CN116848146A - 可溶性碱性磷酸酶构建体和包含编码可溶性碱性磷酸酶构建体的多核苷酸的表达载体 - Google Patents

可溶性碱性磷酸酶构建体和包含编码可溶性碱性磷酸酶构建体的多核苷酸的表达载体 Download PDF

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Publication number
CN116848146A
CN116848146A CN202180066467.4A CN202180066467A CN116848146A CN 116848146 A CN116848146 A CN 116848146A CN 202180066467 A CN202180066467 A CN 202180066467A CN 116848146 A CN116848146 A CN 116848146A
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seq
polypeptide
amino acid
sequence
identity
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CN202180066467.4A
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English (en)
Chinese (zh)
Inventor
I·S·Y·陈
J·S·巴特利特
L·R·布雷顿
严明
甄安杰
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Rampat Biosciences
University of California, San Diego
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Rampat Biosciences
University of California, San Diego
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Publication of CN116848146A publication Critical patent/CN116848146A/zh
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    • 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)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • 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
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/03—Phosphoric monoester hydrolases (3.1.3)
    • C12Y301/03001—Alkaline phosphatase (3.1.3.1)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • C07K2319/30—Non-immunoglobulin-derived peptide or protein having an immunoglobulin constant or Fc region, or a fragment thereof, attached thereto
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
CN202180066467.4A 2020-09-03 2021-09-02 可溶性碱性磷酸酶构建体和包含编码可溶性碱性磷酸酶构建体的多核苷酸的表达载体 Pending CN116848146A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063074418P 2020-09-03 2020-09-03
US63/074,418 2020-09-03
PCT/US2021/048943 WO2022051555A2 (en) 2020-09-03 2021-09-02 Soluble alkaline phosphatase constructs and expression vectors including a polynucleotide encoding for soluble alkaline phosphatase constructs

Publications (1)

Publication Number Publication Date
CN116848146A true CN116848146A (zh) 2023-10-03

Family

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Family Applications (1)

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CN202180066467.4A Pending CN116848146A (zh) 2020-09-03 2021-09-02 可溶性碱性磷酸酶构建体和包含编码可溶性碱性磷酸酶构建体的多核苷酸的表达载体

Country Status (8)

Country Link
US (1) US20240150737A1 (enExample)
EP (1) EP4203989A2 (enExample)
JP (1) JP2023540098A (enExample)
KR (1) KR20230117327A (enExample)
CN (1) CN116848146A (enExample)
AU (1) AU2021338361A1 (enExample)
CA (1) CA3190513A1 (enExample)
WO (1) WO2022051555A2 (enExample)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2025525583A (ja) 2022-07-19 2025-08-05 ランパート バイオサイエンス インコーポレイテッド 非免疫原性環状非ウイルス性ｄｎａベクター
WO2025090786A1 (en) * 2023-10-24 2025-05-01 Rampart Bioscience, Inc. Reduced immunogenic gene therapy protocols using circular, extended cruciform containing non-viral vectors
WO2025097067A2 (en) * 2023-11-02 2025-05-08 Be Biopharma, Inc. Engineered cell preparations for treatment of hypophosphatasia
AU2024407494A1 (en) * 2023-12-19 2026-07-23 Evotec International Gmbh Plasmids, transgenes, vectors and medical uses comprising tissue non-specific alkaline phosphatase
WO2026089719A1 (en) * 2024-10-24 2026-04-30 Rampart Bioscience, Inc. Dna vectors having specialized secondary structures for use in treating hypophosphatasia

Family Cites Families (34)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4579821A (en) 1981-11-23 1986-04-01 University Patents, Inc. Control of DNA sequence transcription
US4656134A (en) 1982-01-11 1987-04-07 Board Of Trustees Of Leland Stanford Jr. University Gene amplification in eukaryotic cells
US4601978A (en) 1982-11-24 1986-07-22 The Regents Of The University Of California Mammalian metallothionein promoter system
US4713339A (en) 1983-01-19 1987-12-15 Genentech, Inc. Polycistronic expression vector construction
GR860984B (en) 1985-04-17 1986-08-18 Zymogenetics Inc Expression of factor vii and ix activities in mammalian cells
US4956288A (en) 1988-04-22 1990-09-11 Biogen, Inc. Method for producing cells containing stably integrated foreign DNA at a high copy number, the cells produced by this method, and the use of these cells to produce the polypeptides coded for by the foreign DNA
US5422120A (en) 1988-05-30 1995-06-06 Depotech Corporation Heterovesicular liposomes
ZA911974B (en) 1990-03-21 1994-08-22 Res Dev Foundation Heterovesicular liposomes
AU6818094A (en) 1993-04-22 1994-11-08 Depotech Corporation Cyclodextrin liposomes encapsulating pharmacologic compounds and methods for their use
HUT75162A (en) 1993-11-16 1997-04-28 Depotech Corp Vesicles with controlled release of actives
IL112372A (en) 1994-02-07 2001-08-26 Res Dev Foundation Non-viral vector for the delivery of genetic information to cells
CA2264482A1 (en) 1996-09-06 1998-03-12 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
US6218181B1 (en) 1998-03-18 2001-04-17 The Salk Institute For Biological Studies Retroviral packaging cell line
GB0024827D0 (en) * 2000-10-10 2000-11-22 Norwegian Inst Of Fisheries & Product
US7575924B2 (en) 2000-11-13 2009-08-18 Research Development Foundation Methods and compositions relating to improved lentiviral vectors and their applications
US7510877B2 (en) 2003-09-26 2009-03-31 The Regents Of The University Of Michigan Hematopoietic stem cell identification and isolation
PL1759001T3 (pl) * 2004-04-21 2011-09-30 Alexion Pharma Inc Koniugaty dostarczane do kości i sposób ich zastosowania do nakierowywania białek do kości
WO2005123142A1 (en) 2004-06-10 2005-12-29 Abraxis Bioscience, Inc. A method using inorganic nanoparticles as non-viral vectors for gene therapy
EP2573185A3 (en) 2005-02-16 2013-06-05 Lentigen Corporation Lentiviral vectors and their use
DE102005023993A1 (de) 2005-05-20 2006-11-23 TransMIT Gesellschaft für Technologietransfer mbH Nicht virales Vektorsystem zum Transport von Nukleinsäure in die Lunge
GB0702695D0 (en) 2007-02-12 2007-03-21 Ark Therapeutics Ltd Production of vectors
DK2662448T3 (en) * 2007-05-11 2017-03-20 Alexion Pharma Inc Bone-targeted alkaline phosphatase, kits and methods for its use
US20090098055A1 (en) * 2007-08-23 2009-04-16 Intrexon Corporation Methods and Compositions for Diagnosing Disease
ES2374245B1 (es) 2010-08-02 2012-12-26 Universidad Castilla La Mancha Vectores no virales para terapia génica.
ES2374243B1 (es) 2010-08-02 2012-12-26 Universidad Castilla-La Mancha Vectores no virales para terapia génica.
KR20140084201A (ko) * 2011-10-19 2014-07-04 알렉시온 파마 홀딩 알칼리성 포스파타제 및/또는 나트륨이뇨 펩티드를 포함하는 조성물 및 그의 사용 방법
CN103316356B (zh) 2012-03-22 2016-08-17 北京三诺佳邑生物技术有限责任公司 一种重组慢病毒载体制剂
US10052366B2 (en) * 2012-05-21 2018-08-21 Alexion Pharmaceuticsl, Inc. Compositions comprising alkaline phosphatase and/or natriuretic peptide and methods of use thereof
GB201220119D0 (en) 2012-11-08 2012-12-26 Univ Cork Vector
WO2016007873A1 (en) * 2014-07-11 2016-01-14 The Regents Of The University Of Michigan Compositions and methods for treating craniosynostosis
FR3024464A1 (fr) 2014-07-30 2016-02-05 Centre Nat Rech Scient Ciblage de vecteurs integratifs non-viraux dans les sequences d'adn nucleolaires chez les eucaryotes
JP6787894B2 (ja) * 2014-12-05 2020-11-18 アレクシオン ファーマシューティカルズ， インコーポレイテッド 組換えアルカリホスファターゼを用いた発作の処置
IL266977B2 (en) * 2016-11-30 2024-01-01 Purdue Research Foundation Fracture-targeted bone regeneration using thyroid hormone receptor stimulation
BR112021009913A2 (pt) * 2018-12-05 2021-08-17 Abeona Therapeutics, Inc. vetor viral adeno-associado recombinante para inserção gênica

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JP2023540098A (ja) 2023-09-21
CA3190513A1 (en) 2022-03-10
KR20230117327A (ko) 2023-08-08
WO2022051555A3 (en) 2022-04-14
US20240150737A1 (en) 2024-05-09
WO2022051555A2 (en) 2022-03-10
EP4203989A2 (en) 2023-07-05
AU2021338361A1 (en) 2023-04-06

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