AU2014223243B2 - Talen-based gene correction - Google Patents

Talen-based gene correction Download PDF

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Publication number
AU2014223243B2
AU2014223243B2 AU2014223243A AU2014223243A AU2014223243B2 AU 2014223243 B2 AU2014223243 B2 AU 2014223243B2 AU 2014223243 A AU2014223243 A AU 2014223243A AU 2014223243 A AU2014223243 A AU 2014223243A AU 2014223243 B2 AU2014223243 B2 AU 2014223243B2
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Australia
Prior art keywords
talen
nucleic acid
cell
sequence
gene
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Ceased
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AU2014223243A
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AU2014223243A1 (en
Inventor
Bruce Blazar
Mark J. Osborn
Jakub Tolar
Daniel F. Voytas
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University of Minnesota System
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University of Minnesota Twin Cities
University of Minnesota System
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Publication of AU2014223243B2 publication Critical patent/AU2014223243B2/en
Priority to AU2020200307A priority Critical patent/AU2020200307A1/en
Ceased legal-status Critical Current
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • 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
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Immunology (AREA)
  • Hematology (AREA)
  • Biophysics (AREA)
  • Diabetes (AREA)
  • Dermatology (AREA)
  • Rheumatology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Neurology (AREA)
  • Plant Pathology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
AU2014223243A 2013-03-01 2014-02-28 Talen-based gene correction Ceased AU2014223243B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2020200307A AU2020200307A1 (en) 2013-03-01 2020-01-16 Talen-based gene correction

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361771735P 2013-03-01 2013-03-01
US61/771,735 2013-03-01
PCT/US2014/019322 WO2014134412A1 (en) 2013-03-01 2014-02-28 Talen-based gene correction

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2020200307A Division AU2020200307A1 (en) 2013-03-01 2020-01-16 Talen-based gene correction

Publications (2)

Publication Number Publication Date
AU2014223243A1 AU2014223243A1 (en) 2015-09-10
AU2014223243B2 true AU2014223243B2 (en) 2019-10-17

Family

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Family Applications (2)

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AU2014223243A Ceased AU2014223243B2 (en) 2013-03-01 2014-02-28 Talen-based gene correction
AU2020200307A Abandoned AU2020200307A1 (en) 2013-03-01 2020-01-16 Talen-based gene correction

Family Applications After (1)

Application Number Title Priority Date Filing Date
AU2020200307A Abandoned AU2020200307A1 (en) 2013-03-01 2020-01-16 Talen-based gene correction

Country Status (7)

Country Link
US (4) US9393257B2 (enExample)
EP (2) EP3567104A1 (enExample)
JP (3) JP6480874B2 (enExample)
AU (2) AU2014223243B2 (enExample)
CA (1) CA2902954A1 (enExample)
ES (1) ES2750550T3 (enExample)
WO (1) WO2014134412A1 (enExample)

Families Citing this family (60)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9850497B2 (en) * 2013-11-04 2017-12-26 Regents Of The University Of Minnesota Gene targeting methods and tools
KR102415811B1 (ko) 2014-01-31 2022-07-04 팩터 바이오사이언스 인크. 핵산 제조 및 송달을 위한 방법 및 산물
RU2756247C2 (ru) 2014-11-17 2021-09-28 Эдисет Био, Инк. Генетически модифицированные гамма дельта т-клетки
US11241505B2 (en) 2015-02-13 2022-02-08 Factor Bioscience Inc. Nucleic acid products and methods of administration thereof
CN104726495B (zh) * 2015-03-25 2018-06-19 西北农林科技大学 一种基于talen介导的基因打靶敲除山羊blg的载体及重组细胞
EP4600366A3 (en) 2015-03-31 2025-10-22 SOHM, Inc. Cas 9 retroviral integrase systems for targeted incorporation of a dna sequence into a genome of a cell
EP3292219B9 (en) 2015-05-04 2022-05-18 Ramot at Tel-Aviv University Ltd. Methods and kits for fragmenting dna
EP3770168A1 (en) 2015-05-18 2021-01-27 TCR2 Therapeutics Inc. Compositions and methods for tcr reprogramming using fusion proteins
EP4339287A3 (en) 2015-07-31 2024-05-22 Regents Of The University Of Minnesota Modified cells and methods of therapy
WO2017075248A1 (en) * 2015-10-27 2017-05-04 The Board Of Trustees Of The Leland Stanford Junior University Treatment of epidermolysis bullosa by injection of autologous collagen vii overexpressing leukocytes
ES2796488T3 (es) * 2016-04-08 2020-11-27 Krystal Biotech Inc Composiciones para el uso en métodos para el tratamiento de heridas, trastornos y enfermedades de la piel
CN118421557A (zh) 2016-05-12 2024-08-02 阿迪塞特治疗公司 选择性扩增γδT细胞群的方法及其组合物
WO2018026953A1 (en) 2016-08-02 2018-02-08 TCR2 Therapeutics Inc. Compositions and methods for tcr reprogramming using fusion proteins
IL264439B2 (en) 2016-08-17 2024-08-01 Factor Bioscience Inc non-viral, cell-free composition comprising a synthetic messenger RNA (MRNA) encoding a gene-editing protein for use in treating cancer, and a synthetic RNA encoding a gene-editing protein for use in treatment
BR112019007100A2 (pt) 2016-10-07 2019-06-25 Tcr2 Therapeutics Inc composições e métodos para reprogramação de receptores de célula t com o uso de proteínas de fusão
EP4338799A3 (en) 2016-10-18 2024-06-05 Regents of the University of Minnesota Tumor infiltrating lymphocytes and methods of therapy
CN110177803A (zh) 2016-11-22 2019-08-27 T细胞受体治疗公司 用于使用融合蛋白进行tcr重新编程的组合物和方法
CA3047999A1 (en) 2016-12-21 2018-06-28 TCR2 Therapeutics Inc. Engineered t cells for the treatment of cancer
WO2018140497A1 (en) 2017-01-25 2018-08-02 Efimov Igor R Apparatus and methods for in vitro preclinical human trials
TW201839136A (zh) 2017-02-06 2018-11-01 瑞士商諾華公司 治療血色素異常症之組合物及方法
WO2018154413A1 (en) * 2017-02-22 2018-08-30 Crispr Therapeutics Ag Materials and methods for treatment of dystrophic epidermolysis bullosa (deb) and other collagen type vii alpha 1 chain (col7a1) gene related conditions or disorders
PL3638776T3 (pl) 2017-06-14 2026-02-23 Technische Universität Dresden Sposoby i środki do zmiany genetycznej genomów z wykorzystaniem projektowanych enzymów rekombinujących dna
JP7712643B2 (ja) 2017-06-19 2025-07-24 国立大学法人東北大学 表皮水疱症の治療剤
CN111511375A (zh) 2017-06-30 2020-08-07 因提玛生物科学公司 用于基因治疗的腺相关病毒载体
CA3082818A1 (en) 2017-11-15 2019-05-23 Adicet Bio Inc. Methods for selective expansion of .delta.3 .gamma..delta. t-cell populations and compositions thereof
EP3775236A1 (en) 2018-04-12 2021-02-17 Krystal Biotech, LLC Compositions and methods for the treatment of autosomal recessive congenital ichthyosis
EP3781677A4 (en) 2018-04-16 2022-01-19 University of Massachusetts COMPOSITIONS AND METHODS FOR IMPROVED GENE EDITTING
MX2020011257A (es) 2018-04-27 2021-01-15 Krystal Biotech Inc Ácidos nucleicos recombinantes que codifican una o más proteínas cosméticas para usos estéticos.
WO2019217837A1 (en) 2018-05-11 2019-11-14 Memorial Sloan-Kettering Cancer Center T cell receptors targeting pik3ca mutations and uses thereof
EP3800998A1 (en) 2018-06-07 2021-04-14 The State of Israel, Ministry of Agriculture & Rural Development, Agricultural Research Organization (ARO) (Volcani Center) Methods of regenerating and transforming cannabis
EP3802839A1 (en) 2018-06-07 2021-04-14 The State of Israel, Ministry of Agriculture & Rural Development, Agricultural Research Organization (ARO) (Volcani Center) Nucleic acid constructs and methods of using same
EP3847243A1 (en) * 2018-09-07 2021-07-14 Wisconsin Alumni Research Foundation Generation of hematopoietic progenitor cells from human pluripotent stem cells
AU2019346549B2 (en) 2018-09-24 2024-09-12 Krystal Biotech, Inc. Compositions and methods for the treatment of Netherton Syndrome
EP3856914A1 (en) 2018-09-26 2021-08-04 Krystal Biotech, Inc. Compositions and methods for the treatment of skin diseases
EP3887395A4 (en) 2018-11-30 2022-07-27 Intima Bioscience, Inc. METHODS FOR IDENTIFICATION OF IMMUNOMODULATORY GENES
CA3126896A1 (en) 2018-12-03 2020-06-11 Adicet Bio Inc. Methods for selective in vivo expansion of .gamma. .delta. t-cell populations and compositions thereof
CN113454105A (zh) 2019-02-08 2021-09-28 克里斯托生物技术股份有限公司 用于递送cftr多肽的组合物和方法
MX2021012207A (es) 2019-04-05 2021-12-10 Rootpath Genomics Inc Composiciones y métodos para ensamblaje de genes del receptor de linfocitos t.
US20220259617A1 (en) 2019-06-13 2022-08-18 The General Hospital Corporation Engineered human-endogenous virus-like particles and methods of use thereof for delivery to cells
WO2020257684A1 (en) 2019-06-20 2020-12-24 University Of Massachusetts Compositions and methods for improved gene editing
CN114245745A (zh) 2019-06-20 2022-03-25 纪念斯隆-凯特琳癌症中心 靶向pik3ca突变的t细胞受体及其用途
CA3145425A1 (en) 2019-07-03 2021-01-07 Factor Bioscience Inc. Cationic lipids and uses thereof
WO2021011936A2 (en) 2019-07-18 2021-01-21 University Of Rochester Cell-type selective immunoprotection of cells
US10501404B1 (en) 2019-07-30 2019-12-10 Factor Bioscience Inc. Cationic lipids and transfection methods
WO2021046243A2 (en) 2019-09-03 2021-03-11 Myeloid Therapeutics, Inc. Methods and compositions for genomic integration
US12091681B2 (en) 2020-03-27 2024-09-17 Mendus B.V. Ex vivo use of modified cells of leukemic origin for enhancing the efficacy of adoptive cell therapy
WO2022003568A1 (en) 2020-06-30 2022-01-06 Dcprime B.V. Use of leukemia-derived cells in ovarian cancer vaccines
BR112023001272A2 (pt) 2020-07-24 2023-04-04 Massachusetts Gen Hospital Partículas semelhantes a vírus aprimoradas e métodos de uso das mesmas para entrega às células
IL301021A (en) 2020-09-01 2023-05-01 64 X Inc Mammalian cells and methods for engineering the same
KR20230088833A (ko) 2020-10-23 2023-06-20 루트패스 제노믹스, 인크. T-세포 수용체 식별을 위한 조성물 및 방법
WO2022155500A1 (en) 2021-01-14 2022-07-21 Senti Biosciences, Inc. Secretable payload regulation
KR20230135075A (ko) 2021-01-22 2023-09-22 멘두스 비.브이. 종양 백신접종 방법
WO2022190058A1 (en) 2021-03-12 2022-09-15 Dcprime B.V. Methods of vaccination and use of cd47 blockade
IL307350A (en) 2021-04-02 2023-11-01 Krystal Biotech Inc Viral vectors for cancer treatment
WO2022241029A1 (en) 2021-05-11 2022-11-17 Myeloid Therapeutics, Inc. Methods and compositions for genomic integration
WO2023086847A1 (en) 2021-11-10 2023-05-19 Encodia, Inc. Methods for barcoding macromolecules in individual cells
CN119677842A (zh) 2022-03-17 2025-03-21 科士华(南京)生物技术有限公司 人工合成肿瘤浸润淋巴细胞
EP4573114A4 (en) 2022-08-19 2026-07-29 Memorial Sloan Kettering Cancer Center T-LYMPHOCYTE RECEPTORS TARGETTING RAS MUTATIONS AND ASSOCIATED USES
WO2024105633A1 (en) 2022-11-18 2024-05-23 Kyoto Prefectural Public University Corporation Compositions for mitophagy induction and uses thereof
WO2025128981A1 (en) 2023-12-14 2025-06-19 Sohm, Inc. Compositions and methods for genome editing

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012065143A1 (en) * 2010-11-12 2012-05-18 The General Hospital Corporation Polycomb-associated non-coding rnas

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2002099070A2 (en) * 2001-06-04 2002-12-12 Corixa Corporation Compositions and methods for high-level, large-scale production of recombinant proteins
PT2816112T (pt) * 2009-12-10 2018-11-20 Univ Iowa State Res Found Inc Modificação do adn modificada pelo efector tal
CN103210087B (zh) * 2010-09-06 2015-04-29 淡马锡生命科学研究院有限公司 Xa10与AvrXa10之间的分子相互作用
EP2758529B1 (en) * 2011-09-21 2019-03-20 Sangamo Therapeutics, Inc. Methods and compositions for regulation of transgene expression

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012065143A1 (en) * 2010-11-12 2012-05-18 The General Hospital Corporation Polycomb-associated non-coding rnas

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
Molecular Therapy, 2012, Vol.20, Supplement 1, S26-S27 *
MUZNY ET AL., "Homo sapiens 3 PAC RP4-751 E10 complete sequence.", GENBANK, (2003-01-08), Database accession no. AC005923, URL: NCBI *
NING SUN ET AL, "Optimized TAL effector nucleases (TALENs) for use in treatment of sickle cell disease", MOLECULAR BIOSYSTEMS, (2012-01-01), vol. 8, no. 4, doi:10.1039/c2mb05461b, ISSN 1742-206X, page 1255 *
OSBORN MARK ET AL, HUMAN GENE THERAPY, vol. 23, no. 10, (2012-10-01), page A109, URL: http://online.liebertpub.com/doi/pdfplus/10.1089/hum.2012.2519 *
ZOU JIZHONG ET AL, MOLECULAR THERAPY, vol. 20, no. Supplement 1, (2012-05-01), pages S110 - S111, URL: http://www.nature.com/mt/journal/v20/n1s/pdf/mt201286a.pdf *

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US20210187006A1 (en) 2021-06-24
EP3567104A1 (en) 2019-11-13
NZ711254A (en) 2021-02-26
JP2016512960A (ja) 2016-05-12
JP2019089821A (ja) 2019-06-13
JP6893945B2 (ja) 2021-06-23
JP2021088565A (ja) 2021-06-10
CA2902954A1 (en) 2014-09-04
US20140256798A1 (en) 2014-09-11
US20160367588A1 (en) 2016-12-22
EP2961262A1 (en) 2016-01-06
AU2020200307A1 (en) 2020-02-06
HK1219021A1 (en) 2017-03-24
WO2014134412A1 (en) 2014-09-04
US10973844B2 (en) 2021-04-13
ES2750550T3 (es) 2020-03-26
US9393257B2 (en) 2016-07-19
US10172880B2 (en) 2019-01-08
EP2961262B1 (en) 2019-07-24
JP6480874B2 (ja) 2019-03-13
AU2014223243A1 (en) 2015-09-10
US20190054111A1 (en) 2019-02-21
EP2961262A4 (en) 2016-10-12

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