AU2017322511B2 - Gene therapy for patients with Fanconi anemia - Google Patents

Gene therapy for patients with Fanconi anemia Download PDF

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Publication number
AU2017322511B2
AU2017322511B2 AU2017322511A AU2017322511A AU2017322511B2 AU 2017322511 B2 AU2017322511 B2 AU 2017322511B2 AU 2017322511 A AU2017322511 A AU 2017322511A AU 2017322511 A AU2017322511 A AU 2017322511A AU 2017322511 B2 AU2017322511 B2 AU 2017322511B2
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Prior art keywords
cells
sequence
cell
fanca
vector
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AU2017322511A1 (en
Inventor
Juan A. BUEREN
Jose Antonio CASADO
Africa GONZALEZ
Guillermo GUENECHEA
Susana NAVARRO
Paula RIO
Jose Carlos SEGOVIA
Julian SEVILLA
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Fundacion Para La Investigacion Biomedica Del Hospital Infantil Universitario Nino Jesus
Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas
Centro de Investigación Biomédica en Red
Hospital Universitario Fundación Jiménez Díaz
Original Assignee
Fundacion Para La Investigacion Biomedica Del Hospital Infantil Univ Nino Jesus
Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas
Centro de Investigación Biomédica en Red
Hospital Universitario Fundación Jiménez Díaz
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Priority to AU2021273525A priority Critical patent/AU2021273525B2/en
Priority to AU2024287211A priority patent/AU2024287211A1/en
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    • 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
    • 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
    • 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
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • A61K48/0066—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
    • 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
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    • 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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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • 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
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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
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    • C12N5/0602—Vertebrate cells
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    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
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    • C12Y207/02—Phosphotransferases with a carboxy group as acceptor (2.7.2)
    • C12Y207/02003—Phosphoglycerate kinase (2.7.2.3)
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    • C12N2730/00011—Details
    • C12N2730/10011—Hepadnaviridae
    • C12N2730/10111—Orthohepadnavirus, e.g. hepatitis B virus
    • C12N2730/10141—Use of virus, viral particle or viral elements as a vector
    • C12N2730/10143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2730/00011—Details
    • C12N2730/10011—Hepadnaviridae
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    • C12N2740/10011—Retroviridae
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    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
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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
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AU2017322511A 2016-09-08 2017-09-08 Gene therapy for patients with Fanconi anemia Active AU2017322511B2 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
AU2021273525A AU2021273525B2 (en) 2016-09-08 2021-11-22 Gene therapy for patients with Fanconi anemia
AU2024287211A AU2024287211A1 (en) 2016-09-08 2024-12-24 Gene therapy for patients with Fanconi anemia

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662385185P 2016-09-08 2016-09-08
US62/385,185 2016-09-08
US201662412028P 2016-10-24 2016-10-24
US62/412,028 2016-10-24
PCT/US2017/050837 WO2018049273A1 (en) 2016-09-08 2017-09-08 Gene therapy for patients with fanconi anemia

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2021273525A Division AU2021273525B2 (en) 2016-09-08 2021-11-22 Gene therapy for patients with Fanconi anemia

Publications (2)

Publication Number Publication Date
AU2017322511A1 AU2017322511A1 (en) 2019-03-28
AU2017322511B2 true AU2017322511B2 (en) 2021-08-26

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AU2017322511A Active AU2017322511B2 (en) 2016-09-08 2017-09-08 Gene therapy for patients with Fanconi anemia
AU2021273525A Active AU2021273525B2 (en) 2016-09-08 2021-11-22 Gene therapy for patients with Fanconi anemia
AU2024287211A Pending AU2024287211A1 (en) 2016-09-08 2024-12-24 Gene therapy for patients with Fanconi anemia

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AU2021273525A Active AU2021273525B2 (en) 2016-09-08 2021-11-22 Gene therapy for patients with Fanconi anemia
AU2024287211A Pending AU2024287211A1 (en) 2016-09-08 2024-12-24 Gene therapy for patients with Fanconi anemia

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US (2) US20190203225A1 (enExample)
EP (1) EP3510162A4 (enExample)
JP (3) JP7197466B2 (enExample)
KR (2) KR20240151726A (enExample)
CN (1) CN110536966A (enExample)
AU (3) AU2017322511B2 (enExample)
CA (1) CA3035605A1 (enExample)
IL (2) IL320371A (enExample)
MX (1) MX2019002699A (enExample)
SG (1) SG11201901718VA (enExample)
WO (1) WO2018049273A1 (enExample)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102673203B1 (ko) 2016-04-20 2024-06-10 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피. Pklr의 유전자 발현 증강을 위한 조성물 및 방법
AU2018352583B2 (en) 2017-10-16 2025-04-17 Centro de Investigaciones Energeticas, Medioambientales Y Tecnologicas, O.A., M.P. Lentiviral vectors for delivery of PKLR to treat Pyruvate Kinase Deficiency
US12539316B2 (en) * 2018-04-11 2026-02-03 Spacecraft Seven, Llc Compositions and methods for stem cell transplant
IL278213B2 (en) 2018-04-27 2026-04-01 Rocket Pharmaceuticals Ltd A gene therapy vector encoding an e3 ubiquitin protein ligase (park2) and use thereof in treating or inhibiting the onset of parkinson’s disease (pd)
EP3820537A4 (en) * 2018-07-12 2022-04-13 Spacecraft Seven, LLC GENE THERAPY VECTORS FOR TREATMENT OF DANON DISEASE
IL280465B2 (en) * 2018-07-30 2025-03-01 Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P Methods for gene modification of hematopoietic cells
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
CN113454235A (zh) * 2019-02-21 2021-09-28 豪夫迈·罗氏有限公司 经改进的核酸靶标富集和相关方法
KR20220012266A (ko) * 2019-05-23 2022-02-03 스페이스크래프트 세븐, 엘엘씨 영아 악성 골화석증을 위한 유전자 요법 벡터
DE102020111571A1 (de) 2020-03-11 2021-09-16 Immatics US, Inc. Wpre-mutantenkonstrukte, zusammensetzungen und zugehörige verfahren
WO2022032226A1 (en) 2020-08-07 2022-02-10 Spacecraft Seven, Llc Plakophilin-2 (pkp2) gene therapy using aav vector
AU2022264498A1 (en) * 2021-04-26 2023-09-28 Strm.Bio Incorporated 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
WO2025137373A1 (en) * 2023-12-19 2025-06-26 Cellarity, Inc. Disease detection systems and methods

Family Cites Families (14)

* Cited by examiner, † Cited by third party
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 (enExample) 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
WO2010126766A1 (en) 2009-04-30 2010-11-04 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
RU2638802C2 (ru) 2011-05-16 2017-12-15 Джензим Корпорейшн Применение антагонистов cxcr4
CN116622704A (zh) * 2012-07-25 2023-08-22 布罗德研究所有限公司 可诱导的dna结合蛋白和基因组干扰工具及其应用
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
WO2015073922A2 (en) 2013-11-15 2015-05-21 Northwestern University Inhibition of oxidative stress in atrial fibrillation
CA2947466A1 (en) 2014-05-01 2015-11-05 University Of Washington In vivo gene engineering with adenoviral vectors
US11030531B2 (en) * 2014-06-06 2021-06-08 Trustees Of Boston University DNA recombinase circuits for logical control of gene expression
US10350245B2 (en) 2015-01-21 2019-07-16 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

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
AFRICA GONZALEZ-MURILLO ET AL, "Development of Lentiviral Vectors with Optimized Transcriptional Activity for the Gene Therapy of Patients with Fanconi Anemia", HUMAN GENE THERAPY, (2010-05-01), vol. 21, no. 5, pages 623 - 630 *
JAKUB TOLAR ET AL, "Stem Cell Gene Therapy for Fanconi Anemia: Report from the 1st International Fanconi Anemia Gene Therapy Working Group Meeting", MOLECULAR THERAPY (2011-07-01), vol. 19, no. 7, pages 1193 - 1198 *
P S BECKER ET AL, "Preclinical correction of human Fanconi anemia complementation group A bone marrow cells using a safety-modified lentiviral vector", GENE THERAPY, GB, (2010-05-20), vol. 17, no. 10, pages 1244 - 1252 *

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