ジェネトン | キメラポリペプチド及びその使用 |
| KR20220098384A (ko) * | 2019-11-19 | 2022-07-12 | 아스클레피오스 바이오파마슈티컬, 인크. | 폼페병 및 리소좀 장애를 치료하기 위한 간-특이적 프로모터를 포함하는 치료적 아데노-관련 바이러스 |
| JP2023507575A (ja) | 2019-12-20 | 2023-02-24 | コデクシス, インコーポレイテッド | 操作された酸性アルファ-グルコシダーゼバリアント |
| US20230321237A1 (en) * | 2020-08-13 | 2023-10-12 | Epivax, Inc. | Regulatory t cell epitopes |
| 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 |
| CA3209738A1 (en) * | 2021-01-26 | 2022-08-04 | Sigilon Therapeutics, Inc. | Compositions, devices and methods for treating immune-mediated inflammatory diseases |
| EP4288535A4 (de) * | 2021-02-05 | 2024-12-18 | Sigilon Therapeutics, Inc. | Zusammensetzungen, vorrichtungen und verfahren zur behandlung der mps-vi-krankheit |
| EP4423264A4 (de) * | 2021-10-27 | 2025-12-17 | Univ North Carolina Chapel Hill | Aav-idua-vektor zur behandlung von 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 |
| 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 |
| AU2024349204A1 (en) * | 2023-09-29 | 2026-04-09 | The Board Of Regents Of The University Of Texas System | Endogenous nucleus targeting signal peptides and uses thereof |
| WO2025080721A2 (en) * | 2023-10-09 | 2025-04-17 | 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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| 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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-
2004
- 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 EP EP04704039A patent/EP1587923B1/de not_active Expired - Lifetime
- 2004-01-21 AT AT04704039T patent/ATE521701T1/de not_active IP Right Cessation
- 2004-01-21 US US10/761,530 patent/US20040248262A1/en not_active Abandoned
- 2004-01-21 WO PCT/US2004/001453 patent/WO2004064750A2/en not_active Ceased
- 2004-01-21 PT PT04704039T patent/PT1587923E/pt unknown
-
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 |
|---|---|
| US20040248262A1 (en) | 2004-12-09 |
| JP2006517404A (ja) | 2006-07-27 |
| PT1587923E (pt) | 2011-12-07 |
| JP5433133B2 (ja) | 2014-03-05 |
| EP1587923B1 (de) | 2011-08-24 |
| WO2004064750A3 (en) | 2004-11-11 |
| US20180371440A1 (en) | 2018-12-27 |
| US20120276072A1 (en) | 2012-11-01 |
| WO2004064750A2 (en) | 2004-08-05 |
| EP1587923A2 (de) | 2005-10-26 |
| US9873868B2 (en) | 2018-01-23 |
| ES2371913T3 (es) | 2012-01-11 |
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| Date | Code | Title | Description |
|---|---|---|---|
| RER | Ceased as to paragraph 5 lit. 3 law introducing patent treaties |