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)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Immunology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Cell Biology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Hematology (AREA)
  • Virology (AREA)
  • Oncology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (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 (https=)
EP (1) EP3370741B1 (https=)
JP (3) JP6866385B2 (https=)
KR (1) KR20180073679A (https=)
CN (1) CN108463227A (https=)
AU (2) AU2016349280B2 (https=)
BR (1) BR112018009098A8 (https=)
CA (1) CA3004053A1 (https=)
CL (1) CL2018001198A1 (https=)
EA (1) EA201891092A1 (https=)
IL (1) IL259084A (https=)
MX (1) MX2018005612A (https=)
MY (1) MY185961A (https=)
PH (1) PH12018500965A1 (https=)
SG (2) SG10202107602XA (https=)
WO (1) WO2017079400A1 (https=)

Families Citing this family (60)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10323236B2 (en) 2011-07-22 2019-06-18 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
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
US10077453B2 (en) 2014-07-30 2018-09-18 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
AU2016337525B2 (en) 2015-10-16 2022-01-20 The Trustees Of Columbia University In The City Of New York Compositions and methods for inhibition of lineage specific antigens
IL310721B2 (en) 2015-10-23 2025-11-01 Harvard College Nucleobase editors and uses thereof
WO2017079400A1 (en) * 2015-11-04 2017-05-11 The Trustees Of The University Of Pennsylvania Methods and compositions for gene editing in hematopoietic stem cells
CN108495640B (zh) * 2016-02-19 2022-11-04 加利福尼亚大学董事会 短发夹rna(shrna734)及其用于阳性选择和消除遗传修饰的细胞的用途
CN110214183A (zh) 2016-08-03 2019-09-06 哈佛大学的校长及成员们 腺苷核碱基编辑器及其用途
WO2018031683A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
KR102622411B1 (ko) 2016-10-14 2024-01-10 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
LT3535392T (lt) * 2016-11-02 2024-04-25 Universität Basel Imunologiškai atpažįstami ląstelių paviršiaus variantai, skirti naudoti ląstelių terapijoje
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
RU2019130504A (ru) * 2017-02-28 2021-03-30 Вор Байофарма, Инк. Композиции и способы ингибирования линиеспецифических белков
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
US12390514B2 (en) 2017-03-09 2025-08-19 President And Fellows Of Harvard College Cancer vaccine
US11542496B2 (en) 2017-03-10 2023-01-03 President And Fellows Of Harvard College Cytosine to guanine base editor
KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
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
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
KR20250107288A (ko) 2017-10-16 2025-07-11 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
KR20210045360A (ko) 2018-05-16 2021-04-26 신테고 코포레이션 가이드 rna 설계 및 사용을 위한 방법 및 시스템
US12157760B2 (en) 2018-05-23 2024-12-03 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
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
CA3110837A1 (en) 2018-08-28 2020-03-05 Vor Biopharma Inc. Genetically engineered hematopoietic stem cells and uses thereof
WO2020061256A1 (en) * 2018-09-19 2020-03-26 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
WO2020092850A1 (en) * 2018-10-31 2020-05-07 Humanigen, Inc. 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
US20220202900A1 (en) * 2019-02-22 2022-06-30 The Trustees Of The University Of Pennsylvania Compositions and methods for crispr/cas9 knock-out of cd33 in human hematopoietic stem / progenitor cells for allogenic transplantation in patients with relapsed - refractory acute myeloid leukemia
MX2021010559A (es) 2019-03-07 2021-12-15 Univ California Polipéptidos efectores de crispr-cas y métodos de uso de estos.
WO2020191233A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
AU2020338011A1 (en) * 2019-08-28 2022-03-10 Vor Biopharma Inc. Compositions and methods for CLL1 modification
WO2021041977A1 (en) * 2019-08-28 2021-03-04 Vor Biopharma, Inc. Compositions and methods for cd123 modification
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
EP4041297A4 (en) 2019-10-11 2024-02-28 The Trustees of The University of Pennsylvania COMPOSITIONS AND METHODS FOR TARGETING CD13 AND TIM-3 WITH CAR-T CELLS FOR TREATING 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
IL297761A (en) 2020-05-08 2022-12-01 Broad Inst Inc Methods and compositions for simultaneously editing two helices of a designated double-helix nucleotide sequence
EP3909612A1 (en) * 2020-05-12 2021-11-17 Life Science Inkubator Betriebs GmbH & Co. KG Composition of nanoparticles
JP2024502820A (ja) * 2020-12-31 2024-01-23 ブイオーアール バイオファーマ インコーポレーテッド Ｃｄ３４遺伝子改変のための組成物及び方法
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
TW202309283A (zh) * 2021-04-28 2023-03-01 日商衛材R&D企管股份有限公司 反義寡核苷酸及其用於治療神經變性障礙之用途
JP2024543369A (ja) * 2021-11-09 2024-11-21 ブイオーアール バイオファーマ インコーポレーテッド Ｅｍｒ２修飾のための化合物及び方法
EP4433512A4 (en) 2021-11-19 2025-12-31 Univ Pennsylvania Genetically modified pan-leukocyte-specific CD45 antigen to facilitate T-lymphocyte therapy
WO2023137335A2 (en) * 2022-01-11 2023-07-20 Actinium Pharmaceuticals, Inc. Methods for treating cd33-positive hematological malignancies
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
WO2008086529A2 (en) * 2007-01-11 2008-07-17 Yale University Compositions and methods for targeted inactivation of hiv cell surface receptors
AU2013329186B2 (en) * 2012-10-10 2019-02-14 Sangamo Therapeutics, Inc. T cell modifying compounds and uses thereof
US9573988B2 (en) * 2013-02-20 2017-02-21 Novartis Ag Effective targeting of primary human leukemia using anti-CD123 chimeric antigen receptor engineered T cells
WO2014191128A1 (en) * 2013-05-29 2014-12-04 Cellectis Methods for engineering t cells for immunotherapy by using rna-guided cas nuclease system
AU2015244039B2 (en) * 2014-04-07 2021-10-21 Novartis Ag Treatment of cancer using anti-CD19 chimeric antigen receptor
AU2016337525B2 (en) * 2015-10-16 2022-01-20 The Trustees Of Columbia University In The City Of New York Compositions and methods for inhibition of lineage specific antigens
WO2017079400A1 (en) * 2015-11-04 2017-05-11 The Trustees Of The University Of Pennsylvania Methods and compositions for gene editing in hematopoietic stem cells

Also Published As

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

Similar Documents

Publication Publication Date Title
CL2018001198A1 (es) Métodos y composiciones para edición génica en hemocitoblastos
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)
MX2019003683A (es) Anticuerpos anti gen 3 de activación linfocítica (lag 3 ) y métodos para usarlos.
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
MX395149B (es) Terapias de combinación que comprenden un receptor quimérico de antígeno cd19 para el cáncer.
BR112019004711A2 (pt) receptores de antígeno quiméricos compreendendo domínios de fibronectina tipo iii específicos para bcma e usos dos mesmos
PL422231A1 (pl) Komórki immunologiczne CAR do leczenia nowotworów