MX2021005435A - Transgen abcb11 con codones optimizados para el tratamiento de colestasis intrahepatica familiar progresiva tipo 2 (pfic2). - Google Patents

Transgen abcb11 con codones optimizados para el tratamiento de colestasis intrahepatica familiar progresiva tipo 2 (pfic2).

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Publication number
MX2021005435A
MX2021005435A MX2021005435A MX2021005435A MX2021005435A MX 2021005435 A MX2021005435 A MX 2021005435A MX 2021005435 A MX2021005435 A MX 2021005435A MX 2021005435 A MX2021005435 A MX 2021005435A MX 2021005435 A MX2021005435 A MX 2021005435A
Authority
MX
Mexico
Prior art keywords
treatment
pfic2
codon
optimized
abcb11
Prior art date
Application number
MX2021005435A
Other languages
English (en)
Inventor
Aseguinolaza Gloria Gonzalez
Cristian Smerdou
Diaz Laura Palomo
Original Assignee
Vivet Therapeutics
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 Vivet Therapeutics filed Critical Vivet Therapeutics
Publication of MX2021005435A publication Critical patent/MX2021005435A/es

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    • 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
    • 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
    • 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
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    • C07—ORGANIC CHEMISTRY
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    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles

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

Abstract

La presente divulgación se relaciona con un vector para terapia génica para usarse en el tratamiento de colestasis intrahepática familiar progresiva tipo 2. Más específicamente, la presente invención se relaciona con un vector de virus adenoasociado que comprende la secuencia de codones optimizados que codifica para la BSEP para el tratamiento de PFIC2.
MX2021005435A 2018-11-07 2019-11-06 Transgen abcb11 con codones optimizados para el tratamiento de colestasis intrahepatica familiar progresiva tipo 2 (pfic2). MX2021005435A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP18306458 2018-11-07
PCT/EP2019/080341 WO2020094693A1 (en) 2018-11-07 2019-11-06 Codon-optimized abcb11 transgene for the treatment of progressive familial intrahepatic cholestasis type 2 (pfic2)

Publications (1)

Publication Number Publication Date
MX2021005435A true MX2021005435A (es) 2021-09-08

Family

ID=65496678

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2021005435A MX2021005435A (es) 2018-11-07 2019-11-06 Transgen abcb11 con codones optimizados para el tratamiento de colestasis intrahepatica familiar progresiva tipo 2 (pfic2).

Country Status (12)

Country Link
US (1) US20220002751A1 (es)
EP (1) EP3877000A1 (es)
JP (1) JP2022512930A (es)
KR (1) KR20210091736A (es)
CN (1) CN112955186A (es)
AU (1) AU2019375450A1 (es)
BR (1) BR112021008434A2 (es)
CA (1) CA3117274A1 (es)
IL (1) IL282973A (es)
MX (1) MX2021005435A (es)
SG (1) SG11202104634UA (es)
WO (1) WO2020094693A1 (es)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4267197A1 (en) * 2020-12-23 2023-11-01 Vivet Therapeutics Minimal bile acid inducible promoters for gene therapy
GB202205606D0 (en) * 2022-04-14 2022-06-01 Genespire S R L Lentiviral vector

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US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
DE69233013T2 (de) 1991-08-20 2004-03-04 The Government Of The United States Of America As Represented By The Secretary Of National Institute Of Health, Office Of Technology Transfer Adenovirus vermittelter gentransfer in den gastrointestinaltrakt
FR2836924B1 (fr) 2002-03-08 2005-01-14 Vivalis Lignees de cellules aviaires utiles pour la production de substances d'interet
WO2005010039A1 (en) * 2003-07-31 2005-02-03 Pfizer Products Inc. Bsep polypeptide variants and uses thereof
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FR2884255B1 (fr) 2005-04-11 2010-11-05 Vivalis Utilisation de lignees de cellules souches aviaires ebx pour la production de vaccin contre la grippe
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JP2007295929A (ja) * 2006-04-05 2007-11-15 Geno Membrane:Kk イヌＢｓｅｐ遺伝子
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AU2022237643A1 (en) * 2021-03-19 2023-09-28 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing pfic therapeutics

Also Published As

Publication number Publication date
AU2019375450A1 (en) 2021-06-17
US20220002751A1 (en) 2022-01-06
IL282973A (en) 2021-06-30
SG11202104634UA (en) 2021-06-29
KR20210091736A (ko) 2021-07-22
CN112955186A (zh) 2021-06-11
CA3117274A1 (en) 2020-05-14
BR112021008434A2 (pt) 2021-09-28
JP2022512930A (ja) 2022-02-07
WO2020094693A1 (en) 2020-05-14
EP3877000A1 (en) 2021-09-15

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