MX2022002461A - Composiciones y métodos para la modificación de cd123. - Google Patents

Composiciones y métodos para la modificación de cd123.

Info

Publication number
MX2022002461A
MX2022002461A MX2022002461A MX2022002461A MX2022002461A MX 2022002461 A MX2022002461 A MX 2022002461A MX 2022002461 A MX2022002461 A MX 2022002461A MX 2022002461 A MX2022002461 A MX 2022002461A MX 2022002461 A MX2022002461 A MX 2022002461A
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MX
Mexico
Prior art keywords
modification
compositions
methods
disclosure
provides
Prior art date
Application number
MX2022002461A
Other languages
English (en)
Inventor
John Lydeard
Bibhu Prasad Mishra
Chong Luo
Michelle Lin
Original Assignee
Vor Biopharma 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 Vor Biopharma Inc filed Critical Vor Biopharma Inc
Publication of MX2022002461A publication Critical patent/MX2022002461A/es

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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
    • C12N15/1138—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 against receptors or cell surface proteins
    • 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
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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
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    • 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
    • C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
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    • 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
    • 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
    • A61K2035/124—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells the cells being hematopoietic, bone marrow derived or blood cells
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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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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/315—Phosphorothioates
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
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    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Hematology (AREA)
  • Immunology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Cell Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

Esta descripción proporciona, por ejemplo, células novedosas que tienen una modificación (por ejemplo, inserción o deleción) en el gen de CD123 endógeno la descripción también proporciona composiciones, por ejemplo, ARNg, que pueden ser utilizados para hacer tal modificación.
MX2022002461A 2019-08-28 2020-08-28 Composiciones y métodos para la modificación de cd123. MX2022002461A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962892888P 2019-08-28 2019-08-28
US202062962135P 2020-01-16 2020-01-16
PCT/US2020/048623 WO2021041977A1 (en) 2019-08-28 2020-08-28 Compositions and methods for cd123 modification

Publications (1)

Publication Number Publication Date
MX2022002461A true MX2022002461A (es) 2022-06-02

Family

ID=72470611

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2022002461A MX2022002461A (es) 2019-08-28 2020-08-28 Composiciones y métodos para la modificación de cd123.

Country Status (10)

Country Link
US (1) US20220333116A1 (es)
EP (1) EP4022064A1 (es)
JP (1) JP2022546505A (es)
KR (1) KR20220047381A (es)
CN (1) CN114787352A (es)
AU (1) AU2020336211A1 (es)
CA (1) CA3151669A1 (es)
IL (1) IL290840A (es)
MX (1) MX2022002461A (es)
WO (1) WO2021041977A1 (es)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3589291A4 (en) 2017-02-28 2020-11-25 Vor Biopharma, Inc. COMPOSITIONS AND METHODS OF INHIBITION OF LINE-SPECIFIC PROTEINS
BR112021003670A2 (pt) 2018-08-28 2021-05-18 Vor Biopharma, Inc. células-tronco hematopoéticas geneticamente modificadas e seus usos
JP2023540277A (ja) * 2020-08-28 2023-09-22 ブイオーアール バイオファーマ インコーポレーテッド Ｃｄ１２３改変のための組成物および方法
WO2023283585A2 (en) 2021-07-06 2023-01-12 Vor Biopharma Inc. Inhibitor oligonucleotides and methods of use thereof
AU2022324093A1 (en) 2021-08-02 2024-02-08 Vor Biopharma Inc. Compositions and methods for gene modification
WO2023196816A1 (en) * 2022-04-04 2023-10-12 Vor Biopharma Inc. Compositions and methods for mediating epitope engineering
WO2024015925A2 (en) 2022-07-13 2024-01-18 Vor Biopharma Inc. Compositions and methods for artificial protospacer adjacent motif (pam) generation
WO2024073751A1 (en) 2022-09-29 2024-04-04 Vor Biopharma Inc. Methods and compositions for gene modification and enrichment

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US10266850B2 (en) 2012-05-25 2019-04-23 The Regents Of The University Of California Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
DK3569619T3 (da) 2014-03-19 2021-06-14 Cellectis Fremgangsmåde til fremstilling af CD123-specifikke CAR T-celler til cancerimmunterapi
EP3129485B2 (en) 2014-04-09 2022-12-21 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating cystic fibrosis
JP7068821B2 (ja) 2014-12-03 2022-05-17 アジレント・テクノロジーズ・インク 化学修飾を有するガイドｒｎａ
CA2981715A1 (en) 2015-04-06 2016-10-13 The Board Of Trustees Of The Leland Stanford Junior University Chemically modified guide rnas for crispr/cas-mediated gene regulation
CN116712535A (zh) 2015-10-16 2023-09-08 纽约市哥伦比亚大学理事会 用于抑制谱系特异性抗原的组合物和方法
WO2017070632A2 (en) 2015-10-23 2017-04-27 President And Fellows Of Harvard College Nucleobase editors and uses thereof
SG11201803701YA (en) * 2015-11-04 2018-06-28 Univ Pennsylvania Methods and compositions for gene editing in hematopoietic stem cells
US10767175B2 (en) 2016-06-08 2020-09-08 Agilent Technologies, Inc. High specificity genome editing using chemically modified guide RNAs
WO2018126176A1 (en) 2016-12-30 2018-07-05 Editas Medicine, Inc. Synthetic guide molecules, compositions and methods relating thereto
EP3589291A4 (en) 2017-02-28 2020-11-25 Vor Biopharma, Inc. COMPOSITIONS AND METHODS OF INHIBITION OF LINE-SPECIFIC PROTEINS
KR20190127797A (ko) 2017-03-10 2019-11-13 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 시토신에서 구아닌으로의 염기 편집제
US11268082B2 (en) 2017-03-23 2022-03-08 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable DNA binding proteins
EP3676372A4 (en) 2017-08-28 2021-06-02 The Trustees of Columbia University in the City of New York CD33 EXON 2 DEFICIENT DONOR STEM CELLS FOR USE WITH AGENTS AGAINST CD33
BR112021003670A2 (pt) * 2018-08-28 2021-05-18 Vor Biopharma, Inc. células-tronco hematopoéticas geneticamente modificadas e seus usos
SG11202108346WA (en) * 2019-02-13 2021-08-30 Beam Therapeutics Inc Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence

Also Published As

Publication number Publication date
WO2021041977A1 (en) 2021-03-04
US20220333116A1 (en) 2022-10-20
IL290840A (en) 2022-04-01
AU2020336211A1 (en) 2022-03-10
KR20220047381A (ko) 2022-04-15
CA3151669A1 (en) 2021-03-04
CN114787352A (zh) 2022-07-22
EP4022064A1 (en) 2022-07-06
JP2022546505A (ja) 2022-11-04

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