IL268406B2 - Compositions and methods for the treatment of hemoglobinopathies - Google Patents

Compositions and methods for the treatment of hemoglobinopathies

Info

Publication number
IL268406B2
IL268406B2 IL268406A IL26840619A IL268406B2 IL 268406 B2 IL268406 B2 IL 268406B2 IL 268406 A IL268406 A IL 268406A IL 26840619 A IL26840619 A IL 26840619A IL 268406 B2 IL268406 B2 IL 268406B2
Authority
IL
Israel
Prior art keywords
cell
seq
cells
grna
population
Prior art date
Application number
IL268406A
Other languages
English (en)
Hebrew (he)
Other versions
IL268406A (en
IL268406B1 (en
Original Assignee
Novartis Ag
Intellia Therapeutics 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 Novartis Ag, Intellia Therapeutics Inc filed Critical Novartis Ag
Publication of IL268406A publication Critical patent/IL268406A/en
Publication of IL268406B1 publication Critical patent/IL268406B1/en
Publication of IL268406B2 publication Critical patent/IL268406B2/en

Links

Classifications

    • 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
    • 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
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
    • 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
    • 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
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Hematology (AREA)
  • Epidemiology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicinal Preparation (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
IL268406A 2017-02-06 2018-02-05 Compositions and methods for the treatment of hemoglobinopathies IL268406B2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762455464P 2017-02-06 2017-02-06
PCT/IB2018/050712 WO2018142364A1 (en) 2017-02-06 2018-02-05 Compositions and methods for the treatment of hemoglobinopathies

Publications (3)

Publication Number Publication Date
IL268406A IL268406A (en) 2019-09-26
IL268406B1 IL268406B1 (en) 2024-04-01
IL268406B2 true IL268406B2 (en) 2024-08-01

Family

ID=61526837

Family Applications (2)

Application Number Title Priority Date Filing Date
IL268406A IL268406B2 (en) 2017-02-06 2018-02-05 Compositions and methods for the treatment of hemoglobinopathies
IL311222A IL311222A (en) 2017-02-06 2018-02-05 Preparations and methods for treating hemoglobin diseases

Family Applications After (1)

Application Number Title Priority Date Filing Date
IL311222A IL311222A (en) 2017-02-06 2018-02-05 Preparations and methods for treating hemoglobin diseases

Country Status (18)

Country Link
US (2) US11466271B2 (enExample)
EP (1) EP3577223A1 (enExample)
JP (3) JP2020505934A (enExample)
KR (2) KR102630217B1 (enExample)
CN (2) CN110546262B (enExample)
AR (1) AR110962A1 (enExample)
AU (3) AU2018215726B2 (enExample)
BR (1) BR112019016205A2 (enExample)
CA (1) CA3052275A1 (enExample)
EA (1) EA201991862A1 (enExample)
IL (2) IL268406B2 (enExample)
MX (1) MX2019009361A (enExample)
MY (1) MY203407A (enExample)
PH (1) PH12019501812A1 (enExample)
SA (1) SA519402400B1 (enExample)
SG (1) SG11201907056XA (enExample)
TW (1) TW201839136A (enExample)
WO (1) WO2018142364A1 (enExample)

Families Citing this family (59)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10704021B2 (en) 2012-03-15 2020-07-07 Flodesign Sonics, Inc. Acoustic perfusion devices
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
CA2935960C (en) 2014-01-08 2023-01-10 Bart Lipkens Acoustophoresis device with dual acoustophoretic chamber
EP3122880B1 (en) 2014-03-26 2021-05-05 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
US11377651B2 (en) 2016-10-19 2022-07-05 Flodesign Sonics, Inc. Cell therapy processes utilizing acoustophoresis
US11708572B2 (en) 2015-04-29 2023-07-25 Flodesign Sonics, Inc. Acoustic cell separation techniques and processes
EP3294896A1 (en) 2015-05-11 2018-03-21 Editas Medicine, Inc. Optimized crispr/cas9 systems and methods for gene editing in stem cells
WO2016201047A1 (en) 2015-06-09 2016-12-15 Editas Medicine, Inc. Crispr/cas-related methods and compositions for improving transplantation
US12043852B2 (en) 2015-10-23 2024-07-23 President And Fellows Of Harvard College Evolved Cas9 proteins for gene editing
US11214789B2 (en) 2016-05-03 2022-01-04 Flodesign Sonics, Inc. Concentration and washing of particles with acoustics
KR20250103795A (ko) 2016-08-03 2025-07-07 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
CN109804066A (zh) 2016-08-09 2019-05-24 哈佛大学的校长及成员们 可编程cas9-重组酶融合蛋白及其用途
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
AU2017342543B2 (en) 2016-10-14 2024-06-27 President And Fellows Of Harvard College AAV delivery of nucleobase editors
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
TW201839136A (zh) * 2017-02-06 2018-11-01 瑞士商諾華公司 治療血色素異常症之組合物及方法
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
EP3592381A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Cancer vaccine
KR20190127797A (ko) 2017-03-10 2019-11-13 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 시토신에서 구아닌으로의 염기 편집제
WO2018170184A1 (en) 2017-03-14 2018-09-20 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
CA3057192A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
WO2018209158A2 (en) 2017-05-10 2018-11-15 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
US20200140896A1 (en) * 2017-06-30 2020-05-07 Novartis Ag Methods for the treatment of disease with gene editing systems
EP3652312A1 (en) 2017-07-14 2020-05-20 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
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
WO2019139645A2 (en) 2017-08-30 2019-07-18 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
US20210180053A1 (en) 2017-11-01 2021-06-17 Novartis Ag Synthetic rnas and methods of use
KR20220066413A (ko) 2017-12-14 2022-05-24 프로디자인 소닉스, 인크. 음향 트랜스듀서 구동기 및 제어기
EP3724214A4 (en) 2017-12-15 2021-09-01 The Broad Institute Inc. SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING
SG11202008956XA (en) 2018-03-14 2020-10-29 Editas Medicine Inc Systems and methods for the treatment of hemoglobinopathies
US12133884B2 (en) 2018-05-11 2024-11-05 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
US12281338B2 (en) 2018-10-29 2025-04-22 The Broad Institute, Inc. Nucleobase editors comprising GeoCas9 and uses thereof
EP3887521A1 (en) * 2018-11-29 2021-10-06 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
WO2020112195A1 (en) * 2018-11-30 2020-06-04 Yale University Compositions, technologies and methods of using plerixafor to enhance gene editing
KR20210102309A (ko) * 2018-12-05 2021-08-19 프레드 헛친슨 켄서 리서치 센터 유전적으로-변형된 세포를 제작하는 줄어든, 그리고 최소한의 조작
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
AU2020223060B2 (en) 2019-02-13 2023-04-13 Beam Therapeutics Inc. Compositions and methods for treating hemoglobinopathies
DE112020001306T5 (de) 2019-03-19 2022-01-27 Massachusetts Institute Of Technology Verfahren und zusammensetzungen zur editierung von nukleotidsequenzen
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
CN111939271A (zh) * 2019-04-30 2020-11-17 博雅辑因（北京）生物科技有限公司 一种血红蛋白病治疗有效性预测方法
PE20220003A1 (es) * 2019-04-30 2022-01-05 Edigene Inc Metodo para la prediccion de eficacia del tratamiento de hemoglobinopatia
CN110106203B (zh) * 2019-05-24 2023-08-11 中国医学科学院血液病医院（血液学研究所） 一种新型hbb过表达载体及其设计方法和应用
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
EP4054710A4 (en) * 2019-11-06 2023-12-06 Syros Pharmaceuticals, Inc. Compositions and methods for treating sickle cell disease
BR112022020407A2 (pt) * 2020-04-09 2023-05-02 Verve Therapeutics Inc Edição base do pcsk9 e métodos de uso do mesmo para tratamento de doenças
JP2023525304A (ja) 2020-05-08 2023-06-15 ザ ブロード インスティテュート，インコーポレーテッド 標的二本鎖ヌクレオチド配列の両鎖同時編集のための方法および組成物
CN111575306B (zh) * 2020-06-11 2022-08-05 南方医科大学 一种激活红细胞中γ珠蛋白基因表达的方法
EP4196260A4 (en) * 2020-08-14 2025-04-30 Navan Technologies, Inc. METHODS AND APPARATUS FOR DELIVERING BIOMOLECULES TO PRIMARY HUMAN HEMATOPOIETIC STEM CELLS USING NANOSTRAWS
WO2022170059A1 (en) 2021-02-05 2022-08-11 Christiana Care Health Services, Inc. Methods of and compositions for reducing gene expression and/or activity
US20250179531A1 (en) * 2022-02-25 2025-06-05 Vor Biopharma Inc. Compositions and methods for homology-directed repair gene modification
WO2024163679A1 (en) * 2023-02-01 2024-08-08 Prime Medicine, Inc. Genome editing compositions and methods for treatment of cystic fibrosis
CN116445402B (zh) * 2023-05-09 2025-04-25 中国科学院生态环境研究中心 红系祖细胞的扩增培养基、扩增培养方法及应用
WO2024254566A2 (en) * 2023-06-08 2024-12-12 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013126794A1 (en) * 2012-02-24 2013-08-29 Fred Hutchinson Cancer Research Center Compositions and methods for the treatment of hemoglobinopathies
WO2015089486A2 (en) * 2013-12-12 2015-06-18 The Broad Institute Inc. Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems
WO2016135558A2 (en) * 2015-02-23 2016-09-01 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies

Family Cites Families (234)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5800815A (en) 1903-05-05 1998-09-01 Cytel Corporation Antibodies to P-selectin and their uses
US6309639B1 (en) 1991-02-05 2001-10-30 The Board Of Regents Of The University Of Oklahoma Method for inhibiting an inflammatory response using antibodies to P-selectin glycoprotein ligand
US5213796A (en) 1991-05-06 1993-05-25 Dana Farber Cancer Institute Assay for polyomavirus in humans and uses thereof
US5726014A (en) 1991-06-27 1998-03-10 Genelabs Technologies, Inc. Screening assay for the detection of DNA-binding molecules
US5578444A (en) 1991-06-27 1996-11-26 Genelabs Technologies, Inc. Sequence-directed DNA-binding molecules compositions and methods
DE69332881T2 (de) 1992-05-05 2004-01-29 Aeres Biomedical Ltd Antikörper gegen p-selectin und ihre verwendung
US7910624B1 (en) 1995-03-03 2011-03-22 The Trustees Of Boston University Compositions for the treatment of blood disorders
AU696167B2 (en) 1993-10-29 1998-09-03 Trustees Of Boston University Physiologically stable compositions of butyric acid, and butyric acid salts and derivatives as anti-neoplastic agents
US5622701A (en) 1994-06-14 1997-04-22 Protein Design Labs, Inc. Cross-reacting monoclonal antibodies specific for E- and P-selectin
US6453242B1 (en) 1999-01-12 2002-09-17 Sangamo Biosciences, Inc. Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
US7013219B2 (en) 1999-01-12 2006-03-14 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US7070934B2 (en) 1999-01-12 2006-07-04 Sangamo Biosciences, Inc. Ligand-controlled regulation of endogenous gene expression
US6780590B2 (en) 1999-09-14 2004-08-24 Sangamo Biosciences, Inc. Gene identification
US20020164575A1 (en) 1999-09-14 2002-11-07 Sangamo Biosciences, Inc., A Delaware Corporation Gene identification
AU776576B2 (en) 1999-12-06 2004-09-16 Sangamo Biosciences, Inc. Methods of using randomized libraries of zinc finger proteins for the identification of gene function
IT1320168B1 (it) 2000-03-13 2003-11-18 Univ Ferrara Oligonucleotidi sintetici in grado di indurre il differenziamentoeritroide.
WO2001085765A2 (en) 2000-05-12 2001-11-15 Merck Patent Gmbh Identification of mfq-110, a human c2h2-type zinc finger protein
US20050054836A1 (en) 2000-11-09 2005-03-10 Cold Spring Harbor Laboratory Chimeric molecules to modulate gene expression
WO2002044386A2 (en) 2000-12-01 2002-06-06 Sangamo Biosciences, Inc. Targeted regulation of gene expression
AU2002309583A1 (en) 2001-04-18 2002-11-05 Protein Desing Labs, Inc. Methods of diagnosis of lung cancer, compositions and methods of screening for modulators of lung cancer
US8735153B2 (en) 2001-09-24 2014-05-27 Sangamo Biosciences, Inc. Modulation of stem cells using zinc finger proteins
JP2006502748A (ja) 2002-09-05 2006-01-26 カリフォルニア インスティテュート オブ テクノロジー 遺伝子ターゲッティングを誘発するキメラヌクレアーゼの使用方法
EP1581056B1 (en) 2002-12-13 2010-07-21 Genetix Pharmaceuticals Inc. Therapeutic retroviral vectors for gene therapy
WO2004098508A2 (en) 2003-04-30 2004-11-18 Napro Biotherapeutics, Inc. Methods for production of non-disease causing hemoglobin by ex vivo oligonucleotide gene editing of human stem/progenitor cells
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
EP3222715A1 (en) 2003-08-08 2017-09-27 Sangamo BioSciences, Inc. Methods and compositions for targeted cleavage and recombination
WO2005060697A2 (en) 2003-12-19 2005-07-07 Chiron Corporation Cell transfecting formulations of small interfering rna, related compositions and methods of making and use
US20050244851A1 (en) 2004-01-13 2005-11-03 Affymetrix, Inc. Methods of analysis of alternative splicing in human
US7972854B2 (en) 2004-02-05 2011-07-05 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
BR122019012028B1 (pt) 2004-04-13 2023-09-26 F. Hoffmann-La Roche Ag Anticorpos anti-p-selectina, molécula de ácido nucléico, vetor, e composição
US20100055793A1 (en) 2005-07-25 2010-03-04 Johns Hopkins University Site-specific modification of the human genome using custom-designed zinc finger nucleases
US8377440B2 (en) 2006-12-01 2013-02-19 Selexys Pharmaceuticals Corporation Anti-P-selectin antibodies and methods of using the same to treat inflammatory diseases
US20110212096A1 (en) 2006-12-01 2011-09-01 Scott Rollins Anti-p-selectin antibodies and methods of their use and identification
US8945565B2 (en) 2006-12-01 2015-02-03 Selexys Pharmaceuticals Corporation Methods of treating inflammatory or thrombotic conditions with anti-P-selectin antibodies
GB0713183D0 (en) 2007-07-06 2007-08-15 King S College London Method
WO2010030963A2 (en) 2008-09-15 2010-03-18 Children's Medical Center Corporation Modulation of bcl11a for treatment of hemoglobinopathies
PE20100362A1 (es) 2008-10-30 2010-05-27 Irm Llc Derivados de purina que expanden las celulas madre hematopoyeticas
EP3721943A1 (en) 2009-12-23 2020-10-14 Novartis AG Lipids, lipid compositions and methods of using them
WO2011103215A1 (en) 2010-02-16 2011-08-25 Mount Sinai School Of Medicine Embedded chimeric peptide nucleic acids and use thereof
WO2012075027A1 (en) 2010-11-29 2012-06-07 Mount Sinai School Of Medicine Embedded chimeric peptide nucleic acids for generation of induced pluripotent stem cells
BR112013000244A2 (pt) 2010-07-06 2016-05-17 Novartis Ag lipossomas com lipídeos apresentando pka vantajoso para administração de rna
US20140127814A1 (en) 2010-08-10 2014-05-08 Srinivasan Chandrasegaran Generation and use of pluripotent stem cells
JP5908477B2 (ja) 2010-08-31 2016-04-26 ノバルティス アーゲー タンパク質をコードするｒｎａのリポソームによる送達に適した脂質
US9127275B2 (en) 2010-12-10 2015-09-08 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of klf-1 and bcl11a genes
WO2012112232A1 (en) 2011-02-18 2012-08-23 Academia Sinica METHODS AND COMPOSITIONS FOR TREATING β-THALASSEMIA AND SICKLE CELL DISEASE
DE202011050611U1 (de) 2011-07-01 2012-10-09 Makita Corporation Anordnung zum Bereitstellen eines Kraftstoff-Luftgemischs für eine Brennkraftmaschine
EP4115875A1 (en) 2011-07-06 2023-01-11 GlaxoSmithKline Biologicals S.A. Liposomes having useful n:p ratio for delivery of rna molecules
WO2013019745A1 (en) 2011-07-29 2013-02-07 Georgia Health Sciences University Methods and compositions for genetically modifiying cells
WO2013055985A1 (en) 2011-10-12 2013-04-18 Children's Medical Center Corporation Combinatorial compositions and methods of treating hemoglobinopathies
JP6219832B2 (ja) 2011-10-17 2017-10-25 マサチューセッツ インスティテュート オブ テクノロジー 細胞内送達
US9394547B2 (en) 2012-01-03 2016-07-19 City University Of Hong Kong Method and apparatus for delivery of molecules to cells
WO2013103467A1 (en) 2012-01-06 2013-07-11 Alcon Research, Ltd. Interfering rna delivery system and uses thereof
SI2807165T1 (sl) 2012-01-27 2019-08-30 Universite De Montreal Pirimido(4,5-B)indol derivati in njihova uporaba pri ekspanziji hematopoetskih matičnih celic
ES2828663T3 (es) 2012-04-18 2021-05-27 Univ Leland Stanford Junior Dirección genética no disruptiva
US20150017136A1 (en) 2013-07-15 2015-01-15 Cellectis Methods for engineering allogeneic and highly active t cell for immunotherapy
CA3133545C (en) 2012-05-25 2023-08-08 Cellectis Use of pre t alpha or functional variant thereof for expanding tcr alpha deficient t cells
FI3597749T3 (fi) 2012-05-25 2023-10-09 Univ California Menetelmiä ja koostumuksia rna-ohjattua kohde-dna-modifikaatiota varten ja rna-ohjattua transkription modulaatiota varten
CN104540382A (zh) 2012-06-12 2015-04-22 弗·哈夫曼-拉罗切有限公司 用于产生条件性敲除等位基因的方法和组合物
WO2014011901A2 (en) 2012-07-11 2014-01-16 Sangamo Biosciences, Inc. Methods and compositions for delivery of biologics
EP2877213B1 (en) 2012-07-25 2020-12-02 The Broad Institute, Inc. Inducible dna binding proteins and genome perturbation tools and applications thereof
AU2013308770B2 (en) 2012-08-29 2019-01-17 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition
AU2013312838B2 (en) 2012-09-04 2018-11-29 Cellectis Multi-chain chimeric antigen receptor and uses thereof
KR101656236B1 (ko) 2012-10-23 2016-09-12 주식회사 툴젠 표적 DNA에 특이적인 가이드 RNA 및 Cas 단백질을 암호화하는 핵산 또는 Cas 단백질을 포함하는, 표적 DNA를 절단하기 위한 조성물 및 이의 용도
AU2013352156B2 (en) 2012-11-27 2018-12-06 Children's Medical Center Corporation Targeting BCL11A distal regulatory elements for fetal hemoglobin reinduction
PT3363902T (pt) 2012-12-06 2019-12-19 Sigma Aldrich Co Llc Modificação e regulação de genoma baseadas em crispr
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
EP3434776A1 (en) 2012-12-12 2019-01-30 The Broad Institute, Inc. Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
PT2784162E (pt) 2012-12-12 2015-08-27 Broad Inst Inc Engenharia de sistemas, métodos e composições guia otimizadas para a manipulação de sequências
EP4234696A3 (en) 2012-12-12 2023-09-06 The Broad Institute Inc. Crispr-cas component systems, methods and compositions for sequence manipulation
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
ES2576126T3 (es) 2012-12-12 2016-07-05 The Broad Institute, Inc. Modificación por tecnología genética y optimización de sistemas, métodos y composiciones enzimáticas mejorados para la manipulación de secuencias
KR20150105956A (ko) 2012-12-12 2015-09-18 더 브로드 인스티튜트, 인코퍼레이티드 서열 조작 및 치료적 적용을 위한 시스템, 방법 및 조성물의 전달, 유전자 조작 및 최적화
EP2931899A1 (en) 2012-12-12 2015-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
EP2932421A1 (en) 2012-12-12 2015-10-21 The Broad Institute, Inc. Methods, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
EP2931898B1 (en) 2012-12-12 2016-03-09 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
EP4282970A3 (en) 2012-12-17 2024-01-17 President and Fellows of Harvard College Rna-guided human genome engineering
CA2901676C (en) 2013-02-25 2023-08-22 Sangamo Biosciences, Inc. Methods and compositions for enhancing nuclease-mediated gene disruption
ES2750550T3 (es) 2013-03-01 2020-03-26 Univ Minnesota Corrección de gen a base de TALEN
CN107879960B (zh) 2013-03-08 2021-06-22 诺华股份有限公司 用于传递活性成分的脂质和脂质组合物
US10612043B2 (en) 2013-03-09 2020-04-07 Agilent Technologies, Inc. Methods of in vivo engineering of large sequences using multiple CRISPR/cas selections of recombineering events
AU2014235794A1 (en) 2013-03-14 2015-10-22 Caribou Biosciences, Inc. Compositions and methods of nucleic acid-targeting nucleic acids
US11332719B2 (en) 2013-03-15 2022-05-17 The Broad Institute, Inc. Recombinant virus and preparations thereof
US10760064B2 (en) 2013-03-15 2020-09-01 The General Hospital Corporation RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
HUE042072T2 (hu) 2013-03-15 2019-06-28 Glaxosmithkline Biologicals Sa Eljárások RNS tisztítására
US20140273230A1 (en) 2013-03-15 2014-09-18 Sigma-Aldrich Co., Llc Crispr-based genome modification and regulation
KR102874079B1 (ko) 2013-03-15 2025-10-22 더 제너럴 하스피탈 코포레이션 Rna-안내 게놈 편집을 위해 특이성을 증가시키기 위한 절단된 안내 rna(tru-grnas)의 이용
EP2968295A1 (en) 2013-03-15 2016-01-20 Global Blood Therapeutics, Inc. Compositions and methods for the modulation of hemoglobin (s)
US11274305B2 (en) 2013-04-04 2022-03-15 Trustees Of Dartmouth College Compositions and methods for in vivo excision of HIV-1 proviral DNA
US20150056629A1 (en) 2013-04-14 2015-02-26 Katriona Guthrie-Honea Compositions, systems, and methods for detecting a DNA sequence
WO2014172470A2 (en) 2013-04-16 2014-10-23 Whitehead Institute For Biomedical Research Methods of mutating, modifying or modulating nucleic acid in a cell or nonhuman mammal
AU2014266833B2 (en) 2013-05-13 2020-07-02 Cellectis Methods for engineering highly active T cell for immunotherapy
RS65484B1 (sr) 2013-05-13 2024-05-31 Cellectis Cd19 specifični himerni antigenski receptor i njegove primene
AU2014265331B2 (en) 2013-05-15 2019-12-05 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition
BR112015029327A2 (pt) 2013-05-24 2017-09-19 Roche Innovation Ct Copenhagen As Moduladores de oligonucleotídeo de linfoma 11a/cll de célula b (bcl11a) e usos dos mesmos
DK3004337T3 (da) 2013-05-29 2017-11-13 Cellectis Fremgangsmåde til konstruktion af T-celler til immunoterapi ved brug af RNA-guidet Cas nuklease-system
US11685935B2 (en) 2013-05-29 2023-06-27 Cellectis Compact scaffold of Cas9 in the type II CRISPR system
RU2690935C2 (ru) 2013-06-04 2019-06-06 Президент Энд Фэллоуз Оф Харвард Коллидж Направляемая рнк регуляция транскрипции
US20140356956A1 (en) 2013-06-04 2014-12-04 President And Fellows Of Harvard College RNA-Guided Transcriptional Regulation
JP7085716B2 (ja) * 2013-06-05 2022-06-17 デューク ユニバーシティ Ｒｎａガイド遺伝子編集及び遺伝子調節
EP4491726A3 (en) 2013-06-11 2025-03-05 Takara Bio USA, Inc. Protein enriched microvesicles and methods of making and using the same
US20150315252A1 (en) 2013-06-11 2015-11-05 Clontech Laboratories, Inc. Protein enriched microvesicles and methods of making and using the same
CN107995927B (zh) 2013-06-17 2021-07-30 布罗德研究所有限公司 用于肝靶向和治疗的crispr-cas系统、载体和组合物的递送与用途
EP3011035B1 (en) 2013-06-17 2020-05-13 The Broad Institute, Inc. Assay for quantitative evaluation of target site cleavage by one or more crispr-cas guide sequences
JP6738729B2 (ja) 2013-06-17 2020-08-12 ザ・ブロード・インスティテュート・インコーポレイテッド 分裂終了細胞の疾患および障害をターゲティングおよびモデリングするための系、方法および組成物の送達、エンジニアリングおよび最適化
CA2915795C (en) 2013-06-17 2021-07-13 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components
EP3725885A1 (en) 2013-06-17 2020-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
EP3674411A1 (en) 2013-06-17 2020-07-01 The Broad Institute, Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
CN105492611A (zh) 2013-06-17 2016-04-13 布罗德研究所有限公司 用于序列操纵的优化的crispr-cas双切口酶系统、方法以及组合物
CN104239331B (zh) 2013-06-19 2018-10-09 阿里巴巴集团控股有限公司 一种用于实现评论搜索引擎排序的方法和装置
US10011850B2 (en) 2013-06-21 2018-07-03 The General Hospital Corporation Using RNA-guided FokI Nucleases (RFNs) to increase specificity for RNA-Guided Genome Editing
EP3019595A4 (en) 2013-07-09 2016-11-30 THERAPEUTIC USES OF A GENERIC CHANGE WITH CRISPR / CAS SYSTEMS
SG10201800213VA (en) 2013-07-10 2018-02-27 Harvard College Orthogonal cas9 proteins for rna-guided gene regulation and editing
DK3019619T3 (da) 2013-07-11 2021-10-11 Modernatx Inc Sammensætninger, der omfatter syntetiske polynukleotider, som koder for crispr-beslægtede proteiner, og syntetiske sgrna'er, og anvendelsesfremgangsmåder
WO2015021426A1 (en) 2013-08-09 2015-02-12 Sage Labs, Inc. A crispr/cas system-based novel fusion protein and its application in genome editing
PL3033184T3 (pl) 2013-08-16 2021-09-20 Massachusetts Institute Of Technology Selektywne dostarczanie materiału do komórek
EP3611268A1 (en) 2013-08-22 2020-02-19 E. I. du Pont de Nemours and Company Plant genome modification using guide rna/cas endonuclease systems and methods of use
GB201315321D0 (en) 2013-08-28 2013-10-09 Koninklijke Nederlandse Akademie Van Wetenschappen Transduction Buffer
WO2015034872A2 (en) 2013-09-05 2015-03-12 Massachusetts Institute Of Technology Tuning microbial populations with programmable nucleases
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
EP3988649B1 (en) 2013-09-18 2024-11-27 Kymab Limited Methods, cells and organisms
US20160237455A1 (en) 2013-09-27 2016-08-18 Editas Medicine, Inc. Crispr-related methods and compositions
US10822606B2 (en) 2013-09-27 2020-11-03 The Regents Of The University Of California Optimized small guide RNAs and methods of use
JP5774657B2 (ja) 2013-10-04 2015-09-09 国立大学法人京都大学 エレクトロポレーションを利用した哺乳類の遺伝子改変方法
WO2015052231A2 (en) 2013-10-08 2015-04-16 Technical University Of Denmark Multiplex editing system
WO2015054507A1 (en) 2013-10-10 2015-04-16 Pronutria, Inc. Nutritive polypeptide production systems, and methods of manufacture and use thereof
WO2015065964A1 (en) 2013-10-28 2015-05-07 The Broad Institute Inc. Functional genomics using crispr-cas systems, compositions, methods, screens and applications thereof
US9850497B2 (en) 2013-11-04 2017-12-26 Regents Of The University Of Minnesota Gene targeting methods and tools
KR20230054509A (ko) 2013-11-07 2023-04-24 에디타스 메디신, 인코포레이티드 지배적인 gRNA를 이용하는 CRISPR-관련 방법 및 조성물
DK3492593T3 (da) 2013-11-13 2021-11-08 Childrens Medical Center Nukleasemedieret regulering af genekspression
US20160298096A1 (en) 2013-11-18 2016-10-13 Crispr Therapeutics Ag Crispr-cas system materials and methods
WO2015075195A1 (en) 2013-11-22 2015-05-28 Cellectis Method of engineering chemotherapy drug resistant t-cells for immunotherapy
CA2928635C (en) 2013-11-28 2022-06-21 Horizon Genomics Gmbh Somatic haploid human cell line
BR112016013201B1 (pt) 2013-12-12 2023-01-31 The Broad Institute, Inc. Uso de uma composição compreendendo um sistema crispr-cas no tratamento de uma doença genética ocular
EP3080266B1 (en) 2013-12-12 2021-02-03 The Regents of The University of California Methods and compositions for modifying a single stranded target nucleic acid
JP2017503485A (ja) 2013-12-12 2017-02-02 ザ・ブロード・インスティテュート・インコーポレイテッド 遺伝子産物の発現、構造情報、及び誘導性モジュラーｃａｓ酵素を変更するためのｃｒｉｓｐｒ−ｃａｓ系並びに方法
SG10201804973TA (en) 2013-12-12 2018-07-30 Broad Inst Inc Compositions and Methods of Use of Crispr-Cas Systems in Nucleotide Repeat Disorders
EP3080259B1 (en) 2013-12-12 2023-02-01 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
EP3540051B1 (en) 2013-12-12 2022-08-17 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for hsv and viral diseases and disorders.
EP3079726B1 (en) 2013-12-12 2018-12-05 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components
US20150191744A1 (en) 2013-12-17 2015-07-09 University Of Massachusetts Cas9 effector-mediated regulation of transcription, differentiation and gene editing/labeling
AU2014368982B2 (en) 2013-12-19 2021-03-25 Amyris, Inc. Methods for genomic integration
PT3083556T (pt) 2013-12-19 2020-03-05 Novartis Ag Lípidos e composições lipídicas para a entrega de agentes ativos
WO2015095346A1 (en) 2013-12-19 2015-06-25 Novartis Ag Lipids and lipid compositions for the delivery of active agents
CA2935032C (en) 2013-12-26 2024-01-23 The General Hospital Corporation Multiplex guide rnas
US9963689B2 (en) 2013-12-31 2018-05-08 The Regents Of The University Of California Cas9 crystals and methods of use thereof
AU2015204784B2 (en) 2014-01-08 2021-01-28 President And Fellows Of Harvard College RNA-guided gene drives
EP3097212A4 (en) 2014-01-24 2017-10-04 North Carolina State University Methods and compositions for sequences guiding cas9 targeting
US10072066B2 (en) 2014-02-03 2018-09-11 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a beta thalessemia
US10287590B2 (en) 2014-02-12 2019-05-14 Dna2.0, Inc. Methods for generating libraries with co-varying regions of polynuleotides for genome modification
WO2015122967A1 (en) 2014-02-13 2015-08-20 Clontech Laboratories, Inc. Methods of depleting a target molecule from an initial collection of nucleic acids, and compositions and kits for practicing the same
KR20160130392A (ko) 2014-02-18 2016-11-11 듀크 유니버시티 바이러스 복제의 불활성화를 위한 조성물 및 그의 제조 및 사용 방법
US11186843B2 (en) 2014-02-27 2021-11-30 Monsanto Technology Llc Compositions and methods for site directed genomic modification
WO2015134812A1 (en) 2014-03-05 2015-09-11 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
EP3553176A1 (en) 2014-03-10 2019-10-16 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)
EP3126498A4 (en) 2014-03-20 2017-08-23 Université Laval Crispr-based methods and products for increasing frataxin levels and uses thereof
PL3122766T3 (pl) 2014-03-24 2021-09-13 IMMCO Diagnostics, Inc. Ulepszone wykrywanie i diagnostyka przeciwciał przeciwjądrowych dla układowych i nieukładowych zaburzeń autoimmunologicznych
CA2943622A1 (en) 2014-03-25 2015-10-01 Editas Medicine Inc. Crispr/cas-related methods and compositions for treating hiv infection and aids
EP3122880B1 (en) 2014-03-26 2021-05-05 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
WO2015148860A1 (en) 2014-03-26 2015-10-01 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating beta-thalassemia
WO2015152305A1 (ja) 2014-03-31 2015-10-08 株式会社サイエンス・ラスター 造血細胞の増殖ペプチドおよびその用途
WO2015153791A1 (en) 2014-04-01 2015-10-08 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating herpes simplex virus type 2 (hsv-2)
WO2015153789A1 (en) 2014-04-01 2015-10-08 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating herpes simplex virus type 1 (hsv-1)
EP3540061A1 (en) 2014-04-02 2019-09-18 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating primary open angle glaucoma
WO2015153940A1 (en) 2014-04-03 2015-10-08 Massachusetts Institute Of Technology Methods and compositions for the production of guide rna
EP3556858A3 (en) 2014-04-09 2020-01-22 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating cystic fibrosis
WO2015159068A1 (en) 2014-04-14 2015-10-22 Nemesis Bioscience Ltd Therapeutic
WO2015161276A2 (en) 2014-04-18 2015-10-22 Editas Medicine, Inc. Crispr-cas-related methods, compositions and components for cancer immunotherapy
SG10201809290SA (en) 2014-04-25 2019-01-30 Childrens Medical Ct Corp Compositions and Methods to Treating Hemoglobinopathies
GB201407852D0 (en) 2014-05-02 2014-06-18 Iontas Ltd Preparation of libraries od protein variants expressed in eukaryotic cells and use for selecting binding molecules
EP3140403A4 (en) 2014-05-09 2017-12-20 Université Laval Prevention and treatment of alzheimer's disease by genome editing using the crispr/cas system
WO2015173436A1 (en) 2014-05-16 2015-11-19 Vrije Universiteit Brussel Genetic correction of myotonic dystrophy type 1
JP2017517256A (ja) 2014-05-20 2017-06-29 リージェンツ オブ ザ ユニバーシティ オブ ミネソタ 遺伝子配列を編集する方法
EP3152319A4 (en) 2014-06-05 2017-12-27 Sangamo BioSciences, Inc. Methods and compositions for nuclease design
BR112016028564A2 (pt) 2014-06-06 2018-01-30 Regeneron Pharma método para modificar um locus-alvo em uma célula.
WO2015191693A2 (en) 2014-06-10 2015-12-17 Massachusetts Institute Of Technology Method for gene editing
WO2015191911A2 (en) 2014-06-12 2015-12-17 Clontech Laboratories, Inc. Protein enriched microvesicles and methods of making and using the same
MX384887B (es) 2014-06-23 2025-03-14 Regeneron Pharma Ensamblaje de adn mediado por nucleasa.
WO2015200555A2 (en) 2014-06-25 2015-12-30 Caribou Biosciences, Inc. Rna modification to engineer cas9 activity
US20150376586A1 (en) 2014-06-25 2015-12-31 Caribou Biosciences, Inc. RNA Modification to Engineer Cas9 Activity
SG10201911411YA (en) 2014-06-26 2020-02-27 Regeneron Pharma Methods and compositions for targeted genetic modifications and methods of use
US10017770B2 (en) 2014-07-03 2018-07-10 Ut-Battelle, Llc TNT cloning system
BR112017000621B1 (pt) 2014-07-11 2024-03-12 Pioneer Hi-Bred International, Inc Método para melhorar um traço agronômico de uma planta de milho ou de soja
US11254933B2 (en) 2014-07-14 2022-02-22 The Regents Of The University Of California CRISPR/Cas transcriptional modulation
US10975406B2 (en) 2014-07-18 2021-04-13 Massachusetts Institute Of Technology Directed endonucleases for repeatable nucleic acid cleavage
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
KR102500531B1 (ko) 2014-09-04 2023-02-17 메모리얼 슬로안 케터링 캔서 센터 헤모글로빈병증 치료용 글로빈 유전자 치료법
CN106795488B (zh) 2014-09-16 2021-03-30 桑格摩治疗股份有限公司 用于造血干细胞中核酸酶介导的基因组工程化和校正的方法和组合物
WO2016049258A2 (en) 2014-09-25 2016-03-31 The Broad Institute Inc. Functional screening with optimized functional crispr-cas systems
EP3204496A1 (en) 2014-10-10 2017-08-16 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
AU2015342749B2 (en) 2014-11-07 2022-01-27 Editas Medicine, Inc. Methods for improving CRISPR/Cas-mediated genome-editing
WO2016094872A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Dead guides for crispr transcription factors
WO2016094880A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Delivery, use and therapeutic applications of crispr systems and compositions for genome editing as to hematopoietic stem cells (hscs)
EP3237615B2 (en) 2014-12-24 2023-07-26 The Broad Institute, Inc. Crispr having or associated with destabilization domains
WO2016103233A2 (en) * 2014-12-24 2016-06-30 Dana-Farber Cancer Institute, Inc. Systems and methods for genome modification and regulation
CN107429263A (zh) 2015-01-15 2017-12-01 斯坦福大学托管董事会 调控基因组编辑的方法
EP3247366A4 (en) 2015-01-21 2018-10-31 Sangamo Therapeutics, Inc. Methods and compositions for identification of highly specific nucleases
JP6734283B2 (ja) 2015-01-21 2020-08-05 フレッド ハッチンソン キャンサー リサーチ センター 遺伝子治療用ポイントオブケア及び／又はポータブルプラットフォーム
EP3247409B1 (en) 2015-01-21 2020-11-04 Cornell University Viral vectors for prophylaxis and therapy of hemoglobinopathies
EP3256487A4 (en) 2015-02-09 2018-07-18 Duke University Compositions and methods for epigenome editing
EP3256170B1 (en) 2015-02-13 2020-09-23 University of Massachusetts Compositions and methods for transient delivery of nucleases
US20180271891A1 (en) 2015-03-11 2018-09-27 The Broad Institute Inc. Selective treatment of prmt5 dependent cancer
WO2016154596A1 (en) 2015-03-25 2016-09-29 Editas Medicine, Inc. Crispr/cas-related methods, compositions and components
EP3274453B1 (en) 2015-03-26 2021-01-27 Editas Medicine, Inc. Crispr/cas-mediated gene conversion
ES2764457T3 (es) 2015-03-31 2020-06-03 Glycotope Gmbh Vectores de expresión eucariotas que incluyen elementos reglamentarios de los grupos de genes de globina
ES2884838T3 (es) 2015-04-06 2021-12-13 Univ Leland Stanford Junior ARN guía químicamente modificados para la regulación génica mediada por CRISPR/CAS
JP7288302B2 (ja) 2015-05-08 2023-06-07 ザ チルドレンズ メディカル センター コーポレーション 胎児型ヘモグロビン再誘導のための、ｂｃｌ１１ａエンハンサー機能性領域を標的とする方法
WO2016182893A1 (en) 2015-05-08 2016-11-17 Teh Broad Institute Inc. Functional genomics using crispr-cas systems for saturating mutagenesis of non-coding elements, compositions, methods, libraries and applications thereof
EP3294896A1 (en) 2015-05-11 2018-03-21 Editas Medicine, Inc. Optimized crispr/cas9 systems and methods for gene editing in stem cells
KR20170141217A (ko) 2015-05-12 2017-12-22 상가모 테라퓨틱스, 인코포레이티드 유전자 발현의 뉴클레아제-매개된 조절
WO2016201272A1 (en) 2015-06-12 2016-12-15 King Abdulaziz City For Science And Technology Method of diagnosing patients with conditions caused by mendelian mutations
WO2016205703A1 (en) 2015-06-17 2016-12-22 The Uab Research Foundation Crispr/cas9 complex for genomic editing
US9957501B2 (en) 2015-06-18 2018-05-01 Sangamo Therapeutics, Inc. Nuclease-mediated regulation of gene expression
WO2017053729A1 (en) 2015-09-25 2017-03-30 The Board Of Trustees Of The Leland Stanford Junior University Nuclease-mediated genome editing of primary cells and enrichment thereof
WO2017059241A1 (en) 2015-10-02 2017-04-06 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Lentiviral protein delivery system for rna-guided genome editing
WO2017077394A2 (en) 2015-11-04 2017-05-11 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
EA201891532A1 (ru) 2015-12-28 2019-01-31 Новартис Аг Композиции и способы лечения гемоглобинопатий
IL313038A (en) * 2016-03-14 2024-07-01 Editas Medicine Inc Methods and preparations related to CRISPR/CAS - for the treatment of diseases in the hemoglobin cell
SG11201809144XA (en) 2016-04-18 2018-11-29 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
WO2017191503A1 (en) 2016-05-05 2017-11-09 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
EP3471743B1 (en) 2016-06-17 2025-08-06 Fred Hutchinson Cancer Center Strategies to assess and/or produce cell populations with predictive engraftment potential
WO2018009506A1 (en) 2016-07-07 2018-01-11 St. Jude Children's Research Hospital Erythroid-specific promoter and method of use thereof
CN109689865A (zh) 2016-07-25 2019-04-26 蓝鸟生物公司 Bcl11a归巢核酸内切酶变体、组合物和使用方法
WO2018035423A1 (en) 2016-08-19 2018-02-22 Bluebird Bio, Inc. Genome editing enhancers
SG11201901531TA (en) 2016-08-24 2019-03-28 Sangamo Therapeutics Inc Regulation of gene expression using engineered nucleases
US10975393B2 (en) 2016-08-24 2021-04-13 Sangamo Therapeutics, Inc. Engineered target specific nucleases
JP2019532672A (ja) 2016-09-28 2019-11-14 ノバルティス アーゲー 多孔質膜系巨大分子送達システム
TW201839136A (zh) * 2017-02-06 2018-11-01 瑞士商諾華公司 治療血色素異常症之組合物及方法

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013126794A1 (en) * 2012-02-24 2013-08-29 Fred Hutchinson Cancer Research Center Compositions and methods for the treatment of hemoglobinopathies
WO2015089486A2 (en) * 2013-12-12 2015-06-18 The Broad Institute Inc. Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems
WO2016135558A2 (en) * 2015-02-23 2016-09-01 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
TRAXLER, ELIZABETH A., ET AL., A GENOME-EDITING STRATEGY TO TREAT B-HEMOGLOBINOPATHIES THAT RECAPITULATES A MUTATION ASSOCIATED WITH A BENIGN GENETIC CONDITION., 15 August 2016 (2016-08-15) *

Also Published As

Publication number Publication date
AU2018215726A1 (en) 2019-08-22
WO2018142364A1 (en) 2018-08-09
US20200102561A1 (en) 2020-04-02
AU2022200457A1 (en) 2022-05-19
MY203407A (en) 2024-06-26
EP3577223A1 (en) 2019-12-11
SG11201907056XA (en) 2019-08-27
RU2019127919A (ru) 2021-03-09
US20230118337A1 (en) 2023-04-20
PH12019501812A1 (en) 2020-09-21
BR112019016205A2 (pt) 2020-07-14
IL268406A (en) 2019-09-26
SA519402400B1 (ar) 2023-11-20
KR20240017098A (ko) 2024-02-06
CA3052275A1 (en) 2018-08-09
EA201991862A1 (ru) 2020-02-04
MX2019009361A (es) 2020-01-30
AR110962A1 (es) 2019-05-22
AU2018215726B2 (en) 2021-11-04
IL311222A (en) 2024-05-01
AU2024205089A1 (en) 2024-10-10
US11466271B2 (en) 2022-10-11
IL268406B1 (en) 2024-04-01
CN110546262B (zh) 2024-08-02
JP2020505934A (ja) 2020-02-27
JP2025134087A (ja) 2025-09-16
CN119286853A (zh) 2025-01-10
JP2023052242A (ja) 2023-04-11
RU2019127919A3 (enExample) 2021-11-03
KR20190121319A (ko) 2019-10-25
TW201839136A (zh) 2018-11-01
CN110546262A (zh) 2019-12-06
KR102630217B1 (ko) 2024-01-29

Similar Documents

Publication Publication Date Title
US20230118337A1 (en) Compositions and methods for the treatment of hemoglobinopathies
US20240002843A1 (en) Compositions and methods for the treatment of hemoglobinopathies
EP4077676A1 (en) Compositions and methods for the treatment of hemoglobinopathies
RU2834231C2 (ru) Композиции и способы, предназначенные для лечения гемоглобинопатий
HK40080440A (en) Compositions and methods for the treatment of hemoglobinopathies
US20250127809A1 (en) Compositions and methods for the treatment of hemoglobinopathies
RU2812491C2 (ru) Композиции и способы лечения гемоглобинопатий
BR122025008935A2 (pt) Molécula de grna, método para alteração de uma célula-tronco hematopoiética (hsc) ou uma célula progenitora hematopoiética (hpc), composição, usos das mesmas, e método para preparação de uma célula- tronco hematopoiética (hsc) ou uma célula progenitora hematopoiética (hpc)
BR122021012669B1 (pt) Molécula de grna, composições, sequência de ácidos nucleicos, vetor, seus respectivos usos, método de alteração de células ex vivo, uso destas, e método de preparação de população de células
BR112018013065B1 (pt) Molécula de grna, composições, sequência de ácidos nucléicos, vetor, seus respectivos usos
BR122021012712B1 (pt) Molécula de grna, composições, sequência de ácidos nucleicos, vetor, seus respectivos usos, método de alteração de células ex vivo, uso destas, e método de preparação de população de células