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

ENHANCED FETAL HEMOGLOBIN FOR GENETIC CORRECTION OF SICKLE CELL DISEASE

Info

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
Authority
CA
Canada
Prior art keywords
globin
vector
mice
sbg
cells
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
CA2938419A
Other languages
English (en)
French (fr)
Other versions
CA2938419A1 (en
Inventor
Punam Malik
Original Assignee
Cincinnati Children's 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 Children's Hospital Medical Center filed Critical Cincinnati Children's Hospital Medical Center
Publication of CA2938419A1 publication Critical patent/CA2938419A1/en
Application granted granted Critical
Publication of CA2938419C publication Critical patent/CA2938419C/en
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)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Hematology (AREA)
  • Diabetes (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 (enExample)
EP (1) EP3099334B1 (enExample)
JP (1) JP6509884B2 (enExample)
CN (1) CN106456802B (enExample)
AU (2) AU2015210775B2 (enExample)
CA (1) CA2938419C (enExample)
ES (1) ES2747817T3 (enExample)
WO (1) WO2015117027A1 (enExample)

Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10072067B2 (en) 2014-01-30 2018-09-11 Children's Hospital Medical Center Fetal hemoglobin for genetic correction of sickle cell disease
BR112017004349A2 (pt) * 2014-09-04 2017-12-05 Memorial Sloan Kettering Cancer Center terapia com gene de globina para o tratamento de hemoglobinopatias
DK3368052T3 (da) 2015-10-27 2022-03-14 Childrens Hospital Med Ct Anvendelse af mapk-hæmmere for at nedbringe tabet af hæmatopoietiske stamceller under ex vivo-dyrkning og -genmanipulation
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
CA3070242A1 (en) * 2017-07-18 2019-01-24 Calimmune, Inc. Compositions and methods for treating beta-hemoglobinopathies
US11970707B2 (en) 2017-09-18 2024-04-30 Children's Hospital Medical Center Strong insulator and uses thereof in gene delivery
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
WO2020091797A1 (en) * 2018-11-01 2020-05-07 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
US20220243224A1 (en) * 2019-04-15 2022-08-04 Children's Hospital Medical Center Viral vector manufacturing methods
CN114127305A (zh) 2019-05-23 2022-03-01 麻省理工学院 经由逆转录病毒表面展示的配体发现和基因递送
EP3790545A4 (en) 2019-07-17 2021-07-28 Emmaus Medical, Inc. METHODS AND COMPOSITIONS FOR TREATMENT OF SICKLE CLEANEMIA AND THALASSEMIA
CA3212006A1 (en) 2021-02-26 2022-09-01 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
EP4784626A1 (en) 2023-09-25 2026-08-05 Kelonia Therapeutics, Inc. Antigen binding polypeptides
IL327310A (en) 2023-09-25 2026-05-01 Kelonia Therapeutics Inc Cancer treatment vehicles
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
US6172039B1 (en) 1990-04-16 2001-01-09 Apex Bioscience, Inc. Expression of recombinant hemoglobin and hemoglobin variants in yeast
CA2080589A1 (en) * 1990-04-16 1991-10-17 Joseph De Angelo Expression of recombinant hemoglobin in yeast
CA2246005A1 (en) 1998-10-01 2000-04-01 Hsc Research And Development Limited Partnership Hybrid genes for gene therapy in erythroid cells
EP1852511B1 (en) * 1999-12-14 2010-02-17 Novartis AG Bovine immunodeficiency virus (biv) based vectors
JP2004537539A (ja) * 2001-06-29 2004-12-16 スローン − ケッタリング インスティチュート フォー キャンサー リサーチ ヒトグロビン遺伝子をコードするベクターとその異常ヘモグロビン血症の治療におけるその使用
CA2506798C (en) 2002-11-22 2013-01-22 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
WO2010127195A2 (en) 2009-05-01 2010-11-04 Curna, Inc. Antisense oligonucleotides of hemoglobins
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
AU2011377617B2 (en) * 2011-09-23 2018-03-08 Bluebird Bio, Inc. Improved gene therapy methods
US10072067B2 (en) 2014-01-30 2018-09-11 Children's Hospital Medical Center Fetal hemoglobin for genetic correction of sickle cell disease

Also Published As

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

Similar Documents

Publication Publication Date Title
US11345740B2 (en) Fetal hemoglobin for genetic correction of sickle cell disease
US20250034591A1 (en) Self-inactivating lentiviral vector encoding beta- or gamma-globin
US11701435B2 (en) Therapeutic retroviral vectors for gene therapy
CA2451173C (en) Vector encoding human globlin gene and use thereof in treatment of hemoglobinopathies
WO2019213011A1 (en) Compositions and methods for hemoglobin production
US20250034568A1 (en) Constructs comprising tandem microrna-adapted short hairpin rna (shmir) for increasing fetal hemoglobin
HK1230949B (en) An improved fetal hemoglobin for genetic correction of sickle cell disease
HK1230949A1 (en) An improved fetal hemoglobin for genetic correction of 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
BR122023012645B1 (pt) Cassete de expressão, vetor de liberação de gene recombinante, célula, composição farmacêutica e usos dos mesmos para tratar anemia de fanconi
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
HK1082379B (en) Therapeutic retroviral vectors for gene therapy
HK1127549A (en) Therapeutic method for blood coagulation disorder

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20200127

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-4-4-W00-W00-W160 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: INACTIVE: COVER PAGE PUBLISHED

Effective date: 20240701

Free format text: ST27 STATUS EVENT CODE: A-4-4-W00-W00-W160 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: ARCHIVED EVENT: COVER PAGE PUBLISHED

Effective date: 20240701

F11 Ip right granted following substantive examination

Free format text: ST27 STATUS EVENT CODE: A-4-4-F10-F11-X000 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: GRANT BY ISSUANCE

Effective date: 20240702

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-4-4-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT

Effective date: 20240702

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-4-4-W00-W00-W604 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: INACTIVE: GRANT DOWNLOADED

Effective date: 20240703

Free format text: ST27 STATUS EVENT CODE: A-4-4-W00-W00-W604 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: ARCHIVED EVENT: GRANT DOWNLOADED

Effective date: 20240703

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-4-4-W00-W00-W160 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: INACTIVE: COVER PAGE PUBLISHED

Effective date: 20240704

Free format text: ST27 STATUS EVENT CODE: A-4-4-W00-W00-W160 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: ARCHIVED EVENT: COVER PAGE PUBLISHED

Effective date: 20240704

MPN Maintenance fee for patent paid

Free format text: FEE DESCRIPTION TEXT: MF (PATENT, 10TH ANNIV.) - STANDARD

Year of fee payment: 10

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-4-4-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20250124

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-4-4-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT DETERMINED COMPLIANT

Effective date: 20250124

Free format text: ST27 STATUS EVENT CODE: A-4-4-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20250124

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-4-4-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT

Effective date: 20260318