SG11202008956XA - Systems and methods for the treatment of hemoglobinopathies - Google Patents

Systems and methods for the treatment of hemoglobinopathies

Info

Publication number
SG11202008956XA
SG11202008956XA SG11202008956XA SG11202008956XA SG11202008956XA SG 11202008956X A SG11202008956X A SG 11202008956XA SG 11202008956X A SG11202008956X A SG 11202008956XA SG 11202008956X A SG11202008956X A SG 11202008956XA SG 11202008956X A SG11202008956X A SG 11202008956XA
Authority
SG
Singapore
Prior art keywords
hemoglobinopathies
systems
treatment
methods
Prior art date
Application number
SG11202008956XA
Inventor
Jennifer Leah Gori
De Lamothe-Dreuzy Edouard Aupepin
Jack Heath
John Anthony Zuris
Kaihsin Chang
Original Assignee
Editas Medicine Inc
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 Editas Medicine Inc filed Critical Editas Medicine Inc
Publication of SG11202008956XA publication Critical patent/SG11202008956XA/en

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    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
    • A61K35/14—Blood; Artificial blood
    • A61K35/17—Lymphocytes; B-cells; T-cells; Natural killer cells; Interferon-activated or cytokine-activated lymphocytes
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0002—Galenical forms characterised by the drug release technique; Application systems commanded by energy
    • A61K9/0009—Galenical forms characterised by the drug release technique; Application systems commanded by energy involving or responsive to electricity, magnetism or acoustic waves; Galenical aspects of sonophoresis, iontophoresis, electroporation or electroosmosis
    • 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
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    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases RNAses, DNAses
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/313—Phosphorodithioates
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Hematology (AREA)
  • Virology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Diabetes (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
SG11202008956XA 2018-03-14 2019-03-14 Systems and methods for the treatment of hemoglobinopathies SG11202008956XA (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201862643168P 2018-03-14 2018-03-14
US201862767488P 2018-11-14 2018-11-14
US201862773073P 2018-11-29 2018-11-29
PCT/US2019/022374 WO2019178426A1 (en) 2018-03-14 2019-03-14 Systems and methods for the treatment of hemoglobinopathies

Publications (1)

Publication Number Publication Date
SG11202008956XA true SG11202008956XA (en) 2020-10-29

Family

ID=65952168

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202008956XA SG11202008956XA (en) 2018-03-14 2019-03-14 Systems and methods for the treatment of hemoglobinopathies

Country Status (12)

Country Link
US (1) US20210254061A1 (en)
EP (1) EP3765614A1 (en)
JP (1) JP2021518102A (en)
KR (1) KR20200132924A (en)
CN (1) CN112020558A (en)
AU (1) AU2019236209A1 (en)
BR (1) BR112020018364A2 (en)
CA (1) CA3093702A1 (en)
IL (1) IL277234A (en)
MX (1) MX2020009487A (en)
SG (1) SG11202008956XA (en)
WO (1) WO2019178426A1 (en)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3981876A1 (en) 2014-03-26 2022-04-13 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
CA3009727A1 (en) * 2015-12-28 2017-07-06 Novartis Ag Compositions and methods for the treatment of hemoglobinopathies
WO2018170184A1 (en) 2017-03-14 2018-09-20 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
EP3622070A2 (en) 2017-05-10 2020-03-18 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
EP3635120A1 (en) * 2017-06-02 2020-04-15 Institut National de la Santé et de la Recherche Médicale (INSERM) Recombinant lentiviral vector for stem cell-based gene therapy of sickle cell disorder
EP3652312A1 (en) 2017-07-14 2020-05-20 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
JP2022503535A (en) * 2018-08-08 2022-01-12 インテグレイテッド ディエヌエイ テクノロジイズ，インコーポレイテッド A novel mutation that enhances the DNA cleavage activity of CPF1 of the genus Lactococcus
US20220047637A1 (en) * 2018-11-29 2022-02-17 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
WO2021119040A1 (en) * 2019-12-08 2021-06-17 Editas Medicine, Inc. Modified cells and methods for the treatment of hemoglobinopathies
CA3170326A1 (en) * 2020-02-13 2021-08-19 Beam Therapeutics Inc. Compositions and methods for engraftment of base edited cells
JP2023526007A (en) * 2020-05-13 2023-06-20 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル A base-editing approach for the treatment of β-hemoglobin disorders
JP2024506645A (en) * 2021-02-11 2024-02-14 コーニンクレッカ ネザーランド アカデミー ヴァン ウェテンシャッペン Cure diseases by editing transcriptional regulatory genes
EP4047095A1 (en) * 2021-02-22 2022-08-24 Eberhard Karls Universität Tübingen Medizinische Fakultät Method and composition for a targeted gene knockout
AU2022325054A1 (en) * 2021-08-02 2024-02-01 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
CN115851706A (en) * 2021-09-24 2023-03-28 华东师范大学 Base editing system and application thereof
KR20230132341A (en) 2022-03-07 2023-09-15 광주과학기술원 Cpf1 mutant protein for rapid DNA diagnosis

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK3066201T3 (en) 2013-11-07 2018-06-06 Editas Medicine Inc CRISPR-RELATED PROCEDURES AND COMPOSITIONS WITH LEADING GRADES
EP3553176A1 (en) 2014-03-10 2019-10-16 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)
WO2015148860A1 (en) 2014-03-26 2015-10-01 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating beta-thalassemia
WO2016073990A2 (en) 2014-11-07 2016-05-12 Editas Medicine, Inc. Methods for improving crispr/cas-mediated genome-editing
CN107250148B (en) * 2014-12-03 2021-04-16 安捷伦科技有限公司 Guide RNAs with chemical modifications
EP3230452A1 (en) * 2014-12-12 2017-10-18 The Broad Institute Inc. Dead guides for crispr transcription factors
AU2016261358B2 (en) 2015-05-11 2021-09-16 Editas Medicine, Inc. Optimized CRISPR/Cas9 systems and methods for gene editing in stem cells
EP3371306B8 (en) * 2015-11-04 2023-02-22 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
US20190233814A1 (en) * 2015-12-18 2019-08-01 The Broad Institute, Inc. Novel crispr enzymes and systems
EP3430142A1 (en) * 2016-03-14 2019-01-23 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating beta hemoglobinopathies
KR102424476B1 (en) 2016-04-19 2022-07-25 더 브로드 인스티튜트, 인코퍼레이티드 Novel CRISPR Enzymes and Systems
WO2017191503A1 (en) * 2016-05-05 2017-11-09 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
WO2018017754A1 (en) * 2016-07-19 2018-01-25 Duke University Therapeutic applications of cpf1-based genome editing
EP3692154A1 (en) * 2017-10-02 2020-08-12 Genedit Inc. Modified cpf1 guide rna
CA3085338A1 (en) * 2017-12-11 2019-06-20 Editas Medicine, Inc. Cpf1-related methods and compositions for gene editing
US20190241911A1 (en) * 2018-02-06 2019-08-08 Ohio State Innovation Foundation Engineered guide rna and uses thereof

Also Published As

Publication number Publication date
IL277234A (en) 2020-10-29
AU2019236209A1 (en) 2020-10-01
KR20200132924A (en) 2020-11-25
MX2020009487A (en) 2020-12-10
CA3093702A1 (en) 2019-09-19
JP2021518102A (en) 2021-08-02
EP3765614A1 (en) 2021-01-20
WO2019178426A1 (en) 2019-09-19
US20210254061A1 (en) 2021-08-19
CN112020558A (en) 2020-12-01
BR112020018364A2 (en) 2020-12-29

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