BR112019004594A2 - terapia genética para pacientes com anemia de fanconi - Google Patents

terapia genética para pacientes com anemia de fanconi

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Publication number
BR112019004594A2
BR112019004594A2 BR112019004594A BR112019004594A BR112019004594A2 BR 112019004594 A2 BR112019004594 A2 BR 112019004594A2 BR 112019004594 A BR112019004594 A BR 112019004594A BR 112019004594 A BR112019004594 A BR 112019004594A BR 112019004594 A2 BR112019004594 A2 BR 112019004594A2
Authority
BR
Brazil
Prior art keywords
gene therapy
patients
fanconi anemia
methods
anemia
Prior art date
Application number
BR112019004594A
Other languages
English (en)
Inventor
Gonzalez Africa
Guenechea Guillermo
Antonio Casado Jose
Carlos Segovia Jose
A Bueren Juan
Sevilla Julian
Rio Paula
Navarro Susana
Original Assignee
Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P
Consorcio Centro De Investig Biomedica En Red M P
Fundacion Instituto De Investig Sanitaria Fundacion Jimenez Diaz
Fundacion Para La Investigacion Biomedica Del Hospital Infantil Univ Nino Jesus
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 Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P, Consorcio Centro De Investig Biomedica En Red M P, Fundacion Instituto De Investig Sanitaria Fundacion Jimenez Diaz, Fundacion Para La Investigacion Biomedica Del Hospital Infantil Univ Nino Jesus filed Critical Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P
Publication of BR112019004594A2 publication Critical patent/BR112019004594A2/pt

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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
    • C—CHEMISTRY; METALLURGY
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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
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
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    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
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    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1217—Phosphotransferases with a carboxyl group as acceptor (2.7.2)
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    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/02—Phosphotransferases with a carboxy group as acceptor (2.7.2)
    • C12Y207/02003—Phosphoglycerate kinase (2.7.2.3)
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    • C12N2730/10011—Hepadnaviridae
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    • C12N2730/10141—Use of virus, viral particle or viral elements as a vector
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    • C12N2730/00—Reverse transcribing DNA viruses
    • C12N2730/00011—Details
    • C12N2730/10011—Hepadnaviridae
    • C12N2730/10111—Orthohepadnavirus, e.g. hepatitis B virus
    • C12N2730/10171—Demonstrated in vivo effect
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2740/00—Reverse transcribing RNA viruses
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    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • 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
    • C12N2740/15071—Demonstrated in vivo effect

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

Abstract

a presente invenção fornece composições e métodos para recuperar a expressão de fanca em células com produto de gene fanca diminuído ou ausente. em particular, métodos e composições para a terapia genética de anemia de fanconi são divulgados.
BR112019004594A 2016-09-08 2017-09-08 terapia genética para pacientes com anemia de fanconi BR112019004594A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662385185P 2016-09-08 2016-09-08
US201662412028P 2016-10-24 2016-10-24
PCT/US2017/050837 WO2018049273A1 (en) 2016-09-08 2017-09-08 Gene therapy for patients with fanconi anemia

Publications (1)

Publication Number Publication Date
BR112019004594A2 true BR112019004594A2 (pt) 2019-07-02

Family

ID=61561668

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112019004594A BR112019004594A2 (pt) 2016-09-08 2017-09-08 terapia genética para pacientes com anemia de fanconi

Country Status (12)

Country Link
US (1) US20190203225A1 (pt)
EP (1) EP3510162A4 (pt)
JP (2) JP7197466B2 (pt)
KR (1) KR102672636B1 (pt)
CN (1) CN110536966A (pt)
AU (2) AU2017322511B2 (pt)
BR (1) BR112019004594A2 (pt)
CA (1) CA3035605A1 (pt)
IL (1) IL265196A (pt)
MX (1) MX2019002699A (pt)
SG (1) SG11201901718VA (pt)
WO (1) WO2018049273A1 (pt)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20210154237A1 (en) * 2018-04-11 2021-05-27 Rocket Pharmaceuticals, Ltd. Compositions and methods for stem cell transplant
KR20210030965A (ko) * 2018-07-12 2021-03-18 로켓 파마슈티컬스, 리미티드 다논병을 치료하기 위한 유전자 요법 벡터
CN110904102A (zh) * 2018-09-18 2020-03-24 中国科学院上海生命科学研究院 一种用于重组蛋白质表达的启动子
WO2020169666A1 (en) * 2019-02-21 2020-08-27 F. Hoffmann-La Roche Ag Improved nucleic acid target enrichment and related methods
IL300263A (en) 2020-08-07 2023-03-01 Spacecraft Seven Llc Plakophilin-2 (PKP2) gene therapy using an AAV vector
CA3213792A1 (en) * 2021-04-26 2022-11-03 Jonathan Thon Compositions and methods related to megakaryocyte-derived extracellular vesicles for fanconi anemia
WO2023183909A2 (en) * 2022-03-25 2023-09-28 Modernatx, Inc. Polynucleotides encoding fanconi anemia, complementation group proteins for the treatment of fanconi anemia

Family Cites Families (14)

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Publication number Priority date Publication date Assignee Title
US5952190A (en) * 1996-10-04 1999-09-14 Fanconi Anemia Research Fund, Inc. cDNA for fanconi anemia complementation group A
GB0024550D0 (pt) * 2000-10-06 2000-11-22 Oxford Biomedica Ltd
JP2007054069A (ja) * 2005-08-24 2007-03-08 Medizinische Hochschule Hannover 自己不活性化レトロウイルスベクター
US8556882B2 (en) * 2009-04-30 2013-10-15 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Inducible interleukin-12
DE102009021592A1 (de) * 2009-05-15 2010-11-18 Medizinische Hochschule Hannover ASLV-Vektorsystem
JP2014513727A (ja) * 2011-05-16 2014-06-05 ジェンザイム・コーポレーション Ｃｘｃｒ４拮抗薬の使用
CA2879997A1 (en) * 2012-07-25 2014-01-30 The Broad Institute, Inc. Inducible dna binding proteins and genome perturbation tools and applications thereof
US20160194660A1 (en) * 2012-12-21 2016-07-07 Merck Sharp & Dohme Corp. Expression vectors for recombinant protein production in mammalian cells
GB201318347D0 (en) * 2013-10-16 2013-11-27 Ucl Business Plc Retroviral vectors
EP3068440B1 (en) * 2013-11-15 2020-01-08 Northwestern University Inhibition of oxidative stress in atrial fibrillation
BR112016025519A2 (pt) * 2014-05-01 2018-01-16 Univ Washington engenharia genética in vivo com vetores de adenovírus
WO2015188191A1 (en) * 2014-06-06 2015-12-10 Wong Wilson W Dna recombinase circuits for logical control of gene expression
EP3247808B1 (en) * 2015-01-21 2021-05-05 Fred Hutchinson Cancer Research Center Point-of-care and/or portable platform for gene therapy
WO2016183593A2 (en) * 2015-05-14 2016-11-17 The Regents Of The University Of California Prenatal therapy

Also Published As

Publication number Publication date
SG11201901718VA (en) 2019-03-28
CN110536966A (zh) 2019-12-03
IL265196A (en) 2019-05-30
RU2019108981A (ru) 2020-10-08
JP2019533434A (ja) 2019-11-21
AU2021273525A1 (en) 2021-12-16
MX2019002699A (es) 2019-12-16
KR102672636B1 (ko) 2024-06-10
JP7197466B2 (ja) 2022-12-27
AU2017322511B2 (en) 2021-08-26
EP3510162A4 (en) 2020-02-19
RU2019108981A3 (pt) 2020-12-24
US20190203225A1 (en) 2019-07-04
KR20190062426A (ko) 2019-06-05
JP2022160505A (ja) 2022-10-19
EP3510162A1 (en) 2019-07-17
AU2017322511A1 (en) 2019-03-28
WO2018049273A1 (en) 2018-03-15
CA3035605A1 (en) 2018-03-15

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