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MX2021008490A - Composiciones y metodos para la inhibicion de antigenos con especificidad de linaje. - Google Patents

Composiciones y metodos para la inhibicion de antigenos con especificidad de linaje.

Info

Publication number
MX2021008490A
MX2021008490A MX2021008490A MX2021008490A MX2021008490A MX 2021008490 A MX2021008490 A MX 2021008490A MX 2021008490 A MX2021008490 A MX 2021008490A MX 2021008490 A MX2021008490 A MX 2021008490A MX 2021008490 A MX2021008490 A MX 2021008490A
Authority
MX
Mexico
Prior art keywords
methods
inhibition
compositions
specific antigens
lineage specific
Prior art date
Application number
MX2021008490A
Other languages
English (en)
Inventor
Siddhartha Mukherjee
Florence Borot
Abdullah Mahmood Ali
Original Assignee
Univ Columbia
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 Univ Columbia filed Critical Univ Columbia
Publication of MX2021008490A publication Critical patent/MX2021008490A/es

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    • 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
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • 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
    • 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
    • C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A61P35/02—Antineoplastic agents specific for leukemia
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70596—Molecules with a "CD"-designation not provided for elsewhere
    • 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
    • C12N15/10—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
    • C12N15/11—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
    • 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
    • C12N15/11—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
    • 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
    • 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 [RNase]; Deoxyribonucleases [DNase]
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • 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
    • C12N2510/00—Genetically modified cells

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Hematology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Physics & Mathematics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Oncology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

En la presente, se describen métodos de administración de un agente dirigido a un antígeno de la superficie celular con especificidad de linaje, p. ej., el CD33, y una población de células hematopoyéticas con deficiencia del antígeno de la superficie celular con especificidad de linaje, p. ej., el CD33, para la inmunoterapia de neoplasias malignas hematológicas. También se proporcionan células que comprenden mutaciones en el CD33, al igual que ARNg dirigido al CD33.
MX2021008490A 2019-01-16 2020-01-16 Composiciones y metodos para la inhibicion de antigenos con especificidad de linaje. MX2021008490A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962793210P 2019-01-16 2019-01-16
US201962852573P 2019-05-24 2019-05-24
PCT/US2020/013887 WO2020150478A1 (en) 2019-01-16 2020-01-16 Compositions and methods for inhibition of lineage specific antigens

Publications (1)

Publication Number Publication Date
MX2021008490A true MX2021008490A (es) 2021-09-28

Family

ID=71613437

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2021008490A MX2021008490A (es) 2019-01-16 2020-01-16 Composiciones y metodos para la inhibicion de antigenos con especificidad de linaje.

Country Status (12)

Country Link
US (1) US20210260130A1 (es)
EP (1) EP3911338A4 (es)
JP (1) JP7784707B2 (es)
KR (1) KR20210129048A (es)
CN (1) CN113474452A (es)
AU (1) AU2020209218A1 (es)
BR (1) BR112021014010A2 (es)
CA (1) CA3126677A1 (es)
IL (1) IL284853A (es)
MX (1) MX2021008490A (es)
SG (1) SG11202107639UA (es)
WO (1) WO2020150478A1 (es)

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JP7386082B2 (ja) 2017-02-28 2023-11-24 ブイオーアール バイオファーマ インコーポレーテッド 系統特異的タンパク質の阻害のための組成物および方法
EP4512890A3 (en) 2018-08-28 2025-04-30 Vor Biopharma, Inc. Genetically engineered hermatopoietic stem cells and uses thereof
EP3973057A1 (en) * 2019-05-23 2022-03-30 Vor Biopharma, Inc. Compositions and methods for cd33 modification
WO2021041971A1 (en) * 2019-08-28 2021-03-04 Vor Biopharma, Inc. Compositions and methods for cll1 modification
JP2023540276A (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 Ｃｌｌ１改変のための組成物および方法
EP4236970A1 (en) * 2020-10-27 2023-09-06 Vor Biopharma Inc. Compositions and methods for treating hematopoietic malignancy
CN120249215A (zh) * 2021-04-27 2025-07-04 上海驯鹿生物技术有限公司 一种基因编辑的造血干细胞及其与car-t细胞的联合应用
US20240384304A1 (en) 2021-07-06 2024-11-21 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
EP4555091A2 (en) 2022-07-13 2025-05-21 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
WO2025030010A1 (en) 2023-08-01 2025-02-06 Vor Biopharma Inc. Compositions comprising genetically engineered hematopoietic stem cells and methods of use thereof
WO2026017800A1 (en) * 2024-07-19 2026-01-22 Medical University – Plovdiv Panels and reagent kits for minimal residual disease measurement in pediatric b-cell precursor acute lymphoblastic leukemia via immunophenotyping

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN106795221B (zh) * 2014-04-03 2022-06-07 塞勒克提斯公司 用于癌症免疫治疗的cd33特异性嵌合抗原受体
US10293058B2 (en) * 2015-04-22 2019-05-21 Curevac Ag RNA containing composition for treatment of tumor diseases
US11390884B2 (en) * 2015-05-11 2022-07-19 Editas Medicine, Inc. Optimized CRISPR/cas9 systems and methods for gene editing in stem cells
CN108290939B (zh) * 2015-10-16 2023-01-13 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
US20210260219A1 (en) * 2015-12-23 2021-08-26 Crispr Therapeutics Ag Materials and methods for treatment of amyotrophic lateral sclerosis and/or frontal temporal lobular degeneration
AU2018224387B2 (en) * 2017-02-22 2024-08-08 Crispr Therapeutics Ag Compositions and methods for gene editing
US11718659B2 (en) * 2017-08-28 2023-08-08 The Trustees Of Columbia University In The City Of New York CD33 exon 2 deficient donor stem cells for use with CD33 targeting agents

Also Published As

Publication number Publication date
WO2020150478A1 (en) 2020-07-23
JP2022517618A (ja) 2022-03-09
JP7784707B2 (ja) 2025-12-12
EP3911338A4 (en) 2023-06-07
KR20210129048A (ko) 2021-10-27
EP3911338A1 (en) 2021-11-24
BR112021014010A2 (pt) 2021-09-21
IL284853A (en) 2021-08-31
SG11202107639UA (en) 2021-08-30
US20210260130A1 (en) 2021-08-26
AU2020209218A1 (en) 2021-07-29
CN113474452A (zh) 2021-10-01
CA3126677A1 (en) 2020-07-23

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