JP2023540098A - 可溶性アルカリホスファターゼ構築物及び可溶性アルカリホスファターゼ構築物をコードするポリヌクレオチドを含む発現ベクター - Google Patents

可溶性アルカリホスファターゼ構築物及び可溶性アルカリホスファターゼ構築物をコードするポリヌクレオチドを含む発現ベクター Download PDF

Info

Publication number
JP2023540098A
JP2023540098A JP2023514458A JP2023514458A JP2023540098A JP 2023540098 A JP2023540098 A JP 2023540098A JP 2023514458 A JP2023514458 A JP 2023514458A JP 2023514458 A JP2023514458 A JP 2023514458A JP 2023540098 A JP2023540098 A JP 2023540098A
Authority
JP
Japan
Prior art keywords
seq
polypeptide
identity
amino acids
amino acid
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2023514458A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023540098A5 (enExample
Inventor
エス．ワイ． チェン，アーヴィン
エス． バートレット，ジェフリー
ランダル ブレトン，ルイス
ヤン，ミン
ズン，アンジー
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
University of California
Original Assignee
University of California, San Diego
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by University of California, San Diego filed Critical University of California, San Diego
Publication of JP2023540098A publication Critical patent/JP2023540098A/ja
Publication of JP2023540098A5 publication Critical patent/JP2023540098A5/ja
Pending legal-status Critical Current

Links

Images

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)
    • C12N9/16—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

Landscapes

  • 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)
JP2023514458A 2020-09-03 2021-09-02 可溶性アルカリホスファターゼ構築物及び可溶性アルカリホスファターゼ構築物をコードするポリヌクレオチドを含む発現ベクター Pending JP2023540098A (ja)

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 (2)

Publication Number Publication Date
JP2023540098A true JP2023540098A (ja) 2023-09-21
JP2023540098A5 JP2023540098A5 (enExample) 2024-09-09

Family

ID=78676616

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023514458A Pending JP2023540098A (ja) 2020-09-03 2021-09-02 可溶性アルカリホスファターゼ構築物及び可溶性アルカリホスファターゼ構築物をコードするポリヌクレオチドを含む発現ベクター

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

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2007533669A (ja) * 2004-04-21 2007-11-22 エノビア ファーマ インコーポレイティド 骨送達複合体ならびにタンパク質に骨を標的化させるためのその使用方法
JP2010526543A (ja) * 2007-05-11 2010-08-05 エノビア ファルマ インコーポレイテッド 骨標的アルカリホスファターゼ、キット及びその使用方法
JP2015502336A (ja) * 2011-10-19 2015-01-22 アレクシオン ファーマ ホールディング アルカリホスファターゼ及び／又はナトリウム利尿ペプチドを含む組成物、並びにそれを使用する方法
US20170175094A1 (en) * 2014-07-11 2017-06-22 Alexion Pharmaceuticals, Inc. Compositions and methods for treating craniosynostosis
JP2018502836A (ja) * 2014-12-05 2018-02-01 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
JP2020500871A (ja) * 2016-11-30 2020-01-16 パーデュー リサーチ ファウンデーション 副甲状腺ホルモン受容体刺激を介する、骨折をターゲティングした骨再生

Family Cites Families (28)

* 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
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
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.
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
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
BR112021009913A2 (pt) * 2018-12-05 2021-08-17 Abeona Therapeutics, Inc. vetor viral adeno-associado recombinante para inserção gênica

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2007533669A (ja) * 2004-04-21 2007-11-22 エノビア ファーマ インコーポレイティド 骨送達複合体ならびにタンパク質に骨を標的化させるためのその使用方法
JP2010526543A (ja) * 2007-05-11 2010-08-05 エノビア ファルマ インコーポレイテッド 骨標的アルカリホスファターゼ、キット及びその使用方法
JP2015502336A (ja) * 2011-10-19 2015-01-22 アレクシオン ファーマ ホールディング アルカリホスファターゼ及び／又はナトリウム利尿ペプチドを含む組成物、並びにそれを使用する方法
US20170175094A1 (en) * 2014-07-11 2017-06-22 Alexion Pharmaceuticals, Inc. Compositions and methods for treating craniosynostosis
JP2018502836A (ja) * 2014-12-05 2018-02-01 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
JP2020500871A (ja) * 2016-11-30 2020-01-16 パーデュー リサーチ ファウンデーション 副甲状腺ホルモン受容体刺激を介する、骨折をターゲティングした骨再生

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
ADV.DRUG DELIV.REV., vol. 65, no. 10, JPN6026024378, 2013, pages 1357 - 1369, ISSN: 0005885447 *
JEFFERY J. NIELSEN ; ET AL: "BONE-TARGETING SYSTEMS TO SYSTEMICALLY DELIVER THERAPEUTICS TO BONE FRACTURES FOR 以下備考", CURRENT OSTEOPOROSIS REPORTS, vol. VOL.18, NR.5, JPN5024003585, 29 August 2020 (2020-08-29), US, pages 449 - 459, ISSN: 0005885448 *
SEIKO YAMAMOTO ET AL.: ""Prolonged survival and phenotypic correction of Akp2-/- hypophosphatasia mice by lentiviral geneth", JOURNAL OF BONE AND MINERAL RESEARCH, vol. 26, no. 1, JPN6025042929, 22 December 2010 (2010-12-22), pages 135 - 142, ISSN: 0005711985 *

Also Published As

Publication number Publication date
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
CN116848146A (zh) 2023-10-03
AU2021338361A1 (en) 2023-04-06

Similar Documents

Publication Publication Date Title
US20240150737A1 (en) Soluble alkaline phosphatase constructs and expression vectors including a polynucleotide encoding for soluble alkaline phosphatase constructs
KR102856802B1 (ko) Vcn 인핸서 조성물 및 이의 사용 방법
AU2024227103A1 (en) Combinatorial cancer immunotherapy
KR102011532B1 (ko) 개선된 바이러스 형질도입을 위한 화합물
EP2229948B1 (en) Mesenchymal CD34- stem cells for use in diabetes gene therapy
US20240425878A1 (en) Genetically modified msc and therapeutic methods
JP7664829B2 (ja) 造血細胞の遺伝子改変のための方法
CN114555809A (zh) 组合癌症免疫疗法
US11326183B2 (en) VCN enhancer compositions and methods of using the same
US20220401518A1 (en) Mesenchymal stem cell expressing hepatocyte growth factor, and use thereof
JP2019536484A (ja) ムコ多糖症ｉ型のための遺伝子治療
WO2002083080A9 (en) Method of treating arthritis using lentiviral vectors in gene therapy
KR20230003477A (ko) 비-바이러스성 dna 벡터 및 인자 ix 치료제 발현을 위한 이의 용도
KR20210148106A (ko) 조정 가능한 조절을 위한 cd40l 조성물 및 방법
EP3768301A1 (en) Compositions and methods of fas inhibition
Ratanamart et al. Plasmid-mediated muscle-targeted gene therapy for circulating therapeutic protein replacement: a tale of the tortoise and the hare?
WO2020177618A1 (en) Recombinant vectors comprising arylsulfatase a and their uses in stem cell therapy for the treatment of metachromatic leukodystrophy
AU2005258052A1 (en) CCR ligands for stem cell homing
AU2007212260A1 (en) Method for treating peripheral arterial disease with zinc finger proteins
EP4700117A2 (en) Compositions and methods for the treatment of ischemia and cardiomyopathy
KR20190015153A (ko) 간세포성장인자를 발현하는 중간엽줄기세포를 유효성분으로 포함하는 혈관계 질환 예방 또는 치료용 약학적 조성물
WO2022115506A2 (en) Salicylic acid-inducible gene expression compositions and systems for cells
CN110072593A (zh) 适用于肾癌治疗的方法和药物组合物
WO2018009506A1 (en) Erythroid-specific promoter and method of use thereof
TW202306589A (zh) 用於改進治療ｘ連鎖肌微管性肌病之組成物及方法

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240830

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20240830

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20251021

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20260120

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20260421

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20260616