CL2018001198A1 - Métodos y composiciones para edición génica en hemocitoblastos - Google Patents

Métodos y composiciones para edición génica en hemocitoblastos

Info

Publication number
CL2018001198A1
CL2018001198A1 CL2018001198A CL2018001198A CL2018001198A1 CL 2018001198 A1 CL2018001198 A1 CL 2018001198A1 CL 2018001198 A CL2018001198 A CL 2018001198A CL 2018001198 A CL2018001198 A CL 2018001198A CL 2018001198 A1 CL2018001198 A1 CL 2018001198A1
Authority
CL
Chile
Prior art keywords
compositions
modified
methods
hemocytoblasts
hemocytoblast
Prior art date
Application number
CL2018001198A
Other languages
English (en)
Spanish (es)
Inventor
Saar Gill
Miriam Kim
Original Assignee
Univ Pennsylvania
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 Pennsylvania filed Critical Univ Pennsylvania
Publication of CL2018001198A1 publication Critical patent/CL2018001198A1/es

Links

Classifications

    • 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
    • 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
    • A61K35/15—Cells of the myeloid line, e.g. granulocytes, basophils, eosinophils, neutrophils, leucocytes, monocytes, macrophages or mast cells; Myeloid precursor cells; Antigen-presenting cells, e.g. dendritic 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
    • 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
    • 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
    • A61K40/00—Cellular immunotherapy
    • A61K40/10—Cellular immunotherapy characterised by the cell type used
    • A61K40/11—T-cells, e.g. tumour infiltrating lymphocytes [TIL] or regulatory T [Treg] cells; Lymphokine-activated killer [LAK] cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/30—Cellular immunotherapy characterised by the recombinant expression of specific molecules in the cells of the immune system
    • A61K40/31—Chimeric antigen receptors [CAR]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/40—Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
    • A61K40/41—Vertebrate antigens
    • A61K40/42—Cancer antigens
    • A61K40/4202—Receptors, cell surface antigens or cell surface determinants
    • A61K40/421—Immunoglobulin superfamily
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/05—Immunological preparations stimulating the reticulo-endothelial system, e.g. against cancer
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • 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
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • 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
    • C12N15/87—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
    • 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]

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Cell Biology (AREA)
  • Hematology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Virology (AREA)
  • Oncology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Mycology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
CL2018001198A 2015-11-04 2018-05-03 Métodos y composiciones para edición génica en hemocitoblastos CL2018001198A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201562250561P 2015-11-04 2015-11-04

Publications (1)

Publication Number Publication Date
CL2018001198A1 true CL2018001198A1 (es) 2018-09-21

Family

ID=58662736

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2018001198A CL2018001198A1 (es) 2015-11-04 2018-05-03 Métodos y composiciones para edición génica en hemocitoblastos

Country Status (16)

Country Link
US (3) US10548922B2 (enExample)
EP (1) EP3370741B1 (enExample)
JP (3) JP6866385B2 (enExample)
KR (1) KR20180073679A (enExample)
CN (1) CN108463227A (enExample)
AU (2) AU2016349280B2 (enExample)
BR (1) BR112018009098A8 (enExample)
CA (1) CA3004053A1 (enExample)
CL (1) CL2018001198A1 (enExample)
EA (1) EA201891092A1 (enExample)
IL (1) IL259084A (enExample)
MX (1) MX2018005612A (enExample)
MY (1) MY185961A (enExample)
PH (1) PH12018500965A1 (enExample)
SG (2) SG10202107602XA (enExample)
WO (1) WO2017079400A1 (enExample)

Families Citing this family (60)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013066438A2 (en) 2011-07-22 2013-05-10 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
US9163284B2 (en) 2013-08-09 2015-10-20 President And Fellows Of Harvard College Methods for identifying a target site of a Cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US20150166984A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting alpha-antitrypsin point mutations
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
US10201606B2 (en) 2014-11-26 2019-02-12 Miltenyi Biotec Gmbh Combination immunotherapy of antigen-recognizing receptors and hematopoietic cells for the treatment of diseases
IL258491B2 (en) 2015-10-16 2023-09-01 Univ Columbia Preparations and methods for inhibiting antigens of a particular lineage
WO2017070632A2 (en) 2015-10-23 2017-04-27 President And Fellows Of Harvard College Nucleobase editors and uses thereof
EP3370741B1 (en) * 2015-11-04 2025-10-08 The Trustees of the University of Pennsylvania Methods and compositions for gene editing in hematopoietic stem cells
WO2017143266A1 (en) * 2016-02-19 2017-08-24 The Regents Of The University Of California Short hairpin rna (shrna734) and use of same to positively select and eliminate genetically modified cells
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
AU2017308889B2 (en) 2016-08-09 2023-11-09 President And Fellows Of Harvard College Programmable Cas9-recombinase fusion proteins and uses thereof
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
KR20240007715A (ko) 2016-10-14 2024-01-16 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
US20190365806A1 (en) * 2016-11-02 2019-12-05 Universität Basel Immunologically discernible cell surface variants for use in cell therapy
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
KR102709603B1 (ko) * 2017-02-28 2024-09-27 보르 바이오파마 인크. 계통 특이적 단백질의 억제를 위한 조성물 및 방법
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
KR20190123328A (ko) 2017-03-09 2019-10-31 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 암 백신
CN110914310A (zh) 2017-03-10 2020-03-24 哈佛大学的校长及成员们 胞嘧啶至鸟嘌呤碱基编辑器
IL306092A (en) 2017-03-23 2023-11-01 Harvard College Nucleic base editors that include nucleic acid programmable DNA binding proteins
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
EP3658573A1 (en) 2017-07-28 2020-06-03 President and Fellows of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (pace)
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
CA3082251A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
CN112654710A (zh) 2018-05-16 2021-04-13 辛瑟高公司 用于指导rna设计和使用的方法和系统
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
IL260445B (en) * 2018-07-05 2019-08-29 Lempo Therapeutics Ltd Methods and compositions for modulating myeloperoxidase (mpo) expression
EP3820495A4 (en) 2018-07-09 2022-07-20 The Broad Institute Inc. RNA-PROGRAMMABLE EPIGENETIC RNA MODIFIERS AND THEIR USES
CA3110837A1 (en) 2018-08-28 2020-03-05 Vor Biopharma Inc. Genetically engineered hematopoietic stem cells and uses thereof
AU2019345052B2 (en) 2018-09-19 2025-09-04 FUJIFILM Cellular Dynamics, Inc. Protein L for activation and expansion of chimeric antigen receptor-modified immune cells
US12331320B2 (en) 2018-10-10 2025-06-17 The Research Foundation For The State University Of New York Genome edited cancer cell vaccines
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
US12624086B2 (en) * 2018-10-31 2026-05-12 Mayo Foundation For Medical Education And Research Materials and methods for treating cancer
CN109852627B (zh) * 2018-12-26 2022-07-05 中国人民解放军军事科学院军事医学研究院 用于CRISPRi系统的质粒、其构建方法及其在使鼠疫菌目的基因定向沉默中的应用
EP3911371A4 (en) * 2019-01-18 2023-04-26 University of Southern California METHODS AND COMPOSITIONS FOR IMPROVING THE SAFETY AND EFFICACY OF CELL THERAPY
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020172638A1 (en) * 2019-02-22 2020-08-27 The Trustees Of The University Of Pennsylvania Crispr/cas9 knock-out of cd33 in human hematopoietic stem/ progenitor cells for allogenic transplantation
EP4219700A1 (en) 2019-03-07 2023-08-02 The Regents of the University of California Crispr-cas effector polypeptides and methods of use thereof
WO2020191241A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
WO2021041977A1 (en) * 2019-08-28 2021-03-04 Vor Biopharma, Inc. Compositions and methods for cd123 modification
AU2020338011A1 (en) * 2019-08-28 2022-03-10 Vor Biopharma Inc. Compositions and methods for CLL1 modification
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
AU2020361683A1 (en) 2019-10-11 2022-05-19 The Trustees Of The University Of Pennsylvania Compositions and methods for targeting CD13 and TIM-3 with CAR T cells to treat acute myeloid leukemia
KR102923195B1 (ko) 2020-02-28 2026-02-04 현대자동차주식회사 리튬이온 전지 음극 제조방법 및 이에 의해 제조된 리튬이온 전지 음극을 포함하는 리튬이온 전지
CA3177481A1 (en) 2020-05-08 2021-11-11 David R. Liu Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
EP3909612A1 (en) * 2020-05-12 2021-11-17 Life Science Inkubator Betriebs GmbH & Co. KG Composition of nanoparticles
AU2021413252A1 (en) * 2020-12-31 2023-06-08 Vor Biopharma Inc. Compositions and methods for cd34 gene modification
CN115443333B (zh) * 2021-04-27 2025-04-25 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
PE20252304A1 (es) * 2021-04-28 2025-09-22 Eisai Randd Man Co Ltd Oligonucleotidos antisentido y su uso para el tratamiento de trastornos neurodegenerativos
EP4430186A1 (en) * 2021-11-09 2024-09-18 Vor Biopharma Inc. Compositions and methods for erm2 modification
EP4433512A4 (en) 2021-11-19 2025-12-31 Univ Pennsylvania Genetically modified pan-leukocyte-specific CD45 antigen to facilitate T-lymphocyte therapy
EP4463190A4 (en) * 2022-01-11 2026-01-14 Actinium Pharmaceuticals Inc TREATMENT METHODS FOR CD33-POSITIVE HEMATOLOGICAL MALIGNITIES
WO2025030010A1 (en) 2023-08-01 2025-02-06 Vor Biopharma Inc. Compositions comprising genetically engineered hematopoietic stem cells and methods of use thereof
WO2025059062A1 (en) * 2023-09-11 2025-03-20 The Trustees Of The University Of Pennsylvania Genetic engineering of human hematopoietic stem/progenitor cells (hspcs) for locus-specific expression of therapeutic proteins

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100172882A1 (en) * 2007-01-11 2010-07-08 Glazer Peter M Compositions and methods for targeted inactivation of hiv cell surface receptors
WO2014059173A2 (en) * 2012-10-10 2014-04-17 Sangamo Biosciences, Inc. T cell modifying compounds and uses thereof
WO2014130635A1 (en) * 2013-02-20 2014-08-28 Novartis Ag Effective targeting of primary human leukemia using anti-cd123 chimeric antigen receptor engineered t cells
JP6608807B2 (ja) * 2013-05-29 2019-11-20 セレクティス Ｒｎａガイドｃａｓヌクレアーゼ系を用いることによって免疫療法のためにｔ細胞を操作するための方法
SG10202109752XA (en) * 2014-04-07 2021-10-28 Novartis Ag Treatment of cancer using anti-cd19 chimeric antigen receptor
IL258491B2 (en) * 2015-10-16 2023-09-01 Univ Columbia Preparations and methods for inhibiting antigens of a particular lineage
EP3370741B1 (en) * 2015-11-04 2025-10-08 The Trustees of the University of Pennsylvania Methods and compositions for gene editing in hematopoietic stem cells

Also Published As

Publication number Publication date
EA201891092A1 (ru) 2018-10-31
US20240066064A1 (en) 2024-02-29
HK1259151A1 (en) 2019-11-29
AU2024200000A1 (en) 2024-03-21
US10548922B2 (en) 2020-02-04
JP2018533625A (ja) 2018-11-15
JP2023115069A (ja) 2023-08-18
JP2021106595A (ja) 2021-07-29
SG10202107602XA (en) 2021-08-30
AU2016349280B2 (en) 2023-10-05
CN108463227A (zh) 2018-08-28
EP3370741C0 (en) 2025-10-08
US20180250339A1 (en) 2018-09-06
BR112018009098A2 (en) 2018-11-06
US20200281975A1 (en) 2020-09-10
JP6866385B2 (ja) 2021-04-28
PH12018500965A1 (en) 2018-11-12
EP3370741A1 (en) 2018-09-12
MX2018005612A (es) 2018-11-09
EP3370741B1 (en) 2025-10-08
AU2016349280A1 (en) 2018-05-24
JP7378439B2 (ja) 2023-11-13
IL259084A (en) 2018-06-28
CA3004053A1 (en) 2017-05-11
BR112018009098A8 (pt) 2019-02-26
SG11201803701YA (en) 2018-06-28
WO2017079400A1 (en) 2017-05-11
KR20180073679A (ko) 2018-07-02
MY185961A (en) 2021-06-14
EP3370741A4 (en) 2019-04-24
US11771719B2 (en) 2023-10-03

Similar Documents

Publication Publication Date Title
PH12018500965A1 (en) Methods and compositions for gene editing in hematopoietic stem cells
MX2024001192A (es) Anticuerpos anti gen 3 de activacion linfocitica (lag-3) y metodos para usarlos.
AR134186A2 (es) Un anticuerpo anti-csf-1r, un proceso para producirlo, una composición farmacéutica que lo comprende, una secuencia de adn aislado, un vector de expresión o clonación y una célula hospedadora
BR112019007288A2 (pt) proteína heterodimérica biespecífica, composições de ácido nucleico e de vetor de expressão, vetor de expressão, célula hospedeira, e, métodos para produzir proteína heterodimérica biespecífica e para tratar câncer em um paciente
MX2017001011A (es) Tratamiento de cancer de usando un receptor quimerico de antigeno anti-bcma.
BR112018067368A2 (pt) terapia de combinação com anticorpos anti-cd73
CL2018001414A1 (es) Anticuerpos contra cd73 y sus usos (divisional solicitud 201701296)
MX2023001878A (es) Celulas modificadas geneticamente que comprenden un gen humano modificado de la region constante alfa del receptor de celulas t.
BR112017000939A2 (pt) tratamento de câncer usando um receptor antigênico quimérico de cll-1
ECSP17028310A (es) Composiciones y métodos para inhibir la expresión del gen HAO1 (hidroxiácido-oxidasa 1 (glicolato-oxidasa))
MX2018010733A (es) Celulas que expresan multiples moleculas del receptor de antigeno quimerico (car) y usos de las mismas.
MX2023000818A (es) Anticuerpos anti-antigeno 4 del linfocito t citotoxico (ctla-4) y metodos para uso de los mismos.
CO2017010190A2 (es) Terapias cd20, terapias cd22 y terapias de combinación con una célula que expresa un receptor quimérico de antígeno (car) de cd19
MX2017005698A (es) Alteracion de la expresion genetica en celulas t modificadas con el receptor de antigeno quimerico (cart) y usos de las mismas.
BR112018005937A2 (pt) conjugados de oligonucleotídeo de afinidade e usos destes
EA202090931A3 (ru) Композиции и способы перепрограммирования tcr с помощью гибридных белков
CO2019003951A2 (es) Proteínas de unión a cd123 y composiciones y métodos relacionados
BR112015021819A2 (pt) Tratamento de câncer utilizando receptor quimérico de antígeno humanizado anti-cd19
MX2015010836A (es) Tratamiento de cancer utilizando un receptor quimerico de antigeno anti egfrviii humanizado.
MX2023002858A (es) Prediccion de epitopos de celulas t utiles para vacunacion.
CO2017001573A2 (es) Receptor quimérico de antígeno anti-cd123
AR102879A1 (es) Métodos para el tratamiento de tumores cerebrales
MX2017014908A (es) Proteinas de union triespecificas y metodos de uso.
BR112019004711A2 (pt) receptores de antígeno quiméricos compreendendo domínios de fibronectina tipo iii específicos para bcma e usos dos mesmos
EP4406610A3 (en) Treatment of cancer using anti-cd19 chimeric antigen receptor