AR117980A1 - Terapia génica de hemofilia b mediante el uso de vectores virales que codifican variantes de fix recombinantes con expresión aumentada - Google Patents

Terapia génica de hemofilia b mediante el uso de vectores virales que codifican variantes de fix recombinantes con expresión aumentada

Info

Publication number
AR117980A1
AR117980A1 ARP200100276A ARP200100276A AR117980A1 AR 117980 A1 AR117980 A1 AR 117980A1 AR P200100276 A ARP200100276 A AR P200100276A AR P200100276 A ARP200100276 A AR P200100276A AR 117980 A1 AR117980 A1 AR 117980A1
Authority
AR
Argentina
Prior art keywords
hemophilia
factor
polynucleotide
gene therapy
fix
Prior art date
Application number
ARP200100276A
Other languages
English (en)
Inventor
Friedrich Scheifilinger
Franziska Horling
Johannes Lengler
Hanspeter Rottensteiner
Original Assignee
Takeda Pharmaceuticals Co
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 Takeda Pharmaceuticals Co filed Critical Takeda Pharmaceuticals Co
Publication of AR117980A1 publication Critical patent/AR117980A1/es

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Classifications

    • 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
    • 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
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • 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
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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

Landscapes

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

Abstract

La presente descripción proporciona, entre otros aspectos, polinucleótidos con alteración de codones que codifican variantes del Factor IX para la expresión en células de mamíferos. En algunas modalidades, la descripción también proporciona vectores de terapia génica de mamíferos y métodos para tratar la hemofilia B. En algunas modalidades, la presente descripción proporciona métodos para la dosificación a un paciente con hemofilia B con un polinucleótido, por ejemplo, un polinucleótido con alteración de codones que codifica un polipéptido del Factor IX. Reivindicación 1: Un método para tratar la hemofilia B que comprende administrar mediante infusión intravenosa, a un sujeto humano diagnosticado con hemofilia B, una dosis de 2,5 ´ 10¹¹ a 2 ´ 10¹² partículas de virus adenoasociados (AAV) por kilogramo de peso corporal del sujeto humano, en donde las partículas de AAV comprenden un polinucleótido del Factor IX que codifica la proteína del Factor IX, dicho polinucleótido del Factor IX comprende la secuencia de ácidos nucleicos de la SEQ ID Nº 17.
ARP200100276A 2019-02-01 2020-01-31 Terapia génica de hemofilia b mediante el uso de vectores virales que codifican variantes de fix recombinantes con expresión aumentada AR117980A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201962800338P 2019-02-01 2019-02-01

Publications (1)

Publication Number Publication Date
AR117980A1 true AR117980A1 (es) 2021-09-08

Family

ID=69743924

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP200100276A AR117980A1 (es) 2019-02-01 2020-01-31 Terapia génica de hemofilia b mediante el uso de vectores virales que codifican variantes de fix recombinantes con expresión aumentada

Country Status (4)

Country Link
US (2) US11596671B2 (es)
AR (1) AR117980A1 (es)
TW (1) TW202045534A (es)
WO (1) WO2020160303A1 (es)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3094859A1 (en) * 2020-10-01 2022-04-01 Entos Pharmaceuticals Inc. Proteolipid vesicles formulated with fusion associated small transmembrane proteins
CN114277057B (zh) * 2021-07-09 2023-10-13 上海天泽云泰生物医药有限公司 用于治疗或预防b型血友病的重组腺相关病毒载体和方法
MX2024005124A (es) * 2021-10-27 2024-05-16 Regeneron Pharma Composiciones y metodos para expresar el factor ix para terapia para la hemofilia b.

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
US6531298B2 (en) 1997-07-21 2003-03-11 The University Of North Carolina At Chapel Hill Factor IX antihemophilic factor with increased clotting activity
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
EP2438931B1 (en) 2004-09-22 2013-11-13 St. Jude Children's Research Hospital Improved expression of factor IX in gene therapy vectors
US9617548B2 (en) 2008-04-22 2017-04-11 Vib Vzw Liver-specific nucleic acid regulatory elements and methods and use thereof
TWI595004B (zh) 2010-11-03 2017-08-11 介控生化科技公司 經修飾之第九因子多胜肽及其用途
JP6454643B2 (ja) 2012-10-26 2019-01-16 フリーイェ・ユニヴェルシテイト・ブリュッセルＶｒｉｊｅ Ｕｎｉｖｅｒｓｉｅｉｔ Ｂｒｕｓｓｅｌ 血友病の肝臓指向性遺伝子治療のためのベクター並びにその方法及び使用
DK3270944T3 (da) 2015-03-17 2020-01-27 Univ Brussel Vrije Optimerede leverspecifikke ekspressionssystemer til FVIII og FIX
BR112017027956A2 (pt) 2015-06-23 2018-08-28 Childrens Hospital Philadelphia fator ix modificado e composições, métodos e usos para a transferência de gene para células, órgãos e tecidos
CN110945127B (zh) * 2017-05-22 2024-07-12 武田药品工业株式会社 用于血友病b的基因疗法的编码具有增加的表达的重组fix的病毒载体

Also Published As

Publication number Publication date
WO2020160303A1 (en) 2020-08-06
US20240100128A1 (en) 2024-03-28
US20200246437A1 (en) 2020-08-06
US11596671B2 (en) 2023-03-07
TW202045534A (zh) 2020-12-16

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