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
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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 Energeticas Medioambientales y Tecnologicas CIEMAT
Centro de Investigacion Biomedica en Red CIBER
Instituto de Investigacion Sanitaria Fundacion Jimenez Diaz
Spacecraft Seven LLC
Original Assignee
Centro de Investigaciones Energeticas Medioambientales y Tecnologicas CIEMAT
Centro de Investigacion Biomedica en Red CIBER
Instituto de Investigacion Sanitaria Fundacion Jimenez Diaz
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 Energeticas Medioambientales y Tecnologicas CIEMAT, Centro de Investigacion Biomedica en Red CIBER, Instituto de Investigacion Sanitaria Fundacion Jimenez Diaz, Spacecraft Seven LLC filed Critical Centro de Investigaciones Energeticas Medioambientales y Tecnologicas CIEMAT
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
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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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)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Hematology (AREA)
  • Organic Chemistry (AREA)
  • Developmental Biology & Embryology (AREA)
  • Immunology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Cell Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Microbiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Plant Pathology (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)

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
AU2026203310 Division 2019-07-30

Publications (2)

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

Family

ID=69232048

Family Applications (1)

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

Country Status (12)

Country Link
US (1) US20210290685A1 (https=)
EP (1) EP3830248A4 (https=)
JP (2) JP7664829B2 (https=)
KR (1) KR20210046004A (https=)
CN (1) CN112888777A (https=)
AU (1) AU2019315438B2 (https=)
BR (1) BR112021001757A2 (https=)
CA (1) CA3106241A1 (https=)
IL (2) IL280465B2 (https=)
MX (1) MX2021001292A (https=)
SG (1) SG11202100025QA (https=)
WO (1) WO2020028430A1 (https=)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HUE055862T2 (hu) 2016-04-20 2021-12-28 Centro De Investig Energeticas Készítmények és eljárások PKLR fokozott génexpressziójára
SG11202002263TA (en) 2017-10-16 2020-04-29 Centro De Investigaciones Energeticas Medioambientales Y Tecnologicas Lentiviral vectors for delivery of pklr to treat pyruvate kinase deficiency
WO2019200167A1 (en) 2018-04-11 2019-10-17 Rocket Pharmaceuticals, Ltd. Compositions and methods for stem cell transplant
MX2020011386A (es) 2018-04-27 2021-01-29 Spacecraft Seven Llc Terapia genica para la degeneracion del snc.
CN115243705A (zh) * 2020-05-27 2022-10-25 苏黎世大学 新颖的转导增强剂及其用途
KR20230043869A (ko) 2020-08-07 2023-03-31 스페이스크래프트 세븐, 엘엘씨 Aav 벡터를 사용한 플라코필린-2(pkp2) 유전자 요법
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
WO2025106604A1 (en) * 2023-11-14 2025-05-22 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 (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3058735A1 (en) 2016-06-13 2017-12-21 Bluebird Bio, Inc. Gene therapy of neuronal ceroid lipofuscinoses
KR20240151726A (ko) * 2016-09-08 2024-10-18 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피. 판코니 빈혈 환자를 위한 유전자 치료
JP2020500562A (ja) * 2016-12-06 2020-01-16 ブルーバード バイオ， インコーポレイテッド ムコ多糖症ｉｉ型のための遺伝子治療

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

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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