ES2747817T3 - Una hemoglobina fetal mejorada para la corrección génica de la anemia falciforme - Google Patents

Una hemoglobina fetal mejorada para la corrección génica de la anemia falciforme Download PDF

Info

Publication number
ES2747817T3
ES2747817T3 ES15744021T ES15744021T ES2747817T3 ES 2747817 T3 ES2747817 T3 ES 2747817T3 ES 15744021 T ES15744021 T ES 15744021T ES 15744021 T ES15744021 T ES 15744021T ES 2747817 T3 ES2747817 T3 ES 2747817T3
Authority
ES
Spain
Prior art keywords
mice
sbg
vector
cells
vectors
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
ES15744021T
Other languages
English (en)
Spanish (es)
Inventor
Punam Malik
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.)
Cincinnati Childrens Hospital Medical Center
Original Assignee
Cincinnati Childrens Hospital Medical Center
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 Cincinnati Childrens Hospital Medical Center filed Critical Cincinnati Childrens Hospital Medical Center
Application granted granted Critical
Publication of ES2747817T3 publication Critical patent/ES2747817T3/es
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

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)
ES15744021T 2014-01-30 2015-01-30 Una hemoglobina fetal mejorada para la corrección génica de la anemia falciforme Active ES2747817T3 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201461933788P 2014-01-30 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 (1)

Publication Number Publication Date
ES2747817T3 true ES2747817T3 (es) 2020-03-11

Family

ID=53757790

Family Applications (1)

Application Number Title Priority Date Filing Date
ES15744021T Active ES2747817T3 (es) 2014-01-30 2015-01-30 Una hemoglobina fetal mejorada para la corrección génica de la anemia falciforme

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
US20170145077A1 (en) 2017-05-25
US11345740B2 (en) 2022-05-31
EP3099334B1 (en) 2019-07-31
CA2938419C (en) 2024-07-02

Similar Documents

Publication Publication Date Title
ES2747817T3 (es) Una hemoglobina fetal mejorada para la corrección génica de la anemia falciforme
US20250034591A1 (en) Self-inactivating lentiviral vector encoding beta- or gamma-globin
US11701435B2 (en) Therapeutic retroviral vectors for gene therapy
ES2885833T3 (es) Composición y métodos para potenciar la expresión génica de PKLR
WO2023081003A1 (en) Constructs comprising tandem microrna-adapted short hairpin rna (shmir) for increasing fetal hemoglobin
EP4490302A2 (en) Vectors combining anti-sickling beta-as3-globin with anti bcel11a sh rna mir to treat beta-hemoglobinopathies
CN102443604B (zh) 一种安全性慢病毒载体及其应用
HK1230949A1 (en) An improved fetal hemoglobin for genetic correction of sickle cell disease
HK1230949B (en) An improved fetal hemoglobin for genetic correction of sickle cell disease
Rivella et al. Globin gene transfer: a paradigm for transgene regulation and vector safety
Klein Advances in viral vector design: Tissue-and cell-type specific promoters can improve the safety and efficacy of lentiviral gene therapy
Persons et al. Prospects for Gene Therapy of Sickle Cell Disease and Thalassemia
Sadelain Globin gene transfer as a potential treatment for the β-thalassaemias and sickle cell disease.
Kloke et al. 437. Overcoming Limitations of Bidirectional Lentiviral and Gammaretroviral Vectors
van Til Lentiviral gene therapy for the treatment of inherited liver disease
Dong Genetic approaches to treat beta-thalassemia
BR122025012421A2 (pt) Cassete de expressão, vetor de liberação de gene recombinante, célula, composições farmacêuticas e usos das ditas composições, de células cd34+ que compreendem um cassete de expressão e de uma população celular transduzida com um vetor lentiviral para tratar anemia de fanconi
Kitowski A lentiviral vector conferring coregulated, erythroid-specific expression of γ-globin and shRNA sequences to BCL11A for the treatment of sickle cell disease
BR122023012645B1 (pt) Cassete de expressão, vetor de liberação de gene recombinante, célula, composição farmacêutica e usos dos mesmos para tratar anemia de fanconi
US20050042204A1 (en) Method to protect transgenes from silencing
Rubin Locus control region activity by 5'HS3 requires a functional interaction with ß-globin gene regulatory elements, identification of effective ß/[gamma]-globin minigenes for gene therapy of the ß-chain hemoglobinopathies