CA2938419C - ENHANCED FETAL HEMOGLOBIN FOR GENETIC CORRECTION OF SICKLE CELL DISEASE - Google Patents

ENHANCED FETAL HEMOGLOBIN FOR GENETIC CORRECTION OF SICKLE CELL DISEASE

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Publication number
CA2938419C
CA2938419C CA2938419A CA2938419A CA2938419C CA 2938419 C CA2938419 C CA 2938419C CA 2938419 A CA2938419 A CA 2938419A CA 2938419 A CA2938419 A CA 2938419A CA 2938419 C CA2938419 C CA 2938419C
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Prior art keywords
globin
vector
mice
sbg
cells
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CA2938419A
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English (en)
French (fr)
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CA2938419A1 (en
Inventor
Punam Malik
Original Assignee
Cincinnati Childrens Hospital Medical Center
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Publication of CA2938419A1 publication Critical patent/CA2938419A1/en
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Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/795—Porphyrin- or corrin-ring-containing peptides
    • C07K14/805—Haemoglobins; Myoglobins
    • 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
    • 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
    • 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
    • C12N2740/10041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/10043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/40—Vector systems having a special element relevant for transcription being an insulator
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (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)
CA2938419A 2014-01-30 2015-01-30 ENHANCED FETAL HEMOGLOBIN FOR GENETIC CORRECTION OF SICKLE CELL DISEASE Active CA2938419C (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201461933788P 2014-01-30 2014-01-30
US61/933,788 2014-01-30
PCT/US2015/013960 WO2015117027A1 (en) 2014-01-30 2015-01-30 An improved fetal hemoglobin for genetic correction of sickle cell disease

Publications (2)

Publication Number Publication Date
CA2938419A1 CA2938419A1 (en) 2015-08-06
CA2938419C true CA2938419C (en) 2024-07-02

Family

ID=53757790

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2938419A Active CA2938419C (en) 2014-01-30 2015-01-30 ENHANCED FETAL HEMOGLOBIN FOR GENETIC CORRECTION OF SICKLE CELL DISEASE

Country Status (8)

Country Link
US (2) US10072067B2 (https=)
EP (1) EP3099334B1 (https=)
JP (1) JP6509884B2 (https=)
CN (1) CN106456802B (https=)
AU (2) AU2015210775B2 (https=)
CA (1) CA2938419C (https=)
ES (1) ES2747817T3 (https=)
WO (1) WO2015117027A1 (https=)

Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN106456802B (zh) 2014-01-30 2019-10-18 儿童医院医疗中心 一种改进的用于镰状细胞疾病基因校正的胎儿血红蛋白
CA2960209C (en) * 2014-09-04 2023-08-29 Memorial Sloan-Kettering Cancer Center Globin gene therapy for treating hemoglobinopathies
WO2017075274A1 (en) 2015-10-27 2017-05-04 Children's Hospital Medical Center Use of mapk inhibitors to reduce loss of hematopoietic stem cells during ex vivo culture and/or genetic manipulation
CN106497976A (zh) * 2016-10-31 2017-03-15 广东铱科基因科技有限公司 一种用于矫正重症β‑地中海贫血患者自体造血干细胞的HBB基因试剂盒
WO2019018383A1 (en) 2017-07-18 2019-01-24 Calimmune, Inc. COMPOSITIONS AND METHODS FOR TREATING BETA-HEMOGLOBINOPATHIES
EP3684421A4 (en) 2017-09-18 2021-08-04 Children's Hospital Medical Center STRONG INSULATOR AND ITS USES IN GENE ADMINISTRATION
CN109722415B (zh) * 2017-10-27 2021-01-26 博雅辑因（北京）生物科技有限公司 一种造血干细胞的培养组合物、培养基以及造血干细胞的培养方法
KR102944997B1 (ko) 2018-08-24 2026-03-30 씨에스엘 베링 진 테라피, 인크. 무혈청 배지에서 벡터 제조
EP3873502A4 (en) * 2018-11-01 2022-06-29 Children's Hospital Medical Center Reduced intensity conditioning with melphalan
US20200325241A1 (en) 2019-04-11 2020-10-15 Massachusetts Institute Of Technology Novel chimeric antigen receptors and libraries
US20220243224A1 (en) * 2019-04-15 2022-08-04 Children's Hospital Medical Center Viral vector manufacturing methods
US12061187B2 (en) 2019-05-23 2024-08-13 Massachusetts Institute Of Technology Ligand discovery and gene delivery via retroviral surface display
EP3790545A4 (en) 2019-07-17 2021-07-28 Emmaus Medical, Inc. METHODS AND COMPOSITIONS FOR TREATMENT OF SICKLE CLEANEMIA AND THALASSEMIA
AU2022227021A1 (en) 2021-02-26 2023-09-21 Kelonia Therapeutics, Inc. Lymphocyte targeted lentiviral vectors
WO2024050450A1 (en) 2022-08-31 2024-03-07 Gigamune, Inc. Engineered enveloped vectors and methods of use thereof
AU2024353718A1 (en) 2023-09-25 2026-04-09 Kelonia Therapeutics, Inc. Compositions for treating cancer
AU2024353243A1 (en) 2023-09-25 2026-04-09 Kelonia Therapeutics, Inc. Antigen binding polypeptides
WO2025151744A1 (en) * 2024-01-10 2025-07-17 University Of Tennessee Research Foundation Improved globin and other lentiviral vectors for gene therapy

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE222600T1 (de) * 1990-04-16 2002-09-15 Apex Bioscience Inc Expression von rekombinantem hämoglobin in hefe
US6172039B1 (en) * 1990-04-16 2001-01-09 Apex Bioscience, Inc. Expression of recombinant hemoglobin and hemoglobin variants in yeast
CA2246005A1 (en) 1998-10-01 2000-04-01 Hsc Research And Development Limited Partnership Hybrid genes for gene therapy in erythroid cells
DE60033180T2 (de) * 1999-12-14 2007-10-25 Novartis Ag Auf dem rinderimmunschwächevirus (biv) basierte vektoren
EP1409025B1 (en) * 2001-06-29 2011-11-30 Sloan Kettering Institute For Cancer Research Vector encoding human globlin gene and use thereof in treatment of hemoglobinopathies
CN1759182A (zh) 2002-11-22 2006-04-12 克雷顿研究院 调节基因的组合物和系统
US20070066548A1 (en) 2003-03-14 2007-03-22 Albert Einstein College Of Medicine Of Yeshiva University Globin variant gene methods and compositions
WO2005030999A1 (en) 2003-09-25 2005-04-07 Dana-Farber Cancer Institute, Inc Methods to detect lineage-specific cells
US7276340B1 (en) 2004-09-22 2007-10-02 University Of Central Florida Research Foundation, Inc. Age determination from biological stains using messenger RNA profiling analysis
US20090156534A1 (en) * 2007-09-13 2009-06-18 Memorial Sloan-Kettering Cancer Center Globin lentiviral vectors for treatment of disease
CA2760589C (en) 2009-05-01 2019-08-20 Joseph Collard Treatment of hemoglobin (hbf/hbg) related diseases by inhibition of natural antisense transcript to hbf/hbg
US20110294114A1 (en) 2009-12-04 2011-12-01 Cincinnati Children's Hospital Medical Center Optimization of determinants for successful genetic correction of diseases, mediated by hematopoietic stem cells
JP6066356B2 (ja) * 2011-09-23 2017-01-25 ブルーバード バイオ， インコーポレイテッド 改善された遺伝子治療方法
CN106456802B (zh) 2014-01-30 2019-10-18 儿童医院医疗中心 一种改进的用于镰状细胞疾病基因校正的胎儿血红蛋白

Also Published As

Publication number Publication date
EP3099334A1 (en) 2016-12-07
AU2020244473B2 (en) 2024-03-07
EP3099334A4 (en) 2017-06-21
AU2020244473A1 (en) 2020-10-29
AU2015210775A1 (en) 2016-08-18
AU2015210775B2 (en) 2020-07-02
WO2015117027A1 (en) 2015-08-06
US20180346552A1 (en) 2018-12-06
CN106456802B (zh) 2019-10-18
CN106456802A (zh) 2017-02-22
JP6509884B2 (ja) 2019-05-08
JP2017505626A (ja) 2017-02-23
CA2938419A1 (en) 2015-08-06
US10072067B2 (en) 2018-09-11
ES2747817T3 (es) 2020-03-11
US20170145077A1 (en) 2017-05-25
US11345740B2 (en) 2022-05-31
EP3099334B1 (en) 2019-07-31

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