MX2023006565A - Composiciones y metodos para la activacion de bcl11a. - Google Patents

Composiciones y metodos para la activacion de bcl11a.

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Publication number
MX2023006565A
MX2023006565A MX2023006565A MX2023006565A MX2023006565A MX 2023006565 A MX2023006565 A MX 2023006565A MX 2023006565 A MX2023006565 A MX 2023006565A MX 2023006565 A MX2023006565 A MX 2023006565A MX 2023006565 A MX2023006565 A MX 2023006565A
Authority
MX
Mexico
Prior art keywords
systems
methods
subjects
nucleic acids
bcl11a
Prior art date
Application number
MX2023006565A
Other languages
English (en)
Inventor
Benjamin Oakes
Sean Higgins
Sarah Denny
Brett T Staahl
Isabel Colin
Cole Urnes
Maroof Adil
Original Assignee
Scribe Therapeutics 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 Scribe Therapeutics Inc filed Critical Scribe Therapeutics Inc
Publication of MX2023006565A publication Critical patent/MX2023006565A/es

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    • 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
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
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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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/11—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
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    • 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
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
    • A61K35/34—Muscles; Smooth muscle cells; Heart; Cardiac stem cells; Myoblasts; Myocytes; Cardiomyocytes
    • 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
    • A61K35/48—Reproductive organs
    • A61K35/54—Ovaries; Ova; Ovules; Embryos; Foetal cells; Germ cells
    • A61K35/545—Embryonic stem cells; Pluripotent stem cells; Induced pluripotent stem cells; Uncharacterised stem cells
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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
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    • C—CHEMISTRY; METALLURGY
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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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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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
    • C12N15/63—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
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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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • C—CHEMISTRY; METALLURGY
    • 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]

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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)
  • Zoology (AREA)
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  • Cell Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Developmental Biology & Embryology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Immunology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Reproductive Health (AREA)
  • Hematology (AREA)
  • Gynecology & Obstetrics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Mycology (AREA)
  • Cardiology (AREA)
  • Vascular Medicine (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

En la presente descripción se proporcionan sistemas que comprenden polipéptidos de CRISPR tipo V clase 2, ácidos nucleicos guía (NAg) y, opcionalmente, ácidos nucleicos de plantilla donantes útiles en la modificación de un gen BCL11A. Los sistemas también son útiles para la modificación de células en sujetos con una enfermedad relacionada con la hemoglobinopatía. También se proporcionan métodos de tratamiento de sujetos que tienen una enfermedad relacionada con la hemoglobinopatía mediante la administración de los sistemas o ácidos nucleicos que codifican tales sistemas que se dirigen al gen BCL11A en tales sujetos.
MX2023006565A 2020-12-03 2021-12-02 Composiciones y metodos para la activacion de bcl11a. MX2023006565A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202063120885P 2020-12-03 2020-12-03
PCT/US2021/061672 WO2022120094A2 (en) 2020-12-03 2021-12-02 Compositions and methods for the targeting of bcl11a

Publications (1)

Publication Number Publication Date
MX2023006565A true MX2023006565A (es) 2023-08-07

Family

ID=81854906

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2023006565A MX2023006565A (es) 2020-12-03 2021-12-02 Composiciones y metodos para la activacion de bcl11a.

Country Status (15)

Country Link
US (1) US20240026386A1 (es)
EP (1) EP4255502A2 (es)
JP (1) JP2023552379A (es)
KR (1) KR20230129230A (es)
CN (1) CN116801913A (es)
AU (1) AU2021391783A1 (es)
BR (1) BR112023010717A2 (es)
CA (1) CA3200815A1 (es)
CL (1) CL2023001593A1 (es)
CO (1) CO2023007900A2 (es)
GB (1) GB2616795A (es)
IL (1) IL303359A (es)
MX (1) MX2023006565A (es)
PE (1) PE20231177A1 (es)
WO (1) WO2022120094A2 (es)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2022261150A2 (en) 2021-06-09 2022-12-15 Scribe Therapeutics Inc. Particle delivery systems
WO2023235888A2 (en) 2022-06-03 2023-12-07 Scribe Therapeutics Inc. COMPOSITIONS AND METHODS FOR CpG DEPLETION
EP4314267A1 (en) 2022-06-07 2024-02-07 Scribe Therapeutics Inc. Compositions and methods for the targeting of pcsk9
TW202413643A (zh) 2022-06-07 2024-04-01 美商斯奎柏治療公司 用於靶向pcsk9的組合物及方法
WO2023240027A1 (en) 2022-06-07 2023-12-14 Scribe Therapeutics Inc. Particle delivery systems
WO2023240162A1 (en) 2022-06-08 2023-12-14 Scribe Therapeutics Inc. Aav vectors for gene editing

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102351329B1 (ko) * 2016-04-18 2022-01-18 크리스퍼 테라퓨틱스 아게 혈색소병증의 치료를 위한 물질 및 방법
CN110168084A (zh) * 2016-12-05 2019-08-23 爱迪塔斯医药公司 用于内源和来源DNA的单发指导RNA(ogRNA)靶向的系统和方法
EP3833761A1 (en) * 2018-08-07 2021-06-16 The Broad Institute, Inc. Novel cas12b enzymes and systems

Also Published As

Publication number Publication date
AU2021391783A1 (en) 2023-06-22
CA3200815A1 (en) 2022-06-09
WO2022120094A2 (en) 2022-06-09
JP2023552379A (ja) 2023-12-15
GB2616795A (en) 2023-09-20
GB202309871D0 (en) 2023-08-16
CO2023007900A2 (es) 2023-06-30
PE20231177A1 (es) 2023-08-01
US20240026386A1 (en) 2024-01-25
WO2022120094A3 (en) 2022-08-25
CN116801913A (zh) 2023-09-22
IL303359A (en) 2023-08-01
CL2023001593A1 (es) 2023-12-15
EP4255502A2 (en) 2023-10-11
KR20230129230A (ko) 2023-09-07
BR112023010717A2 (pt) 2023-10-03

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