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)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hematology (AREA)
  • Diabetes (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
JP6509884B2 (ja) 2014-01-30 2019-05-08 チルドレンズ ホスピタル メディカル センター 鎌状赤血球病の遺伝子的是正のための改善された胎児ヘモグロビン
EP3620521B1 (en) * 2014-09-04 2024-05-29 Memorial Sloan-Kettering Cancer Center Globin gene therapy for treating hemoglobinopathies
EP3368052B1 (en) 2015-10-27 2021-12-08 Children's Hospital Medical Center Use of mapk inhibitors to reduce loss of hematopoietic stem cells during ex vivo culture and 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
CN111164211B (zh) * 2017-07-18 2024-08-02 杰特贝林基因治疗股份有限公司 用于治疗β-血红蛋白病的组合物和方法
CN111093715A (zh) 2017-09-18 2020-05-01 儿童医院医疗中心 强绝缘子和其在基因递送中的用途
CN109722414A (zh) * 2017-10-27 2019-05-07 博雅辑因（北京）生物科技有限公司 一种高效制备成熟红细胞的方法以及用于制备成熟红细胞的培养基
JP7470119B2 (ja) * 2018-08-24 2024-04-17 シーエスエル ベーリング ジーン セラピー インコーポレイテッド 無血清培地におけるベクター産生
US20210380946A1 (en) * 2018-11-01 2021-12-09 Children's Hospital Medical Center Reduced intensity conditioning with melphalan
WO2020209934A1 (en) 2019-04-11 2020-10-15 Massachusetts Institute Of Technology Novel chimeric antigen receptors and libraries
WO2020214676A1 (en) * 2019-04-15 2020-10-22 Children's Hospital Medical Center Viral vector manufacturing methods
JP7658584B2 (ja) 2019-05-23 2025-04-08 マサチューセッツ インスティテュート オブ テクノロジー リガンドの発見およびレトロウイルス表面ディスプレーを介した遺伝子送達
WO2021010937A1 (en) 2019-07-17 2021-01-21 Emmaus Medical, Inc. Methods and compositions for the treatment of sickle cell diseases 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
AU7744491A (en) * 1990-04-16 1991-11-11 Strohtech, Inc. Expression of recombinant hemoglobin in yeast
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
NZ519034A (en) * 1999-12-14 2005-02-25 Novartis Ag Bovine immunodeficiency virus (BIV) based vectors
WO2003002155A1 (en) * 2001-06-29 2003-01-09 Sloan Kettering Institute For Cancer Research Vector encoding human globlin gene and use thereof in treatment of hemoglobinopathies
US20050014166A1 (en) 2002-11-22 2005-01-20 Institut Clayton De La Recherche Compositions and systems for the regulation of genes
WO2004083383A2 (en) 2003-03-14 2004-09-30 Albert Einstein College Of Medicine Of Yeshiva University, A Division 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
EP2424987B1 (en) 2009-05-01 2017-11-15 CuRNA, Inc. 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
KR101931403B1 (ko) * 2011-09-23 2018-12-21 블루버드 바이오, 인코포레이티드. 개선된 유전자 치료 방법
JP6509884B2 (ja) 2014-01-30 2019-05-08 チルドレンズ ホスピタル メディカル センター 鎌状赤血球病の遺伝子的是正のための改善された胎児ヘモグロビン

Also Published As

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

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