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MX2020005849A - Células madre y progenitoras hematopoyéticas humanas cd34+ modificadas con crispr-cas9 y sus usos. - Google Patents

Células madre y progenitoras hematopoyéticas humanas cd34+ modificadas con crispr-cas9 y sus usos.

Info

Publication number
MX2020005849A
MX2020005849A MX2020005849A MX2020005849A MX2020005849A MX 2020005849 A MX2020005849 A MX 2020005849A MX 2020005849 A MX2020005849 A MX 2020005849A MX 2020005849 A MX2020005849 A MX 2020005849A MX 2020005849 A MX2020005849 A MX 2020005849A
Authority
MX
Mexico
Prior art keywords
crispr
progenitor cells
hematopoietic stem
human hematopoietic
cas9 modified
Prior art date
Application number
MX2020005849A
Other languages
English (en)
Inventor
Ante Sven Lundberg
Tony Ho
Ewelina Morawa
Tirtha Chakraborty
Laura Sandler
Brenda Eustace
Jerome Rossert
Robert Kauffman
Original Assignee
Vertex Pharma
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 Vertex Pharma filed Critical Vertex Pharma
Publication of MX2020005849A publication Critical patent/MX2020005849A/es

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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
    • A61K35/28—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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/21—Esters, e.g. nitroglycerine, selenocyanates
    • A61K31/255—Esters, e.g. nitroglycerine, selenocyanates of sulfoxy acids or sulfur analogues thereof
    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445—Non condensed piperidines, e.g. piperocaine
    • A61K31/4523—Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems
    • A61K31/4535—Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems containing a heterocyclic ring having sulfur as a ring hetero atom, e.g. pizotifen
    • 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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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • 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
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
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    • C12N5/0602—Vertebrate cells
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    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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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 [CRISPR]
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    • C12N2310/00—Structure or type of the nucleic acid
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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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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
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  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
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  • Wood Science & Technology (AREA)
  • Immunology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Developmental Biology & Embryology (AREA)
  • Cell Biology (AREA)
  • Hematology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Emergency Medicine (AREA)
  • Diabetes (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

En algunas modalidades, en la presente se proporcionan métodos y composiciones para el tratamiento de sujetos con talasemia-ß y sujetos con enfermedad de células falciformes grave mediante el uso de células madre y progenitoras hematopoyéticas humanas CD34+ modificadas con CRISPR-Cas9 autólogas.
MX2020005849A 2017-12-05 2018-12-05 Células madre y progenitoras hematopoyéticas humanas cd34+ modificadas con crispr-cas9 y sus usos. MX2020005849A (es)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201762594689P 2017-12-05 2017-12-05
US201862664023P 2018-04-27 2018-04-27
US201862671770P 2018-05-15 2018-05-15
US201862734431P 2018-09-21 2018-09-21
US201862734543P 2018-09-21 2018-09-21
PCT/US2018/063973 WO2019113149A1 (en) 2017-12-05 2018-12-05 Crispr-cas9 modified cd34+ human hematopoietic stem and progenitor cells and uses thereof

Publications (1)

Publication Number Publication Date
MX2020005849A true MX2020005849A (es) 2020-11-06

Family

ID=64902426

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2020005849A MX2020005849A (es) 2017-12-05 2018-12-05 Células madre y progenitoras hematopoyéticas humanas cd34+ modificadas con crispr-cas9 y sus usos.

Country Status (13)

Country Link
US (2) US12161674B2 (es)
EP (1) EP3720473A1 (es)
JP (1) JP2021505587A (es)
KR (2) KR20200106159A (es)
CN (1) CN111629747A (es)
AU (1) AU2018378479B2 (es)
BR (1) BR112020011255A2 (es)
CA (1) CA3084955A1 (es)
IL (1) IL275037A (es)
MA (1) MA51107A (es)
MX (1) MX2020005849A (es)
SG (1) SG11202005147WA (es)
WO (1) WO2019113149A1 (es)

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US11866726B2 (en) 2017-07-14 2024-01-09 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
WO2019213273A1 (en) 2018-05-01 2019-11-07 The Children's Medical Center Corporation Enhanced bcl11a rnp / crispr delivery & editing using a 3xnls-cas9
ES3023563T3 (en) 2018-10-16 2025-06-02 Blueallele Corp Methods for targeted insertion of dna in genes
EP4017980A4 (en) * 2019-09-30 2024-08-07 The Children's Medical Center Corporation BCL11A GUIDE AND BASIC EDITOR DISTRIBUTION
GB202017555D0 (en) * 2020-11-06 2020-12-23 Harbour Antibodies Bv Antibody-conjugated nanoparticles
JP2023552806A (ja) 2020-12-08 2023-12-19 フランク オヴォカイツ トッド 幹細胞の増産のための方法およびシステム
KR20230126707A (ko) * 2020-12-31 2023-08-30 보르 바이오파마 인크. Cd34 유전자 변형을 위한 조성물 및 방법
US20240245725A1 (en) * 2021-05-13 2024-07-25 The Children's Medical Center Corporation Methods for stratifying subjects for fetal hemoglobin reinduction
US20250381304A1 (en) * 2022-06-21 2025-12-18 Crispr Therapeutics Ag Methods and compositions for in vivo editing of stem cells
WO2024077157A2 (en) * 2022-10-05 2024-04-11 Garuda Therapeutics, Inc. Myeloid lineages derived from pluripotent cells
WO2024211616A1 (en) * 2023-04-04 2024-10-10 Ovokaitys Todd Frank Methods and systems for improved therapies of genetic diseases using photo-activated allogenic stem cells
CN117338902B (zh) * 2023-10-25 2024-07-09 南京医科大学 一种用于治疗卵子成熟障碍的复方制剂
JPWO2025094917A1 (es) * 2023-10-30 2025-05-08
WO2025250969A1 (en) 2024-05-31 2025-12-04 Vertex Pharmaceuticals Incorporated Anti-cd74 antibodies, conjugates and uses thereof

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