AU2019315438B2 - Methods for gene modification of hematopoietic cells - Google Patents

Methods for gene modification of hematopoietic cells

Info

Publication number
AU2019315438B2
AU2019315438B2 AU2019315438A AU2019315438A AU2019315438B2 AU 2019315438 B2 AU2019315438 B2 AU 2019315438B2 AU 2019315438 A AU2019315438 A AU 2019315438A AU 2019315438 A AU2019315438 A AU 2019315438A AU 2019315438 B2 AU2019315438 B2 AU 2019315438B2
Authority
AU
Australia
Prior art keywords
cells
poloxamer
pge2
hematopoietic cells
recombinant
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
AU2019315438A
Other languages
English (en)
Other versions
AU2019315438A1 (en
Inventor
Elena Almarza Novoa
Brian Beard
Juan Antonio Bueren Roncero
Kenneth Law
Cristina MESA NUNEZ
Susana NAVARRO ORDONEZ
Kinnari PATEL
Oscar QUINTANA BUSTAMANTE
Paula RIO GALDO
Jose Carlos Segovia Sanz
Gaurav D. SHAH
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas
Centro de Investigación Biomédica en Red
Hospital Universitario Fundación Jiménez Díaz
Spacecraft Seven LLC
Original Assignee
Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas
Centro de Investigación Biomédica en Red
Hospital Universitario Fundación Jiménez Díaz
Spacecraft Seven LLC
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 Investigaciones Energéticas, Medioambientales y Tecnológicas, Centro de Investigación Biomédica en Red, Hospital Universitario Fundación Jiménez Díaz, Spacecraft Seven LLC filed Critical Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas
Publication of AU2019315438A1 publication Critical patent/AU2019315438A1/en
Assigned to FUNDACION INSTITUTO DE INVESTIGACION SANITARIA FUNDACION JIMENEZ DIAZ, CONSORCIO CENTRO DE INVESTIGACION BIOMEDICA EN RED, M.P., Centro de Investigaciones Energeticas, Medioambientales Y Tecnologicas, O.A., M.P., SPACECRAFT SEVEN, LLC reassignment FUNDACION INSTITUTO DE INVESTIGACION SANITARIA FUNDACION JIMENEZ DIAZ Request for Assignment Assignors: Centro de Investigaciones Energeticas, Medioambientales Y Tecnologicas, O.A., M.P., CONSORCIO CENTRO DE INVESTIGACION BIOMEDICA EN RED, M.P., FUNDACION INSTITUTO DE INVESTIGACION SANITARIA FUNDACION JIMENEZ DIAZ, ROCKET PHARMACEUTICALS, LTD.
Application granted granted Critical
Publication of AU2019315438B2 publication Critical patent/AU2019315438B2/en
Priority to AU2026203310A priority Critical patent/AU2026203310A1/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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
    • 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
    • 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
    • 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
    • C12N2500/00—Specific components of cell culture medium
    • C12N2500/50—Soluble polymers, e.g. polyethyleneglycol [PEG]
    • 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
    • C12N2510/00—Genetically modified cells
    • 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
    • C12N2740/15041—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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Hematology (AREA)
  • Wood Science & Technology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Immunology (AREA)
  • General Health & Medical Sciences (AREA)
  • Cell Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Molecular Biology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
AU2019315438A 2018-07-30 2019-07-30 Methods for gene modification of hematopoietic cells Active AU2019315438B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2026203310A AU2026203310A1 (en) 2018-07-30 2026-04-30 Methods for gene modification of hematopoietic cells

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862712146P 2018-07-30 2018-07-30
US62/712,146 2018-07-30
PCT/US2019/044237 WO2020028430A1 (en) 2018-07-30 2019-07-30 Methods for gene modification of hematopoietic cells

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2026203310A Division AU2026203310A1 (en) 2018-07-30 2026-04-30 Methods for gene modification of hematopoietic cells

Publications (2)

Publication Number Publication Date
AU2019315438A1 AU2019315438A1 (en) 2021-01-28
AU2019315438B2 true AU2019315438B2 (en) 2026-02-05

Family

ID=69232048

Family Applications (2)

Application Number Title Priority Date Filing Date
AU2019315438A Active AU2019315438B2 (en) 2018-07-30 2019-07-30 Methods for gene modification of hematopoietic cells
AU2026203310A Pending AU2026203310A1 (en) 2018-07-30 2026-04-30 Methods for gene modification of hematopoietic cells

Family Applications After (1)

Application Number Title Priority Date Filing Date
AU2026203310A Pending AU2026203310A1 (en) 2018-07-30 2026-04-30 Methods for gene modification of hematopoietic cells

Country Status (12)

Country Link
US (1) US12685751B2 (enExample)
EP (1) EP3830248A4 (enExample)
JP (2) JP7664829B2 (enExample)
KR (1) KR20210046004A (enExample)
CN (1) CN112888777A (enExample)
AU (2) AU2019315438B2 (enExample)
BR (1) BR112021001757A2 (enExample)
CA (1) CA3106241A1 (enExample)
IL (2) IL280465B2 (enExample)
MX (1) MX2021001292A (enExample)
SG (1) SG11202100025QA (enExample)
WO (1) WO2020028430A1 (enExample)

Families Citing this family (10)

* 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)
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
BR112022021862A2 (pt) * 2020-05-27 2023-02-28 Univ Zuerich Aperfeiçoadores de transdução inovadores e usos dos mesmos
WO2022032226A1 (en) 2020-08-07 2022-02-10 Spacecraft Seven, Llc Plakophilin-2 (pkp2) gene therapy using aav vector
CN114763563B (zh) * 2021-01-12 2024-04-30 深圳华大临床检验中心 一种提高慢病毒感染效率的方法
AU2022264498A1 (en) 2021-04-26 2023-09-28 Strm.Bio Incorporated Compositions and methods related to megakaryocyte-derived extracellular vesicles for fanconi anemia
AU2024381806A1 (en) * 2023-11-14 2026-05-28 The University Of North Carolina At Chapel Hill Compositions and methods relating to antiviral therapeutics

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017139576A1 (en) * 2016-02-12 2017-08-17 Bluebird Bio, Inc. Vcn enhancer compositions and methods of using the same
WO2018106807A1 (en) * 2016-12-06 2018-06-14 Bluebird Bio, Inc. Gene therapy for mucopolysaccharidosis, type i

Family Cites Families (77)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0377676B1 (en) 1987-09-17 1995-05-24 Massachusetts Institute Of Technology Human erythroid-specific transcriptional enhancer
US5278056A (en) 1988-02-05 1994-01-11 The Trustees Of Columbia University In The City Of New York Retroviral packaging cell lines and process of using same
JP3866760B2 (ja) 1993-02-22 2007-01-10 ザ ロックフェラー ユニバーシティ 一過性トランスフェクションによる高力価のヘルパーフリーのレトロウイルスの生産
FR2705361B1 (fr) 1993-05-18 1995-08-04 Centre Nat Rech Scient Vecteurs viraux et utilisation en thérapie génique.
DE69435223D1 (de) 1993-10-25 2009-09-03 Canji Inc Rekombinanter Adenoviren-Vektor und Verfahren zur Verwendung
US5484720A (en) 1994-09-08 1996-01-16 Genentech, Inc. Methods for calcium phosphate transfection
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
US6027721A (en) 1996-05-20 2000-02-22 Cytotherapeutics, Inc. Device and method for encapsulated gene therapy
US6136597A (en) 1997-09-18 2000-10-24 The Salk Institute For Biological Studies RNA export element
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
US20020065236A1 (en) 1998-09-09 2002-05-30 Yew Nelson S. CpG reduced plasmids and viral vectors
US7994278B1 (en) 1999-08-06 2011-08-09 Nobel Biosciences Llc Biologically active polypeptides derived from a novel early stage pregnancy factor designated maternin (MA)
HK1048302A1 (zh) 1999-08-13 2003-03-28 大正制药株式会社 前列腺素衍生物
HK1050210B (en) 1999-10-12 2007-08-24 Institut Pasteur Lentiviral triplex dna, and vectors and recombinant cells containing lentiviral triplex dna
US20020013294A1 (en) 2000-03-31 2002-01-31 Delong Mitchell Anthony Cosmetic and pharmaceutical compositions and methods using 2-decarboxy-2-phosphinico derivatives
US6627442B1 (en) 2000-08-31 2003-09-30 Virxsys Corporation Methods for stable transduction of cells with hiv-derived viral vectors
GB0024550D0 (enExample) 2000-10-06 2000-11-22 Oxford Biomedica Ltd
US7575924B2 (en) 2000-11-13 2009-08-18 Research Development Foundation Methods and compositions relating to improved lentiviral vectors and their applications
ATE527347T1 (de) 2001-08-02 2011-10-15 Inst Clayton De La Rech Verfahren und zusammensetzungen im zusammenhang mit verbesserten lentivirusvektor- produktionssystemen
CA2462628C (en) 2001-10-02 2019-08-20 Institut Clayton De La Recherche Restricted expression lentiviral vectors
ATE446077T1 (de) 2002-05-02 2009-11-15 Univ Rochester Vektoren mit beiden isoformen von beta- hexosaminidase
WO2004094642A2 (en) 2003-04-24 2004-11-04 Fondazione Centro San Raffaele Del Monte Tabor Lentiviral vectors carrying synthetic bi-directional promoters and uses thereof
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
BE1016793A4 (fr) 2005-10-04 2007-06-05 Artelis Procede de culture de cellules et dispositif permettant sa mise en oeuvre.
US8324367B2 (en) 2006-11-03 2012-12-04 Medtronic, Inc. Compositions and methods for making therapies delivered by viral vectors reversible for safety and allele-specificity
WO2008136670A2 (en) 2007-05-02 2008-11-13 Erasmus University Medical Center Rotterdam Improved methods and means for lentiviral gene delivery
AU2008262478B9 (en) 2007-05-29 2014-06-19 Aldevron, L.L.C. Vectors and methods for genetic immunization
EP2520643B8 (en) 2007-12-07 2019-11-27 Miltenyi Biotec B.V. & Co. KG Sample processing systems and methods
DE102008063606A1 (de) 2008-12-18 2010-06-24 Eufets Ag Ein-Vektor Tet-System und dessen Verwendung
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
EP2486137B1 (en) 2009-10-05 2018-05-30 Ya-Fang Mei Replication-competent ad11p based viral vectors
EP3868888A1 (en) 2012-02-29 2021-08-25 Helmholtz Zentrum München - Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH) Retroviral transduction using poloxamers
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
US10280391B2 (en) 2012-12-11 2019-05-07 Pall Technology Uk Limited Recipient for cell cultivation
KR102955556B1 (ko) 2012-12-12 2026-04-22 더 브로드 인스티튜트, 인코퍼레이티드 서열 조작을 위한 crispr-cas 성분 시스템, 방법 및 조성물
US20160194660A1 (en) 2012-12-21 2016-07-07 Merck Sharp & Dohme Corp. Expression vectors for recombinant protein production in mammalian cells
EP2792742A1 (en) 2013-04-17 2014-10-22 Universitätsklinikum Hamburg-Eppendorf (UKE) Gene-therapy vectors for treating cardiomyopathy
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
DK3194602T3 (da) * 2014-09-18 2020-02-17 Univ Montreal Forbindelser og fremgangsmåder til at forstærke virusgenoverførsel til humane hæmatopoietiske celler
GB201417042D0 (en) 2014-09-29 2014-11-12 Fkd Therapies Oy Method
MA41346A (fr) 2015-01-12 2017-11-21 Juno Therapeutics Inc Eléments régulateurs post-transcriptionnels d'hépatite modifiée
US10350245B2 (en) 2015-01-21 2019-07-16 Fred Hutchinson Cancer Research Center Point-of-care and/or portable platform for gene therapy
MA41506A (fr) 2015-02-13 2017-12-19 Takeda Vaccines Inc Procédés de production de virus pour produire des vaccins
MX2017011615A (es) 2015-03-10 2018-04-11 Univ Columbia Construcciones de vectores virales adeno-asociados de glut1 recombinantes y métodos relacionados para restablecer la expresión de glut1.
WO2017083750A1 (en) 2015-11-11 2017-05-18 Intrexon Corporation Compositions and methods for expression of multiple biologically active polypeptides from a single vector for treatment of cardiac conditions and other pathologies
EP3405215B1 (en) 2016-01-19 2022-06-15 The Regents of the University of California Methods for the treatment of danon disease and other disorders of autophagy
CA3011939A1 (en) 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
RS67367B1 (sr) 2016-04-14 2025-11-28 Ferring Ventures Ltd Bioreaktor sa fiksiranim slojem sa pumpom/sistemom creva sa konstantnim protokom
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
US20220323612A1 (en) * 2016-06-13 2022-10-13 Bluebird Bio, Inc. Gene therapy of neuronal ceroid lipofuscinoses
US20190345450A1 (en) 2016-06-17 2019-11-14 Fred Hutchinson Cancer Research Center Strategies to assess and/or produce cell populations with predictive engraftment potential
IL320371A (en) 2016-09-08 2025-06-01 Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P Gene therapy for patients with fanconi anemia
CN110199028A (zh) * 2016-12-06 2019-09-03 蓝鸟生物公司 用于ii型黏多糖贮积症的基因治疗
US20180326022A1 (en) 2017-04-21 2018-11-15 The Trustees Of The University Of Pennsylvania Compositions and methods for improving heart function and treating heart failure
EP3615044A4 (en) 2017-04-27 2021-01-20 Fred Hutchinson Cancer Research Center THERAPEUTIC FORMULAS CONTAINING CD34 + STEM CELLS DERIVED FROM NEGATIVE SELECTION
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
JP2021533831A (ja) 2018-08-16 2021-12-09 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 ウイルスベクターのための生成方法
US20220143215A1 (en) 2019-02-12 2022-05-12 Spacecraft Seven, Llc Gene therapy vectors for treatment of danon disease
KR20220012266A (ko) 2019-05-23 2022-02-03 스페이스크래프트 세븐, 엘엘씨 영아 악성 골화석증을 위한 유전자 요법 벡터
CA3177341A1 (en) 2020-05-20 2021-11-25 Chester Bittencort SACRAMENTO Engineered parkin and uses thereof
WO2022017630A1 (en) 2020-07-23 2022-01-27 Ucl Business Ltd GENE THERAPY VECTOR FOR eEF1A2 AND USES THEREOF
AU2021321412A1 (en) 2020-08-05 2023-04-06 Spacecraft Seven, Llc Adeno-associated viral vector for GLUT1 expression and uses thereof
IL300187A (en) 2020-08-05 2023-03-01 Spacecraft Seven Llc Gene therapy CSRP3 (protein 3 rich in cysteine and glycine)
WO2022032226A1 (en) 2020-08-07 2022-02-10 Spacecraft Seven, Llc Plakophilin-2 (pkp2) gene therapy using aav vector
CN116887868A (zh) 2020-12-07 2023-10-13 太空飞船七有限责任公司 达农病的治疗
CA3240080A1 (en) 2021-12-08 2023-06-15 Christopher Dean HERZOG Junctophilin-2 (jph2) gene therapy using aav vector
CA3240616A1 (en) 2021-12-10 2023-06-15 Christopher Dean HERZOG Troponin c (tnnc1) gene therapy using aav vector
MX2024009685A (es) 2022-02-08 2024-08-19 Spacecraft Seven Llc Vector viral adenoasociado para la expresion de glut1 y usos del mismo.
JP2025512574A (ja) 2022-04-22 2025-04-17 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ベクターを使用するＢ細胞リンパ腫２関連Ａｎｔｈａｎｏｇｅｎｅ ３ （ＢＡＧ３）遺伝子治療

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017139576A1 (en) * 2016-02-12 2017-08-17 Bluebird Bio, Inc. Vcn enhancer compositions and methods of using the same
WO2018106807A1 (en) * 2016-12-06 2018-06-14 Bluebird Bio, Inc. Gene therapy for mucopolysaccharidosis, type i

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Lee, Hyun-Joo, et al. "Retronectin enhances lentivirus-mediated gene delivery into hematopoietic progenitor cells." Biologicals 37.4 (2009): 203-209. (Year: 2009) *

Also Published As

Publication number Publication date
WO2020028430A1 (en) 2020-02-06
BR112021001757A2 (pt) 2021-05-11
AU2019315438A1 (en) 2021-01-28
US20210290685A1 (en) 2021-09-23
IL280465B2 (en) 2025-03-01
MX2021001292A (es) 2021-07-15
CA3106241A1 (en) 2020-02-06
JP7664829B2 (ja) 2025-04-18
JP2025038075A (ja) 2025-03-18
KR20210046004A (ko) 2021-04-27
AU2026203310A1 (en) 2026-06-25
IL316185A (en) 2024-12-01
US12685751B2 (en) 2026-07-21
IL280465A (en) 2021-03-25
JP2021532776A (ja) 2021-12-02
EP3830248A4 (en) 2022-05-04
SG11202100025QA (en) 2021-01-28
CN112888777A (zh) 2021-06-01
EP3830248A1 (en) 2021-06-09
IL280465B1 (en) 2024-11-01

Similar Documents

Publication Publication Date Title
US20210290685A1 (en) Methods for gene modification of hematopoietic cells
US12539316B2 (en) Compositions and methods for stem cell transplant
US20200338131A1 (en) Method
US11155833B2 (en) Method for producing transgenic cell
AU2014338555A1 (en) Method
US20080124308A1 (en) Gene Therapy of Solid Tumours by Means of Retroviral Vectors Pseudotyped With Arenavirus Glycoproteins
JP7766110B2 (ja) レンチウイルスベクターおよびその使用
CN114746546B (zh) 工程化红细胞用于治疗痛风和高尿酸血症
KR20220049568A (ko) 엔젤만 증후군의 치료를 위한 ube3a
RU2824194C2 (ru) Способы генной модификации гемопоэтических клеток

Legal Events

Date Code Title Description
PC1 Assignment before grant (sect. 113)

Owner name: SPACECRAFT SEVEN, LLC

Free format text: FORMER APPLICANT(S): CONSORCIO CENTRO DE INVESTIGACION BIOMEDICA EN RED, M.P.; CENTRO DE INVESTIGACIONES ENERGETICAS, MEDIOAMBIENTALES Y TECNOLOGICAS, O.A., M.P.; ROCKET PHARMACEUTICALS, LTD.; FUNDACION INSTITUTO DE INVESTIGACION SANITARIA FUNDACION JIMENEZ DIAZ

Owner name: CENTRO DE INVESTIGACIONES ENERGETICAS, MEDIOAMBIENTALES Y TECNOLOGICAS, O.A., M.P.

Free format text: FORMER APPLICANT(S): CONSORCIO CENTRO DE INVESTIGACION BIOMEDICA EN RED, M.P.; CENTRO DE INVESTIGACIONES ENERGETICAS, MEDIOAMBIENTALES Y TECNOLOGICAS, O.A., M.P.; ROCKET PHARMACEUTICALS, LTD.; FUNDACION INSTITUTO DE INVESTIGACION SANITARIA FUNDACION JIMENEZ DIAZ

Owner name: CONSORCIO CENTRO DE INVESTIGACION BIOMEDICA EN RED, M.P.

Free format text: FORMER APPLICANT(S): CONSORCIO CENTRO DE INVESTIGACION BIOMEDICA EN RED, M.P.; CENTRO DE INVESTIGACIONES ENERGETICAS, MEDIOAMBIENTALES Y TECNOLOGICAS, O.A., M.P.; ROCKET PHARMACEUTICALS, LTD.; FUNDACION INSTITUTO DE INVESTIGACION SANITARIA FUNDACION JIMENEZ DIAZ

Owner name: FUNDACION INSTITUTO DE INVESTIGACION SANITARIA FUNDACION JIMENEZ DIAZ

Free format text: FORMER APPLICANT(S): CONSORCIO CENTRO DE INVESTIGACION BIOMEDICA EN RED, M.P.; CENTRO DE INVESTIGACIONES ENERGETICAS, MEDIOAMBIENTALES Y TECNOLOGICAS, O.A., M.P.; ROCKET PHARMACEUTICALS, LTD.; FUNDACION INSTITUTO DE INVESTIGACION SANITARIA FUNDACION JIMENEZ DIAZ

FGA Letters patent sealed or granted (standard patent)