SG10201606959PA - Compositions and methods for the treatment of hemoglobinopathies - Google Patents

Compositions and methods for the treatment of hemoglobinopathies

Info

Publication number
SG10201606959PA
SG10201606959PA SG10201606959PA SG10201606959PA SG10201606959PA SG 10201606959P A SG10201606959P A SG 10201606959PA SG 10201606959P A SG10201606959P A SG 10201606959PA SG 10201606959P A SG10201606959P A SG 10201606959PA SG 10201606959P A SG10201606959P A SG 10201606959PA
Authority
SG
Singapore
Prior art keywords
hemoglobinopathies
compositions
treatment
methods
Prior art date
Application number
SG10201606959PA
Other languages
English (en)
Inventor
Michael A Bender
Mark T Groudine
Barry L Stoddard
Ryo Takeuchi
Original Assignee
Hutchinson Fred Cancer Res
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 Hutchinson Fred Cancer Res filed Critical Hutchinson Fred Cancer Res
Publication of SG10201606959PA publication Critical patent/SG10201606959PA/en

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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
    • 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]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • 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
    • 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
    • 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)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
SG10201606959PA 2012-02-24 2013-02-22 Compositions and methods for the treatment of hemoglobinopathies SG10201606959PA (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201261603231P 2012-02-24 2012-02-24

Publications (1)

Publication Number Publication Date
SG10201606959PA true SG10201606959PA (en) 2016-09-29

Family

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Family Applications (2)

Application Number Title Priority Date Filing Date
SG10201606959PA SG10201606959PA (en) 2012-02-24 2013-02-22 Compositions and methods for the treatment of hemoglobinopathies
SG11201405103SA SG11201405103SA (en) 2012-02-24 2013-02-22 Compositions and methods for the treatment of hemoglobinopathies

Family Applications After (1)

Application Number Title Priority Date Filing Date
SG11201405103SA SG11201405103SA (en) 2012-02-24 2013-02-22 Compositions and methods for the treatment of hemoglobinopathies

Country Status (18)

Country Link
US (2) US20150166969A1 (enExample)
EP (2) EP2836226B1 (enExample)
JP (2) JP6170080B2 (enExample)
KR (2) KR20180009383A (enExample)
CN (1) CN104284669A (enExample)
AU (2) AU2013222170B2 (enExample)
BR (1) BR112014020625A2 (enExample)
CA (1) CA2865129A1 (enExample)
DK (1) DK2836226T3 (enExample)
ES (1) ES2641840T3 (enExample)
HK (1) HK1249861A1 (enExample)
IL (2) IL234225B (enExample)
IN (1) IN2014DN07853A (enExample)
NZ (1) NZ630900A (enExample)
RU (2) RU2018109507A (enExample)
SG (2) SG10201606959PA (enExample)
WO (1) WO2013126794A1 (enExample)
ZA (1) ZA201406187B (enExample)

Families Citing this family (189)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3613852A3 (en) 2011-07-22 2020-04-22 President and Fellows of Harvard College Evaluation and improvement of nuclease cleavage specificity
JP2014531908A (ja) 2011-10-14 2014-12-04 プレジデント アンド フェローズ オブ ハーバード カレッジ 構造アッセンブリによる配列決定
ES2991004T3 (es) 2011-12-22 2024-12-02 Harvard College Métodos para la detección de analitos
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
BR112014020625A2 (pt) 2012-02-24 2017-07-04 Hutchinson Fred Cancer Res polinucleotídeo, polipeptídeo, composição, célula e célula tronco editada por genoma
RS59199B1 (sr) 2012-05-25 2019-10-31 Univ California Metode i jedinjenja za rnk-upravljanu ciljanu dnk modifikaciju i za rnk- upravljanu modulaciju transkripta
AU2013308770B2 (en) 2012-08-29 2019-01-17 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition
JP6517143B2 (ja) * 2012-10-23 2019-05-22 ツールゲン インコーポレイテッド 標的ｄｎａに特異的なガイドｒｎａおよびｃａｓタンパク質コード核酸またはｃａｓタンパク質を含む、標的ｄｎａを切断するための組成物、ならびにその使用
DK2925864T3 (en) 2012-11-27 2019-02-11 Childrens Medical Ct Corp DIRECTIONAL TARGETING OF DISTANT BCL11A CONTROLS FOR FETAL HEMOGLOBIN REINUCTION
KR102479178B1 (ko) 2012-12-06 2022-12-19 시그마-알드리치 컴퍼니., 엘엘씨 Crispr-기초된 유전체 변형과 조절
EP4299741A3 (en) 2012-12-12 2024-02-28 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
JP6700788B2 (ja) 2012-12-17 2020-05-27 プレジデント アンド フェローズ オブ ハーバード カレッジ Ｒｎａ誘導性ヒトゲノム改変
EP2971184B1 (en) 2013-03-12 2019-04-17 President and Fellows of Harvard College Method of generating a three-dimensional nucleic acid containing matrix
NZ712727A (en) 2013-03-14 2017-05-26 Caribou Biosciences Inc Compositions and methods of nucleic acid-targeting nucleic acids
WO2014204578A1 (en) 2013-06-21 2014-12-24 The General Hospital Corporation Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing
KR102271292B1 (ko) 2013-03-15 2021-07-02 더 제너럴 하스피탈 코포레이션 Rna-안내 게놈 편집의 특이성을 증가시키기 위한 rna-안내 foki 뉴클레아제(rfn)의 용도
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
JP2016522679A (ja) 2013-04-04 2016-08-04 プレジデント アンド フェローズ オブ ハーバード カレッジ ＣＲＩＳＰＲ／Ｃａｓ系を用いたゲノム編集の治療的使用
HK1223401A1 (zh) * 2013-05-15 2017-07-28 桑格摩生物科学股份有限公司 用於治疗遗传病状的方法和组合物
US9267135B2 (en) 2013-06-04 2016-02-23 President And Fellows Of Harvard College RNA-guided transcriptional regulation
EP3003392B1 (en) 2013-06-04 2019-10-23 President and Fellows of Harvard College Rna-guideded transcriptional regulation
BR122021009076B1 (pt) 2013-06-17 2024-02-15 The Broad Institute Inc. Vetor viral contendo molécula(s) de ácido nucleico heterólogo, composição, uso e métodos do mesmo
BR112015031608A2 (pt) 2013-06-17 2017-08-22 Massachusetts Inst Technology Aplicação e uso dos sistemas crispr-cas, vetores e composições para direcionamento e terapia hepáticos
ES2767318T3 (es) 2013-06-17 2020-06-17 Broad Inst Inc Suministro, modificación y optimización de sistemas, métodos y composiciones para generar modelos y actuar sobre enfermedades y trastornos de células posmitóticas
JP2016528890A (ja) * 2013-07-09 2016-09-23 プレジデント アンド フェローズ オブ ハーバード カレッジ ＣＲＩＳＰＲ／Ｃａｓ系を用いるゲノム編集の治療用の使用
AU2014287397B2 (en) 2013-07-10 2019-10-10 President And Fellows Of Harvard College Orthogonal Cas9 proteins for RNA-guided gene regulation and editing
US10563225B2 (en) 2013-07-26 2020-02-18 President And Fellows Of Harvard College Genome engineering
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
CN111218447B (zh) 2013-11-07 2024-10-11 爱迪塔斯医药有限公司 使用统治型gRNA的CRISPR相关方法和组合物
HRP20211706T1 (hr) * 2013-11-13 2022-02-04 The Children's Medical Center Corporation Nukleazom posredovana regulacija ekspresije gena
US20160298096A1 (en) * 2013-11-18 2016-10-13 Crispr Therapeutics Ag Crispr-cas system materials and methods
US9074199B1 (en) 2013-11-19 2015-07-07 President And Fellows Of Harvard College Mutant Cas9 proteins
US10787684B2 (en) 2013-11-19 2020-09-29 President And Fellows Of Harvard College Large gene excision and insertion
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
WO2015089462A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for genome editing
SG10201804977UA (en) 2013-12-12 2018-07-30 Broad Inst Inc Delivery, Use and Therapeutic Applications of the Crispr-Cas Systems and Compositions for Targeting Disorders and Diseases Using Particle Delivery Components
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
EP3105325B2 (en) 2014-02-13 2024-10-23 Takara Bio USA, Inc. Methods of depleting a target molecule from an initial collection of nucleic acids, and compositions and kits for practicing the same
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
US11339437B2 (en) 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
ES2745769T3 (es) 2014-03-10 2020-03-03 Editas Medicine Inc Procedimientos y composiciones relacionados con CRISPR/CAS para tratar la amaurosis congénita de Leber 10 (LCA10)
WO2015148860A1 (en) * 2014-03-26 2015-10-01 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating beta-thalassemia
WO2015148863A2 (en) * 2014-03-26 2015-10-01 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
US20170173083A1 (en) * 2014-03-26 2017-06-22 The Brigham And Women's Hospital, Inc. Compositions and methods for ex vivo expansion of human hematopoietic stem/progenitor cells
AU2015249381B2 (en) 2014-04-25 2020-04-30 Children's Medical Center Corporation Compositions and methods to treating hemoglobinopathies
WO2016007839A1 (en) 2014-07-11 2016-01-14 President And Fellows Of Harvard College Methods for high-throughput labelling and detection of biological features in situ using microscopy
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
WO2016025759A1 (en) * 2014-08-14 2016-02-18 Shen Yuelei Dna knock-in system
KR20170045344A (ko) 2014-09-07 2017-04-26 셀렉타 바이오사이언시즈, 인크. 항-바이러스 전달 벡터 면역 반응을 약화시키기 위한 방법 및 조성물
KR20170054445A (ko) * 2014-09-16 2017-05-17 상가모 테라퓨틱스, 인코포레이티드 뉴클레아제-매개된 게놈 엔지니어링 및 조혈 줄기세포에서의 교정을 위한 방법 및 조성물
CN113016623A (zh) 2014-09-26 2021-06-25 谱赛科美国股份有限公司 用于甜菊的单核苷酸多态性(snp)标志物
EP3215623A4 (en) 2014-11-06 2018-09-26 President and Fellows of Harvard College Cells lacking b2m surface expression and methods for allogeneic administration of such cells
AU2015355546B2 (en) 2014-12-03 2021-10-14 Agilent Technologies, Inc. Guide RNA with chemical modifications
WO2016094874A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Escorted and functionalized guides for crispr-cas systems
EP3985115A1 (en) 2014-12-12 2022-04-20 The Broad Institute, Inc. Protected guide rnas (pgrnas)
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)
CA2970370A1 (en) 2014-12-24 2016-06-30 Massachusetts Institute Of Technology Crispr having or associated with destabilization domains
US20180002379A1 (en) * 2015-01-21 2018-01-04 Sangamo Therapeutics, Inc. Methods and compositions for identification of highly specific nucleases
CN114058582A (zh) 2015-01-26 2022-02-18 菲特治疗公司 用于诱导造血细胞分化的方法和组合物
US20180030438A1 (en) * 2015-02-23 2018-02-01 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
US12129471B2 (en) 2015-02-23 2024-10-29 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of human genetic diseases including hemoglobinopathies
WO2016164356A1 (en) 2015-04-06 2016-10-13 The Board Of Trustees Of The Leland Stanford Junior University Chemically modified guide rnas for crispr/cas-mediated gene regulation
US10155938B2 (en) * 2015-04-14 2018-12-18 City Of Hope Coexpression of CAS9 and TREX2 for targeted mutagenesis
CN104805118A (zh) * 2015-04-22 2015-07-29 扬州大学 一种苏禽黄鸡胚胎干细胞特定基因进行靶向敲除方法
EP3292219B9 (en) * 2015-05-04 2022-05-18 Ramot at Tel-Aviv University Ltd. Methods and kits for fragmenting dna
AU2016261600B2 (en) 2015-05-08 2021-09-23 President And Fellows Of Harvard College Universal donor stem cells and related methods
WO2016182917A1 (en) * 2015-05-08 2016-11-17 Children's Medical Center Corporation Targeting bcl11a enhancer functional regions for fetal hemoglobin reinduction
EP3294896A1 (en) * 2015-05-11 2018-03-21 Editas Medicine, Inc. Optimized crispr/cas9 systems and methods for gene editing in stem cells
MX382223B (es) * 2015-05-12 2025-03-13 Sangamo Therapeutics Inc Regulacion de expresion genica mediada por nucleasa.
JP7396783B2 (ja) 2015-06-09 2023-12-12 エディタス・メディシン、インコーポレイテッド 移植を改善するためのｃｒｉｓｐｒ／ｃａｓ関連方法および組成物
WO2016205680A1 (en) 2015-06-17 2016-12-22 The Uab Research Foundation Crispr/cas9 complex for introducing a functional polypeptide into cells of blood cell lineage
US11643668B2 (en) 2015-06-17 2023-05-09 The Uab Research Foundation CRISPR/Cas9 complex for genomic editing
CA3012607A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Crispr enzymes and systems
HK1257676A1 (zh) 2015-08-28 2019-10-25 The General Hospital Corporation 工程化 crispr-cas 9 核酸酶
US9512446B1 (en) 2015-08-28 2016-12-06 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US9926546B2 (en) 2015-08-28 2018-03-27 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US9897378B2 (en) 2015-10-08 2018-02-20 Nyc Designed Inspirations Llc Cosmetic makeup sponge/blender container
US12043852B2 (en) 2015-10-23 2024-07-23 President And Fellows Of Harvard College Evolved Cas9 proteins for gene editing
EP3368052B1 (en) * 2015-10-27 2021-12-08 Children's Hospital Medical Center Use of mapk inhibitors to reduce loss of hematopoietic stem cells during ex vivo culture and genetic manipulation
JP6882282B2 (ja) 2015-11-03 2021-06-02 プレジデント アンド フェローズ オブ ハーバード カレッジ 三次元核酸含有マトリックスの立体撮像のための方法と装置
EP3371301A4 (en) 2015-11-04 2019-06-26 Fate Therapeutics, Inc. METHOD AND COMPOSITIONS FOR INDUCING THE DIFFERENTIATION OF HEMATOPOIETIC CELLS
CN115806940A (zh) 2015-11-04 2023-03-17 菲特治疗公司 多能细胞的基因组工程改造
ES2942309T3 (es) * 2015-11-04 2023-05-31 Vertex Pharma Materiales y métodos para el tratamiento de hemoglobinopatías
NZ783532A (en) * 2015-12-28 2025-09-26 Novartis Ag Compositions and methods for the treatment of hemoglobinopathies
JP2019508063A (ja) 2016-01-27 2019-03-28 オンコラス， インコーポレイテッド 腫瘍溶解性ウイルスベクター及びその使用
WO2017147278A1 (en) 2016-02-25 2017-08-31 The Children's Medical Center Corporation Customized class switch of immunoglobulin genes in lymphoma and hybridoma by crispr/cas9 technology
US20200255857A1 (en) * 2016-03-14 2020-08-13 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating beta hemoglobinopathies
EP3219799A1 (en) 2016-03-17 2017-09-20 IMBA-Institut für Molekulare Biotechnologie GmbH Conditional crispr sgrna expression
SI3445388T1 (sl) * 2016-04-18 2024-07-31 Vertex Pharmaceuticals Incorporated Materiali in metode za zdravljenje hemoglobinopatij
EP3449016B1 (en) 2016-04-25 2025-07-23 President and Fellows of Harvard College Hybridization chain reaction methods for in situ molecular detection
WO2017191503A1 (en) * 2016-05-05 2017-11-09 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
US10767175B2 (en) 2016-06-08 2020-09-08 Agilent Technologies, Inc. High specificity genome editing using chemically modified guide RNAs
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
GB2568182A (en) 2016-08-03 2019-05-08 Harvard College Adenosine nucleobase editors and uses thereof
US11078481B1 (en) 2016-08-03 2021-08-03 KSQ Therapeutics, Inc. Methods for screening for cancer targets
WO2018031683A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
CN106244555A (zh) * 2016-08-23 2016-12-21 广州医科大学附属第三医院 一种提高基因打靶的效率的方法及β‑球蛋白基因位点的碱基原位修复方法
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
US11078483B1 (en) 2016-09-02 2021-08-03 KSQ Therapeutics, Inc. Methods for measuring and improving CRISPR reagent function
IL265045B2 (en) * 2016-09-08 2023-09-01 Bluebird Bio Inc Variants of endonuclease pd-1, compositions and methods of use
GB2573062A (en) 2016-10-14 2019-10-23 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
CA3050514A1 (en) 2017-01-27 2018-08-02 Children's Hospital Medical Center Methods of enhancing engraftment activity of hematopoietic stem cells
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
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
WO2018165631A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Cancer vaccine
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
EP3596217A1 (en) 2017-03-14 2020-01-22 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
US11268082B2 (en) 2017-03-23 2022-03-08 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable DNA binding proteins
KR102746223B1 (ko) 2017-04-20 2024-12-27 이제네시스, 인크. 유전자 변형 동물의 생성 방법
CN110799525A (zh) 2017-04-21 2020-02-14 通用医疗公司 具有改变的PAM特异性的CPF1(CAS12a)的变体
EP3615664A4 (en) * 2017-04-24 2021-01-27 Seattle Children's Hospital (DBA Seattle Children's Research Institute) HOMOLOGY-DIRECTED REPAIR COMPOSITIONS FOR THE TREATMENT OF HEMOGLOBINOPATHIES
EP3622070A2 (en) 2017-05-10 2020-03-18 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
WO2018218135A1 (en) 2017-05-25 2018-11-29 The Children's Medical Center Corporation Bcl11a guide delivery
JP2020521451A (ja) 2017-05-25 2020-07-27 ザ ジェネラル ホスピタル コーポレイション 望ましくないオフターゲット塩基エディター脱アミノ化を制限するためのスプリットデアミナーゼの使用
EP3635120A1 (en) * 2017-06-02 2020-04-15 Institut National de la Santé et de la Recherche Médicale (INSERM) Recombinant lentiviral vector for stem cell-based gene therapy of sickle cell disorder
WO2019014564A1 (en) 2017-07-14 2019-01-17 Editas Medicine, Inc. SYSTEMS AND METHODS OF TARGETED INTEGRATION AND GENOME EDITING AND DETECTION THEREOF WITH INTEGRATED PRIMING SITES
WO2019023483A1 (en) 2017-07-26 2019-01-31 Oncorus, Inc. ONCOLYTIC VIRAL VECTORS AND USES THEREOF
JP2020534795A (ja) 2017-07-28 2020-12-03 プレジデント アンド フェローズ オブ ハーバード カレッジ ファージによって支援される連続的進化（ｐａｃｅ）を用いて塩基編集因子を進化させるための方法および組成物
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
US11779654B2 (en) 2017-10-04 2023-10-10 2Seventy Bio, Inc. PCSK9 endonuclease variants, compositions, and methods of use
BR112020007157A2 (pt) 2017-10-13 2020-09-24 Selecta Biosciences, Inc. métodos e composições para a atenuação de respostas de igm antivetor de transferência viral
CA3082251A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
WO2019081982A1 (en) * 2017-10-26 2019-05-02 Crispr Therapeutics Ag SUBSTANCES AND METHODS FOR THE TREATMENT OF HEMOGLOBINOPATHIES
CN109722415B (zh) * 2017-10-27 2021-01-26 博雅辑因（北京）生物科技有限公司 一种造血干细胞的培养组合物、培养基以及造血干细胞的培养方法
SG11202005147WA (en) 2017-12-05 2020-06-29 Vertex Pharma Crispr-cas9 modified cd34+ human hematopoietic stem and progenitor cells and uses thereof
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
US11268077B2 (en) 2018-02-05 2022-03-08 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
EP3749767A1 (en) 2018-02-05 2020-12-16 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
CN112384204B (zh) 2018-02-26 2023-03-14 安托瑞斯公司 致耐受性脂质体及其使用方法
CA3093702A1 (en) 2018-03-14 2019-09-19 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
US10968257B2 (en) 2018-04-03 2021-04-06 The Broad Institute, Inc. Target recognition motifs and uses thereof
EP3781677A4 (en) 2018-04-16 2022-01-19 University of Massachusetts COMPOSITIONS AND METHODS FOR IMPROVED GENE EDITTING
JP2021521838A (ja) * 2018-04-27 2021-08-30 シアトル チルドレンズ ホスピタル （ディービーエイ シアトル チルドレンズ リサーチ インスティテュート） ブルトン型チロシンキナーゼに対するｔａｌｅｎベースのおよびｃｒｉｓｐｒ／ｃａｓベースのゲノム編集
US12350284B2 (en) 2018-05-02 2025-07-08 The Children's Medical Center Corporation BCL11A microRNAs for treating hemoglobinopathies
EP3799603A4 (en) 2018-05-11 2022-03-02 Beam Therapeutics, Inc. METHODS OF PATHOGENIC AMINO ACID SUBSTITUTION USING PROGRAMMABLE BASE EDITOR SYSTEMS
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
EP4365194A3 (en) 2018-06-14 2024-07-24 Regeneron Pharmaceuticals, Inc. Cd79a chimeric antigen receptors
WO2020041217A1 (en) 2018-08-18 2020-02-27 President And Fellows Of Harvard College In situ gene editing
WO2020041380A1 (en) 2018-08-20 2020-02-27 The Broad Institute, Inc. Methods and compositions for optochemical control of crispr-cas9
US20200102563A1 (en) * 2018-10-02 2020-04-02 Exosome Therapeutics, Inc. Exosome loaded therapeutics for treating sickle cell disease
US12312584B2 (en) 2018-10-02 2025-05-27 Exosome Therapeutics, Inc. cGMP exosome loaded therapeutics for treating sickle cell disease
WO2020076976A1 (en) 2018-10-10 2020-04-16 Readcoor, Inc. Three-dimensional spatial molecular indexing
WO2020092453A1 (en) 2018-10-29 2020-05-07 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
CA3122278A1 (en) 2018-12-10 2020-06-18 Bluebird Bio, Inc. Homing endonuclease variants
US12264323B2 (en) 2018-12-17 2025-04-01 The Broad Institute, Inc. CRISPR CPF1 direct repeat variants
CN113544266A (zh) 2018-12-17 2021-10-22 博德研究所 Crispr相关转座酶系统和其使用方法
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
US11946040B2 (en) 2019-02-04 2024-04-02 The General Hospital Corporation Adenine DNA base editor variants with reduced off-target RNA editing
CN116497067B (zh) 2019-02-13 2025-01-24 比姆医疗股份有限公司 治疗血红素病变的组合物和方法
AU2020240109A1 (en) 2019-03-19 2021-09-30 President And Fellows Of Harvard College 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
CN110042124A (zh) * 2019-04-25 2019-07-23 国家卫生健康委科学技术研究所 基因组碱基编辑增加人红细胞中胎儿血红蛋白水平的试剂盒及应用
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
KR20220002565A (ko) * 2019-04-30 2022-01-06 에디진 인크. 이상헤모글로빈증 치료의 효과를 예측하는 방법
AU2020284555A1 (en) 2019-05-28 2021-12-23 Selecta Biosciences, Inc. Methods and compositions for attenuated anti-viral transfer vector immune response
EP3990650A4 (en) * 2019-06-28 2024-03-20 ASC Therapeutics Inc. METHODS AND COMPOSITIONS FOR TREATING THALASSEMIA AND SICKLE CELL ANEMIA
CA3153197A1 (en) 2019-10-03 2021-04-08 Ryan T. Gill Crispr systems with engineered dual guide nucleic acids
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
EP4045637A4 (en) 2019-10-16 2023-11-22 The Broad Institute, Inc. TECHNICAL COMPOSITIONS FOR MUSCLE BUILDING
BR112022007950A2 (pt) * 2019-11-15 2022-07-12 Univ Leland Stanford Junior Integração direcionada no locus de alfa-globina em células-tronco e células progenitoras hematopoiéticas humanas
CN112979823B (zh) * 2019-12-18 2022-04-08 华东师范大学 一种用于治疗和/或预防β血红蛋白病的产品及融合蛋白
EP4127139A1 (en) 2020-03-27 2023-02-08 Mendus B.V. Ex vivo use of modified cells of leukemic origin for enhancing the efficacy of adoptive cell therapy
BR112022022603A2 (pt) 2020-05-08 2023-01-17 Broad Inst Inc Métodos e composições para edição simultânea de ambas as fitas de sequência alvo de nucleotídeos de fita dupla
US20230279438A1 (en) * 2020-05-13 2023-09-07 Inserm (Institut National De La Santé Et La Recherche Médicale) Base editing approaches for the treatment of betahemoglobinopathies
AU2021285983A1 (en) * 2020-06-04 2023-02-02 Emendobio Inc. Novel OMNI-59, 61, 67, 76, 79, 80, 81, and 82 CRISPR nucleases
KR20230033647A (ko) 2020-06-30 2023-03-08 멘두스 비.브이. 난소암 백신에서 백혈병 유래 세포의 용도
US20220049303A1 (en) 2020-08-17 2022-02-17 Readcoor, Llc Methods and systems for spatial mapping of genetic variants
AU2021338062A1 (en) * 2020-09-04 2023-05-04 Hiroshima University Method for editing target dna, method for producing cell having edited target dna, and dna edition system for use in said methods
CA3202889A1 (en) * 2020-12-21 2022-06-30 Jordan JARJOUR Compositions and methods for site-directed mutagenesis
CA3209863A1 (en) 2021-02-25 2022-09-01 Andrea BARGHETTI Compositions and methods for targeting, editing, or modifying genes
JP2024510989A (ja) 2021-03-12 2024-03-12 メンドゥス・ベスローテン・フェンノートシャップ ワクチン接種方法及びｃｄ４７遮断薬の使用
WO2022256448A2 (en) 2021-06-01 2022-12-08 Artisan Development Labs, Inc. Compositions and methods for targeting, editing, or modifying genes
CN113481184A (zh) * 2021-08-06 2021-10-08 北京大学 融合蛋白以及其使用方法
WO2023039586A1 (en) 2021-09-10 2023-03-16 Agilent Technologies, Inc. Guide rnas with chemical modification for prime editing
WO2023064367A1 (en) 2021-10-12 2023-04-20 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector igm responses
US20230279442A1 (en) 2021-12-15 2023-09-07 Versitech Limited Engineered cas9-nucleases and method of use thereof
WO2023167882A1 (en) 2022-03-01 2023-09-07 Artisan Development Labs, Inc. Composition and methods for transgene insertion
US20230357437A1 (en) 2022-03-09 2023-11-09 Selecta Biosciences, Inc. Immunosuppressants in combination with anti-igm agents and related dosing
CN114457119B (zh) * 2022-04-11 2022-08-12 中吉智药(南京)生物技术有限公司 慢病毒载体在制备治疗β-地中海贫血药物中的应用
EP4273242A1 (en) * 2022-05-03 2023-11-08 ETH Zurich Crispr-based modification of human hbd gene
CN117427169A (zh) * 2023-10-24 2024-01-23 中国医学科学院基础医学研究所 同时破坏HBB竞争和HBG表观沉默在治疗β-血红蛋白病中的应用

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20020182673A1 (en) * 1998-05-15 2002-12-05 Genentech, Inc. IL-17 homologous polypedies and therapeutic uses thereof
JP2006518372A (ja) 2003-01-28 2006-08-10 セレクティス 脊椎動物の体組織においてエクスビボかつイントトで相同的組換えを誘発するためのメガヌクレアーゼの使用およびその応用
WO2007049095A1 (en) 2005-10-25 2007-05-03 Cellectis Laglidadg homing endonuclease variants having mutations in two functional subdomains and use thereof
WO2008010009A1 (en) 2006-07-18 2008-01-24 Cellectis Meganuclease variants cleaving a dna target sequence from a rag gene and uses thereof
CA2678526A1 (en) 2007-02-19 2008-08-28 Cellectis Laglidadg homing endonuclease variants having novel substrate specificity and use thereof
WO2009013559A1 (en) * 2007-07-23 2009-01-29 Cellectis Meganuclease variants cleaving a dna target sequence from the human hemoglobin beta gene and uses thereof
DK2334794T3 (en) * 2008-09-15 2017-02-20 Children's Medical Center Corp MODULATION OF BCL11A FOR TREATMENT OF HEMOGLOBINOPATHIES
US20140148361A1 (en) 2010-06-07 2014-05-29 Barry L. Stoddard Generation and Expression of Engineered I-ONUI Endonuclease and Its Homologues and Uses Thereof
ES2906059T3 (es) * 2011-02-28 2022-04-13 Seattle Childrens Res Institute Endonucleasas de acoplamiento con enzimas de procesamiento de extremos que impulsan la interrupción génica de alta eficiencia
BR112014020625A2 (pt) 2012-02-24 2017-07-04 Hutchinson Fred Cancer Res polinucleotídeo, polipeptídeo, composição, célula e célula tronco editada por genoma
AU2013308770B2 (en) * 2012-08-29 2019-01-17 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition

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