[go: up one dir, main page]
More Web Proxy on the site http://driver.im/

MX2018005272A - Construcciones específicas del hígado, casetes de expresión del factor viii y métodos de uso de estos. - Google Patents

Construcciones específicas del hígado, casetes de expresión del factor viii y métodos de uso de estos.

Info

Publication number
MX2018005272A
MX2018005272A MX2018005272A MX2018005272A MX2018005272A MX 2018005272 A MX2018005272 A MX 2018005272A MX 2018005272 A MX2018005272 A MX 2018005272A MX 2018005272 A MX2018005272 A MX 2018005272A MX 2018005272 A MX2018005272 A MX 2018005272A
Authority
MX
Mexico
Prior art keywords
liver
transgene
methods
factor viii
expression cassettes
Prior art date
Application number
MX2018005272A
Other languages
English (en)
Inventor
E Riley Brigit
K Lee Gary
Original Assignee
Sangamo Therapeutics Inc
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 Sangamo Therapeutics Inc filed Critical Sangamo Therapeutics Inc
Publication of MX2018005272A publication Critical patent/MX2018005272A/es

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • 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
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/36—Blood coagulation or fibrinolysis factors
    • A61K38/37—Factors VIII
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/02—Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • 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
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145—Special targeting system for viral vectors
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/60—Vector systems having a special element relevant for transcription from viruses

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Hematology (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Immunology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Toxicology (AREA)
  • Diabetes (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

La presente invención pertenece al ámbito de la terapia génica, particularmente la administración dirigida de construcciones que codifican transgenes al hígado para la expresión de proteínas beneficiosas (terapéuticas). En particular, la invención se refiere al tratamiento de hemofilias, tal como la hemofilia A. La presente invención describe composiciones y métodos para expresar un transgén en una célula hepática. El transgén se puede expresar de forma extracromosómica (episomica) o puede integrarse en el genoma de la célula hepática. En algunas modalidades, el transgén codifica una proteína implicada en la cascada de coagulación. En modalidades preferidas, el transgén codifica un polipéptido FVIII.
MX2018005272A 2015-10-28 2016-07-13 Construcciones específicas del hígado, casetes de expresión del factor viii y métodos de uso de estos. MX2018005272A (es)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201562247469P 2015-10-28 2015-10-28
US201662307897P 2016-03-14 2016-03-14
US201662315438P 2016-03-30 2016-03-30
US201662326229P 2016-04-22 2016-04-22
US201662355106P 2016-06-27 2016-06-27
PCT/US2016/042099 WO2017074526A1 (en) 2015-10-28 2016-07-13 Liver-specific constructs, factor viii expression cassettes and methods of use thereof

Publications (1)

Publication Number Publication Date
MX2018005272A true MX2018005272A (es) 2018-09-18

Family

ID=58631984

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2018005272A MX2018005272A (es) 2015-10-28 2016-07-13 Construcciones específicas del hígado, casetes de expresión del factor viii y métodos de uso de estos.

Country Status (18)

Country Link
US (4) US10143760B2 (es)
EP (1) EP3368664A4 (es)
JP (2) JP6909212B2 (es)
KR (1) KR20180069054A (es)
CN (2) CN115141836B (es)
AU (1) AU2016343887B2 (es)
BR (1) BR112018008519A2 (es)
CA (1) CA3002524A1 (es)
CO (1) CO2018005215A2 (es)
IL (1) IL258826B2 (es)
MX (1) MX2018005272A (es)
MY (1) MY189674A (es)
PE (1) PE20181206A1 (es)
PH (1) PH12018500927A1 (es)
SA (1) SA518391456B1 (es)
SG (1) SG11201803547RA (es)
WO (1) WO2017074526A1 (es)
ZA (1) ZA201802723B (es)

Families Citing this family (67)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3613852A3 (en) 2011-07-22 2020-04-22 President and Fellows of Harvard College Evaluation and improvement of nuclease cleavage specificity
US9163284B2 (en) 2013-08-09 2015-10-20 President And Fellows Of Harvard College Methods for identifying a target site of a Cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US20150166982A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting pi3k point mutations
CA2956224A1 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
CA2991301A1 (en) 2015-07-13 2017-01-19 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
WO2017070632A2 (en) 2015-10-23 2017-04-27 President And Fellows Of Harvard College Nucleobase editors and uses thereof
EP3368664A4 (en) * 2015-10-28 2019-08-28 Sangamo Therapeutics, Inc. LIFE-SPECIFIC CONSTRUCTS, FACTOR VIII EXPRESSION CASSETTES AND METHOD OF USE THEREOF
PE20231949A1 (es) 2015-10-30 2023-12-05 Spark Therapeutics Inc VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA
SG11201811603WA (en) 2016-07-26 2019-02-27 Biomarin Pharm Inc Novel adeno-associated virus capsid proteins
IL264565B2 (en) 2016-08-03 2024-07-01 Harvard College Adenosine nuclear base editors and their uses
AU2017308889B2 (en) 2016-08-09 2023-11-09 President And Fellows Of Harvard College Programmable Cas9-recombinase fusion proteins and uses thereof
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
EP3509645A4 (en) 2016-09-07 2020-06-17 Sangamo Therapeutics, Inc. MODULATION OF LIVER GENES
EP3526320A1 (en) 2016-10-14 2019-08-21 President and Fellows of Harvard College Aav delivery of nucleobase editors
US11219695B2 (en) 2016-10-20 2022-01-11 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of Fabry disease
JP7292204B2 (ja) 2016-12-01 2023-06-16 サンガモ セラピューティクス， インコーポレイテッド タウモジュレーターならびにその送達のための方法および組成物
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
US11542496B2 (en) 2017-03-10 2023-01-03 President And Fellows Of Harvard College Cytosine to guanine base editor
IL269458B2 (en) 2017-03-23 2024-02-01 Harvard College Nucleic base editors that include nucleic acid programmable DNA binding proteins
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
JP2020534795A (ja) 2017-07-28 2020-12-03 プレジデント アンド フェローズ オブ ハーバード カレッジ ファージによって支援される連続的進化（ｐａｃｅ）を用いて塩基編集因子を進化させるための方法および組成物
MX2020001402A (es) * 2017-08-01 2021-01-29 Spark Therapeutics Inc Metodos de terapia genica del factor viii (fviii).
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
EP3697906A1 (en) 2017-10-16 2020-08-26 The Broad Institute, Inc. Uses of adenosine base editors
SG11202003464VA (en) * 2017-10-17 2020-05-28 Crispr Therapeutics Ag Compositions and methods for gene editing for hemophilia a
US20210254057A1 (en) * 2018-01-12 2021-08-19 Crispr Therapeutics Ag Compositions and methods for gene editing by targeting transferrin
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
JP7275152B2 (ja) 2018-02-08 2023-05-17 サンガモ セラピューティクス， インコーポレイテッド 操作された標的特異的なヌクレアーゼ
US20210130824A1 (en) * 2018-02-16 2021-05-06 Crispr Therapeutics Ag Compositions and methods for gene editing by targeting fibrinogen-alpha
CN112041437A (zh) 2018-02-19 2020-12-04 同源药物公司 用于恢复f8基因功能的腺相关病毒组合物和其使用方法
AU2019265560A1 (en) 2018-05-09 2020-11-26 Biomarin Pharmaceutical Inc. Methods of treating phenylketonuria
TW202005978A (zh) 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 新穎肝靶向腺相關病毒載體
MX2020012167A (es) * 2018-05-14 2022-07-13 Biomarin Pharm Inc Expresion estable de vectores de aav en sujetos juveniles.
US11690921B2 (en) 2018-05-18 2023-07-04 Sangamo Therapeutics, Inc. Delivery of target specific nucleases
CA3104113A1 (en) * 2018-06-22 2019-12-26 Asklepios Biopharmaceutical, Inc. Vectors for gene delivery that persist within cells
EP3829650A4 (en) * 2018-08-03 2022-05-04 Sangamo Therapeutics, Inc. CLINICAL PARAMETERS IMPROVED BY FACTOR VIII EXPRESSION
BR112021003399A2 (pt) * 2018-08-24 2021-05-18 Spark Therapeutics, Inc. sequências de promotores otimizadas, construtos de expressão livres de íntron e métodos de uso
DE112019005166T5 (de) * 2018-10-16 2021-07-29 Blueallele, Llc Verfahren zur gezielten insertion von dna in gene
US20210403948A1 (en) * 2018-10-26 2021-12-30 Vrije Universiteit Brussel Liver-Specific Nucleic Acid Regulatory Elements and Methods and Use Thereof
JP7523449B2 (ja) 2019-01-11 2024-07-26 アクイタス セラピューティクス インコーポレイテッド 活性剤の脂質ナノ粒子送達のための脂質
US11857641B2 (en) 2019-02-06 2024-01-02 Sangamo Therapeutics, Inc. Method for the treatment of mucopolysaccharidosis type I
CA3129532A1 (en) 2019-02-15 2020-08-20 Crispr Therapeutics Ag Gene editing for hemophilia a with improved factor viii expression
MX2021011039A (es) * 2019-03-13 2021-12-15 Generation Bio Co Vectores de adn no virales y usos de estos para expresar agentes terapéuticos con fviii.
JP2022527740A (ja) 2019-03-19 2022-06-06 ザ ブロード インスティテュート，インコーポレーテッド 編集ヌクレオチド配列を編集するための方法および組成物
CN110229849B (zh) * 2019-05-31 2021-09-17 中国科学院深圳先进技术研究院 时空调控型uPA基因表达非病毒载体及其制备方法和应用
CA3137764A1 (en) 2019-06-07 2020-12-10 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized albumin locus
CN110684798A (zh) * 2019-09-03 2020-01-14 深圳新诺微环生物科技有限公司 肌肉靶向的微环dna基因治疗
CN113058041B (zh) * 2020-08-27 2022-04-05 华东师范大学 一种用于治疗庞贝氏病的产品
CN113088519B (zh) * 2019-12-23 2024-02-02 苏州华毅乐健生物科技有限公司 一种分离的核酸分子及其应用
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
IL297761A (en) 2020-05-08 2022-12-01 Broad Inst Inc Methods and compositions for simultaneously editing two helices of a designated double-helix nucleotide sequence
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法
CN116096702A (zh) 2020-07-16 2023-05-09 爱康泰生治疗公司 用于脂质纳米颗粒的阳离子脂质
WO2022097008A1 (en) 2020-11-03 2022-05-12 Pfizer Inc. Methods for purification of aav vectors by anion exchange chromatography
KR102536321B1 (ko) 2020-12-09 2023-05-26 재단법인 아산사회복지재단 인체 유래 유전자 발현 유도 모듈에 기반한 간 특이 유전자 발현 시스템
JP2024500133A (ja) 2020-12-18 2024-01-04 サンガモ セラピューティクス， インコーポレイテッド アデノ随伴ウイルスベクターを含む改善された医薬組成物
CA3205924A1 (en) 2020-12-23 2022-06-30 Pfizer Inc. Methods for purification of aav vectors by affinity chromatography
WO2022269466A1 (en) 2021-06-22 2022-12-29 Pfizer Inc. Production of adeno-associated virus vector in insect cells
CN118647600A (zh) 2021-12-16 2024-09-13 爱康泰生治疗公司 用于脂质纳米颗粒制剂的脂质
WO2024003687A1 (en) 2022-06-28 2024-01-04 Pfizer Inc. Nucleic acids encoding acid alpha-glucosidase (gaa) and vectors for gene therapy
CN115948408A (zh) * 2022-09-23 2023-04-11 上海信致医药科技有限公司 改进的人凝血因子viii基因表达盒及其应用

Family Cites Families (55)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1998024479A1 (en) 1996-12-02 1998-06-11 Cell Genesys, Inc. Adeno-associated viral vector-mediated delivery of dna to cells of the liver
US6221349B1 (en) 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
CA2394850C (en) 1999-12-06 2012-02-07 Sangamo Biosciences, Inc. Methods of using randomized libraries of zinc finger proteins for the identification of gene function
AU5077401A (en) 2000-02-08 2001-08-20 Sangamo Biosciences Inc Cells for drug discovery
US7067317B2 (en) 2000-12-07 2006-06-27 Sangamo Biosciences, Inc. Regulation of angiogenesis with zinc finger proteins
US7262054B2 (en) 2002-01-22 2007-08-28 Sangamo Biosciences, Inc. Zinc finger proteins for DNA binding and gene regulation in plants
US8106255B2 (en) 2002-01-23 2012-01-31 Dana Carroll Targeted chromosomal mutagenasis using zinc finger nucleases
US20030232410A1 (en) 2002-03-21 2003-12-18 Monika Liljedahl Methods and compositions for using zinc finger endonucleases to enhance homologous recombination
US7238346B2 (en) 2002-07-08 2007-07-03 Vlaams Interuniversitair Instituut Voor Biotechnologie Vzw High capacity recombinant adenoviral vector for treatment of hemophilia A
US9447434B2 (en) 2002-09-05 2016-09-20 California Institute Of Technology Use of chimeric nucleases to stimulate gene targeting
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
SI1675956T1 (sl) 2003-10-21 2010-12-31 Merck Serono Sa Minimalno zaporedje dna, ki deluje kot izolirni element kromatina in njegova uporaba pri ekspresiji proteinov
US7972854B2 (en) 2004-02-05 2011-07-05 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US9023617B2 (en) * 2004-02-17 2015-05-05 University Of Florida Research Foundation, Inc. Insulated herpesvirus-derived gene expression cassettes for sustained and regulatable gene expression
CA2579677A1 (en) 2004-09-16 2006-03-30 Sangamo Biosciences, Inc. Compositions and methods for protein production
JP2008543283A (ja) * 2005-06-10 2008-12-04 ジェネンテック・インコーポレーテッド インスレーターエレメントを含むベクターを使用する抗体発現の改良
EP2765195A1 (en) 2006-05-25 2014-08-13 Sangamo BioSciences, Inc. Methods and compositions for gene inactivation
CA2651494C (en) 2006-05-25 2015-09-29 Sangamo Biosciences, Inc. Engineered cleavage half-domains
EP1985631A1 (en) * 2007-04-20 2008-10-29 LFB Biotechnologies Demannosylated recombinant factor VIII for the treatment of patients with hemophiila A
DE602008003684D1 (de) 2007-04-26 2011-01-05 Sangamo Biosciences Inc Gezielte integration in die ppp1r12c-position
CA2720903C (en) 2008-04-14 2019-01-15 Sangamo Biosciences, Inc. Linear donor constructs for targeted integration
DK2282764T3 (da) 2008-04-22 2019-10-14 Vib Vzw Leverspecifikke nukleinsyreregulerende elementer samt fremgangsmåder og anvendelser heraf
CN102159722B (zh) 2008-08-22 2014-09-03 桑格摩生物科学股份有限公司 用于靶向单链切割和靶向整合的方法和组合物
AU2009322964B2 (en) 2008-12-04 2014-10-09 Sangamo Therapeutics, Inc. Genome editing in rats using zinc-finger nucleases
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
US8956828B2 (en) 2009-11-10 2015-02-17 Sangamo Biosciences, Inc. Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
AU2011213242B2 (en) 2010-02-08 2015-01-29 Sangamo Therapeutics, Inc. Engineered cleavage half-domains
WO2011100058A1 (en) 2010-02-09 2011-08-18 Sangamo Biosciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
JP5952263B2 (ja) 2010-04-26 2016-07-13 サンガモ バイオサイエンシーズ， インコーポレイテッド ジンクフィンガーヌクレアーゼを使ったｒｏｓａ遺伝子座のゲノム編集
EP2571512B1 (en) 2010-05-17 2017-08-23 Sangamo BioSciences, Inc. Novel dna-binding proteins and uses thereof
WO2012012667A2 (en) 2010-07-21 2012-01-26 Sangamo Biosciences, Inc. Methods and compositions for modification of a hla locus
AU2011316575B2 (en) * 2010-10-12 2015-10-29 Sangamo Therapeutics, Inc. Methods and compositions for treating hemophilia B
EP3366312A1 (en) * 2011-06-23 2018-08-29 Alnylam Pharmaceuticals, Inc. Serpina 1 sirnas: compositions of matter and methods of treatment
SG10201811736QA (en) 2011-09-21 2019-02-27 Sangamo Therapeutics Inc Methods and compositions for regulation of transgene expression
CA2852955C (en) 2011-10-27 2021-02-16 Sangamo Biosciences, Inc. Methods and compositions for modification of the hprt locus
WO2013074999A1 (en) 2011-11-16 2013-05-23 Sangamo Biosciences, Inc. Modified dna-binding proteins and uses thereof
EA035323B1 (ru) * 2012-01-12 2020-05-28 Биовератив Терапьютикс Инк. Полипептиды химерного фактора viii и их применение
EP3816281A1 (en) 2012-07-11 2021-05-05 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of lysosomal storage diseases
US9650648B2 (en) 2012-08-29 2017-05-16 Sangamo Biosciences, Inc. Methods and compositions for treatment of a genetic condition
US20150224209A1 (en) * 2012-09-14 2015-08-13 The Regents Of The University Of California Lentiviral vector for stem cell gene therapy of sickle cell disease
DK2911687T3 (da) * 2012-10-26 2019-05-13 Univ Brussel Vrije Vektor til levermålrettet genterapi af hæmofili og fremgangsmåder og anvendelse deraf
US10398787B2 (en) * 2012-10-26 2019-09-03 Vrije Universiteit Brussel Vectors for liver-directed gene therapy of hemophilia and methods and use thereof
CA2892448A1 (en) 2012-12-05 2014-06-12 Sangamo Biosciences, Inc. Methods and compositions for regulation of metabolic disorders
AU2014246731A1 (en) * 2013-03-30 2015-11-19 Usha Biotech Limited Methods and constructs for expressing biologically active proteins in mammalian cells
EP3730615A3 (en) 2013-05-15 2020-12-09 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition
WO2015038625A1 (en) * 2013-09-12 2015-03-19 Biomarin Pharmaceutical Inc. Adeno-associated virus factor viii vectors
US10081661B2 (en) 2013-12-09 2018-09-25 Sangamo Therapeutics, Inc. Methods and compositions for genome engineering
CA2991301A1 (en) 2015-07-13 2017-01-19 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
NZ780570A (en) 2015-09-24 2024-08-30 Biomarin Pharm Inc Adeno-associated virus factor viii vectors, associated viral particles and therapeutic formulations comprising the same
EP3368664A4 (en) * 2015-10-28 2019-08-28 Sangamo Therapeutics, Inc. LIFE-SPECIFIC CONSTRUCTS, FACTOR VIII EXPRESSION CASSETTES AND METHOD OF USE THEREOF
MY190067A (en) 2015-11-13 2022-03-24 Baxalta Inc Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a

Also Published As

Publication number Publication date
EP3368664A4 (en) 2019-08-28
CN108473976A (zh) 2018-08-31
ZA201802723B (en) 2019-07-31
KR20180069054A (ko) 2018-06-22
SG11201803547RA (en) 2018-05-30
SA518391456B1 (ar) 2022-12-18
CN115141836B (zh) 2024-08-27
CO2018005215A2 (es) 2018-11-30
US20190151472A1 (en) 2019-05-23
JP7324249B2 (ja) 2023-08-09
US20170119906A1 (en) 2017-05-04
JP2021176305A (ja) 2021-11-11
RU2018117179A3 (es) 2019-12-27
CA3002524A1 (en) 2017-05-04
IL258826B2 (en) 2023-07-01
EP3368664A1 (en) 2018-09-05
PH12018500927A1 (en) 2018-11-12
JP2018531614A (ja) 2018-11-01
AU2016343887B2 (en) 2023-04-06
CN108473976B (zh) 2022-07-19
IL258826B1 (en) 2023-03-01
IL258826A (en) 2018-06-28
BR112018008519A2 (pt) 2018-11-06
WO2017074526A1 (en) 2017-05-04
PE20181206A1 (es) 2018-07-23
US10828376B2 (en) 2020-11-10
US20230119850A1 (en) 2023-04-20
US20190142970A1 (en) 2019-05-16
JP6909212B2 (ja) 2021-07-28
RU2018117179A (ru) 2019-11-28
US10143760B2 (en) 2018-12-04
AU2016343887A1 (en) 2018-05-10
MY189674A (en) 2022-02-24
US11452782B2 (en) 2022-09-27
CN115141836A (zh) 2022-10-04

Similar Documents

Publication Publication Date Title
PH12018500927A1 (en) Liver-specific constructs, factor viii expression cassettes and methods of use thereof
PH12018501055A1 (en) Cpg reduced factor viii variants, compositions and methods and uses for treatment of hemostasis disorders
PH12019500694A1 (en) Factor viii compositions and methods of making and using the same
PH12018502361A1 (en) Gdf15 fusion proteins and uses thereof
MX2020001593A (es) Moleculas de acido nucleico y usos de las mismas.
MX2021015182A (es) Casetes optimizados de expresion del gen humano del factor viii de coagulacion y su uso.
SA518400424B1 (ar) جزيئات الجسم المضاد لعلاج السرطان
PH12020550258A1 (en) Trispecific proteins and methods of use
SA517390590B1 (ar) Ix عامل معدل، وتركيبات وطرق واستخدامات لنقل الجين إلى الخلايا، والأعضاء والأنسجة
MX362028B (es) Polipeptidos recombinantes extendidos y composiciones que comprenden los mismos.
MX354555B (es) Polipeptidos de la hormona de crecimiento y metodos de preparacion y su uso.
MX2022015079A (es) Variantes de hsd17b13 y usos de las mismas.
EP4406585A3 (en) Nucleic acid products and methods of administration thereof
EP3626274A3 (en) Recombinant promoters and vectors for protein expression in liver and use thereof
SA522432139B1 (ar) بروتينات ربط متعددة الخصوصية لمعالجة السرطان
MX362513B (es) Vectores que expresan de manera condicional proteinas terapeuticas, celulas hospedadoras que comprenden vectores, y usos de los mismos.
WO2016019144A3 (en) Gene correction of scid-related genes in hematopoietic stem and progenitor cells
MX357803B (es) Moléculas de ácido nucleico artificiales.
WO2011123830A3 (en) Alpha 1-antitrypsin compositions and methods of making and using same
TN2012000372A1 (en) Fibronectin based scaffold domain proteins that bind il-23
UA113487C2 (xx) Злитий білок, що містить гормон росту та рекомбінантний поліпептид xten, і способи його виробництва та застосування