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 Energeticas Medioambientales y Tecnologicas CIEMAT
Centro de Investigacion Biomedica en Red CIBER
Instituto de Investigacion Sanitaria Fundacion Jimenez Diaz
Original Assignee
Fundacion Para La Investigacion Biomedica Del Hospital Infantil Univ Nino Jesus
Centro de Investigaciones Energeticas Medioambientales y Tecnologicas CIEMAT
Centro de Investigacion Biomedica en Red CIBER
Instituto de Investigacion Sanitaria Fundacion Jimenez Diaz
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Priority to AU2021273525A priority Critical patent/AU2021273525B2/en
Priority to AU2024287211A priority patent/AU2024287211A1/en
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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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    • 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
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    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
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    • 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
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    • C12Y207/02—Phosphotransferases with a carboxy group as acceptor (2.7.2)
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    • C12N2730/10141—Use of virus, viral particle or viral elements as a vector
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    • C12N2730/10011—Hepadnaviridae
    • C12N2730/10111—Orthohepadnavirus, e.g. hepatitis B virus
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    • C12N2740/10011—Retroviridae
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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
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    • 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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    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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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

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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

Country Status (11)

Country Link
US (1) US20190203225A1 (https=)
EP (1) EP3510162A4 (https=)
JP (3) JP7197466B2 (https=)
KR (2) KR102672636B1 (https=)
CN (1) CN110536966A (https=)
AU (3) AU2017322511B2 (https=)
CA (1) CA3035605A1 (https=)
IL (2) IL265196B2 (https=)
MX (1) MX2019002699A (https=)
SG (1) SG11201901718VA (https=)
WO (1) WO2018049273A1 (https=)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3021572A1 (en) 2016-04-20 2017-10-26 Centro De Investigaciones Energeticas, Medioambientales Y Tecnologicas Compositions and methods for enhanced gene expression of pklr
EP3697452A4 (en) 2017-10-16 2021-11-24 Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, O.A., M.P. LENTIVIRAL VECTORS FOR ADMINISTRATION OF PKLR TO TREAT PYRUVATE KINASE DEFECTS
BR112020020841A2 (pt) * 2018-04-11 2021-01-19 Fundacion Para La Investigacion Biomedica Del Hospital Infantil Universitario Nino Jesus Composições e métodos para transplante de célulastronco
CA3096293A1 (en) 2018-04-27 2019-10-31 Rocket Pharmaceuticals, Ltd. Gene therapy for cns degeneration
WO2020014523A1 (en) * 2018-07-12 2020-01-16 Rocket Pharmaceuticals, Ltd. Gene therapy vectors for treatment of danon disease
KR20210046004A (ko) * 2018-07-30 2021-04-27 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피. 조혈 세포의 유전자 변형 방법
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
CN113994008A (zh) * 2019-05-23 2022-01-28 太空飞船七有限责任公司 用于婴儿恶性骨硬化病的基因治疗载体
DE102020111571A1 (de) 2020-03-11 2021-09-16 Immatics US, Inc. Wpre-mutantenkonstrukte, zusammensetzungen und zugehörige verfahren
JP2023538519A (ja) 2020-08-07 2023-09-08 スペースクラフト セブン リミテッド ライアビリティ カンパニー Ａａｖベクターを使用したプラコフィリン－２（ｐｋｐ２）遺伝子治療
JP2024518770A (ja) * 2021-04-26 2024-05-02 ストーム バイオ インコーポレイテッド ファンコニ貧血のための巨核球由来細胞外小胞に関する組成物及び方法
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 (https=) * 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
AU2010241864B2 (en) * 2009-04-30 2014-02-20 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
KR102530118B1 (ko) * 2012-07-25 2023-05-08 더 브로드 인스티튜트, 인코퍼레이티드 유도 ｄｎａ 결합 단백질 및 게놈 교란 도구 및 이의 적용
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
CN107405411A (zh) * 2014-05-01 2017-11-28 华盛顿大学 使用腺病毒载体的体内基因改造
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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KR20240151726A (ko) 2024-10-18
IL265196A (en) 2019-05-30
RU2019108981A (ru) 2020-10-08
CA3035605A1 (en) 2018-03-15
BR112019004594A2 (pt) 2019-07-02
US20190203225A1 (en) 2019-07-04
EP3510162A1 (en) 2019-07-17
KR102672636B1 (ko) 2024-06-10
IL320371A (en) 2025-06-01
AU2017322511A1 (en) 2019-03-28
IL265196B2 (en) 2025-10-01
JP2022160505A (ja) 2022-10-19
JP7197466B2 (ja) 2022-12-27
AU2024287211A1 (en) 2025-01-30
SG11201901718VA (en) 2019-03-28
AU2021273525B2 (en) 2024-09-26
JP2025043346A (ja) 2025-03-28
KR20190062426A (ko) 2019-06-05
MX2019002699A (es) 2019-12-16
EP3510162A4 (en) 2020-02-19
WO2018049273A1 (en) 2018-03-15
RU2019108981A3 (https=) 2020-12-24
JP2019533434A (ja) 2019-11-21
CN110536966A (zh) 2019-12-03
AU2021273525A1 (en) 2021-12-16
IL265196B1 (en) 2025-06-01

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