MX2021012681A - Composiciones y metodos para el tratamiento de la fibrosis quistica. - Google Patents

Composiciones y metodos para el tratamiento de la fibrosis quistica.

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Publication number
MX2021012681A
MX2021012681A MX2021012681A MX2021012681A MX2021012681A MX 2021012681 A MX2021012681 A MX 2021012681A MX 2021012681 A MX2021012681 A MX 2021012681A MX 2021012681 A MX2021012681 A MX 2021012681A MX 2021012681 A MX2021012681 A MX 2021012681A
Authority
MX
Mexico
Prior art keywords
treatment
methods
cystic fibrosis
compositions
pharmaceutical compositions
Prior art date
Application number
MX2021012681A
Other languages
English (en)
Inventor
Ziying Yan
John F Engelhardt
Yinghua Tang
Eric Yuen
Shen Lin
Original Assignee
Spirovant Sciences 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 Spirovant Sciences Inc filed Critical Spirovant Sciences Inc
Publication of MX2021012681A publication Critical patent/MX2021012681A/es

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    • 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
    • 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
    • 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
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • 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
    • C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (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

En la presente se proveen polinucleótidos, vectores de rAAV, composiciones farmacéuticas y métodos para elaborarlos y usarlos, por ejemplo, en el tratamiento de la fibrosis quística (CF). Por ejemplo, la descripción proporciona un virus adenoasociado recombinante (rAAV) que incluye, en una modalidad, una proteína de la cáspide AV.TL65 y un polinucleótido que incluye un potenciador F5 y un promotor tg83 enlazados de forma operativa a un minigén CFTR¿R, composiciones farmacéuticas y métodos para usarlo, por ejemplo, en el tratamiento de la CF.
MX2021012681A 2019-04-15 2020-04-15 Composiciones y metodos para el tratamiento de la fibrosis quistica. MX2021012681A (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201962833972P 2019-04-15 2019-04-15
US201962926308P 2019-10-25 2019-10-25
US202062967214P 2020-01-29 2020-01-29
PCT/US2020/028264 WO2020214668A1 (en) 2019-04-15 2020-04-15 Compositions and methods for treatment of cystic fibrosis

Publications (1)

Publication Number Publication Date
MX2021012681A true MX2021012681A (es) 2022-03-25

Family

ID=70847479

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2021012681A MX2021012681A (es) 2019-04-15 2020-04-15 Composiciones y metodos para el tratamiento de la fibrosis quistica.

Country Status (13)

Country Link
US (1) US20220241436A1 (es)
EP (1) EP3955971A1 (es)
JP (1) JP2022529457A (es)
KR (1) KR20220044899A (es)
CN (1) CN114641318A (es)
AU (1) AU2020260076A1 (es)
BR (1) BR112021020708A2 (es)
CA (1) CA3137015A1 (es)
CL (1) CL2021002702A1 (es)
IL (1) IL287260A (es)
MX (1) MX2021012681A (es)
SG (1) SG11202111334SA (es)
WO (1) WO2020214668A1 (es)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11702672B2 (en) 2016-02-08 2023-07-18 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
MX2018010842A (es) 2016-03-07 2019-07-04 Univ Iowa Res Found Expresion mediada por el virus adeno-asociado (aav) usando un promotor y pontenciador sintetico.
US11142775B2 (en) 2017-01-13 2021-10-12 University Of Iowa Research Foundation Bocaparvovirus small noncoding RNA and uses thereof
WO2020214672A1 (en) * 2019-04-15 2020-10-22 University Of Iowa Research Foundation Methods and compositions for transgene expression
WO2022006253A2 (en) * 2020-06-30 2022-01-06 University Of Iowa Research Foundation Methods and compositions for administering recombinant viral vectors
US20240115738A1 (en) 2021-04-15 2024-04-11 Spirovant Sciences, Inc. Methods and compositions for treatment of cystic fibrosis

Family Cites Families (15)

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Publication number Priority date Publication date Assignee Title
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US5837484A (en) 1993-11-09 1998-11-17 Medical College Of Ohio Stable cell lines capable of expressing the adeno-associated virus replication gene
CA2176117C (en) 1993-11-09 2006-01-03 Terence R. Flotte Generation of high titers of recombinant aav vectors
US5656785A (en) 1995-08-07 1997-08-12 The Charles Stark Draper Laboratory, Inc. Micromechanical contact load force sensor for sensing magnitude and distribution of loads and tool employing micromechanical contact load force sensor
DE69737424T2 (de) 1996-11-19 2007-06-21 Surgx Corp., Fremont Schutzvorrichtung gegen transiente spannungen und verfahren zu deren herstellung
EP0950091A2 (en) 1996-12-18 1999-10-20 Targeted Genetics Corporation Aav split-packaging genes and cell lines comprising such genes for use in the production of recombinant aav vectors
WO2004090145A2 (en) 2003-03-31 2004-10-21 University Of Iowa Research Foundation Compounds and methods to enhance raav transduction
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
WO2005056762A2 (en) * 2003-12-05 2005-06-23 University Of Iowa Research Foundation Truncated cmv promoters and vectors containing same
EP3446712A1 (en) * 2013-03-14 2019-02-27 Translate Bio Ma, Inc. Cftr mrna compositions and related methods and uses
JP6516725B2 (ja) * 2013-04-08 2019-05-22 ユニバーシテイ・オブ・アイオワ・リサーチ・フアウンデーシヨン キメラアデノ随伴ウイルス／ボカウイルスパルボウイルスベクター
WO2016118787A1 (en) * 2015-01-21 2016-07-28 University Of Florida Research Foundation, Inc. Engineered receptor/ligand system for delivery of therapeutic agents
MX2018010842A (es) * 2016-03-07 2019-07-04 Univ Iowa Res Found Expresion mediada por el virus adeno-asociado (aav) usando un promotor y pontenciador sintetico.
US10550405B2 (en) * 2017-03-15 2020-02-04 The University Of North Carolina At Chapel Hill Rational polyploid adeno-associated virus vectors and methods of making and using the same
WO2020214672A1 (en) * 2019-04-15 2020-10-22 University Of Iowa Research Foundation Methods and compositions for transgene expression

Also Published As

Publication number Publication date
US20220241436A1 (en) 2022-08-04
IL287260A (en) 2021-12-01
BR112021020708A2 (pt) 2022-03-15
CN114641318A (zh) 2022-06-17
SG11202111334SA (en) 2021-11-29
KR20220044899A (ko) 2022-04-12
WO2020214668A1 (en) 2020-10-22
JP2022529457A (ja) 2022-06-22
CA3137015A1 (en) 2020-10-22
AU2020260076A1 (en) 2021-12-09
EP3955971A1 (en) 2022-02-23
CL2021002702A1 (es) 2022-11-11

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