IL234225B - Preparations and methods for the treatment of hemoglobin diseases - Google Patents

Preparations and methods for the treatment of hemoglobin diseases

Info

Publication number
IL234225B
IL234225B IL234225A IL23422514A IL234225B IL 234225 B IL234225 B IL 234225B IL 234225 A IL234225 A IL 234225A IL 23422514 A IL23422514 A IL 23422514A IL 234225 B IL234225 B IL 234225B
Authority
IL
Israel
Prior art keywords
hemoglobinopathies
compositions
treatment
methods
Prior art date
Application number
IL234225A
Other languages
English (en)
Hebrew (he)
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 IL234225B publication Critical patent/IL234225B/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
    • 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

Landscapes

  • 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)
IL234225A 2012-02-24 2014-08-21 Preparations and methods for the treatment of hemoglobin diseases IL234225B (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201261603231P 2012-02-24 2012-02-24
PCT/US2013/027459 WO2013126794A1 (en) 2012-02-24 2013-02-22 Compositions and methods for the treatment of hemoglobinopathies

Publications (1)

Publication Number Publication Date
IL234225B true IL234225B (en) 2019-05-30

Family

ID=49006265

Family Applications (2)

Application Number Title Priority Date Filing Date
IL234225A IL234225B (en) 2012-02-24 2014-08-21 Preparations and methods for the treatment of hemoglobin diseases
IL266582A IL266582A (en) 2012-02-24 2019-05-13 Preparations and methods for the treatment of hemoglobin diseases

Family Applications After (1)

Application Number Title Priority Date Filing Date
IL266582A IL266582A (en) 2012-02-24 2019-05-13 Preparations and methods for the treatment of hemoglobin diseases

Country Status (18)

Country Link
US (2) US20150166969A1 (cg-RX-API-DMAC7.html)
EP (2) EP3272356A1 (cg-RX-API-DMAC7.html)
JP (2) JP6170080B2 (cg-RX-API-DMAC7.html)
KR (2) KR101833589B1 (cg-RX-API-DMAC7.html)
CN (1) CN104284669A (cg-RX-API-DMAC7.html)
AU (2) AU2013222170B2 (cg-RX-API-DMAC7.html)
BR (1) BR112014020625A2 (cg-RX-API-DMAC7.html)
CA (1) CA2865129A1 (cg-RX-API-DMAC7.html)
DK (1) DK2836226T3 (cg-RX-API-DMAC7.html)
ES (1) ES2641840T3 (cg-RX-API-DMAC7.html)
HK (1) HK1249861A1 (cg-RX-API-DMAC7.html)
IL (2) IL234225B (cg-RX-API-DMAC7.html)
IN (1) IN2014DN07853A (cg-RX-API-DMAC7.html)
NZ (1) NZ630900A (cg-RX-API-DMAC7.html)
RU (2) RU2018109507A (cg-RX-API-DMAC7.html)
SG (2) SG11201405103SA (cg-RX-API-DMAC7.html)
WO (1) WO2013126794A1 (cg-RX-API-DMAC7.html)
ZA (1) ZA201406187B (cg-RX-API-DMAC7.html)

Families Citing this family (189)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2853829C (en) 2011-07-22 2023-09-26 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
RU2018109507A (ru) 2012-02-24 2019-02-28 Фред Хатчинсон Кэнсер Рисерч Сентер Композиции и способы лечения гемоглобинопатии
GB2537000C (en) 2012-05-25 2019-10-09 Univ California Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
SG11201500852WA (en) 2012-08-29 2015-04-29 Sangamo Biosciences Inc Methods and compositions for treatment of a genetic condition
EP4556564A3 (en) * 2012-10-23 2025-09-03 Toolgen Incorporated Composition for cleaving a target dna comprising a guide rna specific for the target dna and cas protein-encoding nucleic acid or cas protein, and use thereof
AU2013352156B2 (en) 2012-11-27 2018-12-06 Children's Medical Center Corporation Targeting BCL11A distal regulatory elements for fetal hemoglobin reinduction
CA2891347C (en) 2012-12-06 2018-02-27 Sigma-Aldrich Co. Llc Crispr-based genome modification and regulation
IL239317B (en) 2012-12-12 2022-07-01 Broad Inst Inc Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
US9970024B2 (en) 2012-12-17 2018-05-15 President And Fellows Of Harvard College RNA-guided human genome engineering
EP3578666A1 (en) 2013-03-12 2019-12-11 President and Fellows of Harvard College Method of generating a three-dimensional nucleic acid containing matrix
AU2014235794A1 (en) 2013-03-14 2015-10-22 Caribou Biosciences, Inc. Compositions and methods of nucleic acid-targeting nucleic acids
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
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
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
CN115261411A (zh) 2013-04-04 2022-11-01 哈佛学院校长同事会 利用CRISPR/Cas系统的基因组编辑的治疗性用途
WO2014186585A2 (en) * 2013-05-15 2014-11-20 Sangamo Biosciences, Inc. Methods and compositions for treatment of a genetic condition
WO2014197568A2 (en) 2013-06-04 2014-12-11 President And Fellows Of Harvard College Rna-guideded transcriptional regulation
US20140356956A1 (en) 2013-06-04 2014-12-04 President And Fellows Of Harvard College RNA-Guided Transcriptional Regulation
EP3620524A1 (en) 2013-06-17 2020-03-11 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells
CN105793425B (zh) 2013-06-17 2021-10-26 布罗德研究所有限公司 使用病毒组分靶向障碍和疾病的crispr-cas系统和组合物的递送、用途和治疗应用
AU2014281028B2 (en) 2013-06-17 2020-09-10 Massachusetts Institute Of Technology Delivery and use of the CRISPR-Cas systems, vectors and compositions for hepatic targeting and therapy
WO2015006498A2 (en) * 2013-07-09 2015-01-15 President And Fellows Of Harvard College Therapeutic uses of genome editing with crispr/cas systems
JP6718813B2 (ja) 2013-07-10 2020-07-08 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誘導性遺伝子制御および編集のための直交性ｃａｓ９タンパク質
US10563225B2 (en) 2013-07-26 2020-02-18 President And Fellows Of Harvard College Genome engineering
US9163284B2 (en) 2013-08-09 2015-10-20 President And Fellows Of Harvard College Methods for identifying a target site of a Cas9 nuclease
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
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
WO2015073683A2 (en) * 2013-11-13 2015-05-21 Children's Medical Center Corporation Nuclease-mediated regulation of gene expression
EP3071695A2 (en) * 2013-11-18 2016-09-28 Crispr Therapeutics AG Crispr-cas system materials and methods
US10787684B2 (en) 2013-11-19 2020-09-29 President And Fellows Of Harvard College Large gene excision and insertion
US9074199B1 (en) 2013-11-19 2015-07-07 President And Fellows Of Harvard College Mutant Cas9 proteins
CN118813621A (zh) 2013-12-12 2024-10-22 布罗德研究所有限公司 用于基因组编辑的crispr-cas系统和组合物的递送、用途和治疗应用
US20150166982A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting pi3k point mutations
WO2015089419A2 (en) 2013-12-12 2015-06-18 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
US9963689B2 (en) 2013-12-31 2018-05-08 The Regents Of The University Of California Cas9 crystals and methods of use thereof
SG10201805815YA (en) 2014-01-08 2018-08-30 Harvard College Rna-guided gene drives
CA2939621C (en) 2014-02-13 2019-10-15 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
US9938521B2 (en) 2014-03-10 2018-04-10 Editas Medicine, Inc. CRISPR/CAS-related methods and compositions for treating leber's congenital amaurosis 10 (LCA10)
US11339437B2 (en) 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
US11242525B2 (en) 2014-03-26 2022-02-08 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
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
CN106794260B (zh) 2014-04-25 2021-02-23 儿童医疗中心有限公司 治疗血红蛋白病的组合物和方法
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
EP3177718B1 (en) 2014-07-30 2022-03-16 President and Fellows of Harvard College Cas9 proteins including ligand-dependent inteins
US11071289B2 (en) * 2014-08-14 2021-07-27 Biocytogen Boston Corp DNA knock-in system
IL292574A (en) 2014-09-07 2022-06-01 Selecta Biosciences Inc Methods and compositions for attenuating anti-viral transfer vector immune responses
CN113699113A (zh) * 2014-09-16 2021-11-26 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
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
KR102763527B1 (ko) 2014-12-03 2025-02-05 애질런트 테크놀로지스, 인크. 화학적 변형을 갖는 가이드 rna
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)
EP3889260A1 (en) 2014-12-12 2021-10-06 The Broad Institute, Inc. Protected guide rnas (pgrnas)
WO2016094874A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Escorted and functionalized guides for crispr-cas systems
EP3230452B1 (en) 2014-12-12 2025-06-11 The Broad Institute, Inc. Dead guides for crispr transcription factors
AU2015369725A1 (en) 2014-12-24 2017-06-29 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
AU2016211671B2 (en) 2015-01-26 2022-05-26 Fate Therapeutics, Inc. Methods and compositions for inducing hematopoietic cell differentiation
SG11201706766WA (en) 2015-02-23 2017-09-28 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
EP3262173A2 (en) 2015-02-23 2018-01-03 Crispr Therapeutics AG Materials and methods for treatment of human genetic diseases including hemoglobinopathies
CA2981715A1 (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
US11572543B2 (en) * 2015-05-08 2023-02-07 The Children's Medical Center. Corporation Targeting BCL11A enhancer functional regions for fetal hemoglobin reinduction
CA2988854A1 (en) 2015-05-08 2016-11-17 President And Fellows Of Harvard College Universal donor stem cells and related methods
JP7030522B2 (ja) * 2015-05-11 2022-03-07 エディタス・メディシン、インコーポレイテッド 幹細胞における遺伝子編集のための最適化ｃｒｉｓｐｒ／ｃａｓ９システムおよび方法
MX382223B (es) * 2015-05-12 2025-03-13 Sangamo Therapeutics Inc Regulacion de expresion genica mediada por nucleasa.
KR102796744B1 (ko) 2015-06-09 2025-04-15 에디타스 메디신, 인코포레이티드 이식의 개선을 위한 crispr/cas-관련 방법 및 조성물
WO2016205703A1 (en) * 2015-06-17 2016-12-22 The Uab Research Foundation Crispr/cas9 complex for genomic editing
AU2016278959A1 (en) 2015-06-17 2018-01-18 The Uab Research Foundation CRISPR/Cas9 complex for introducing a functional polypeptide into cells of blood cell lineage
WO2016205749A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems
US9926546B2 (en) 2015-08-28 2018-03-27 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
EP3341477B1 (en) 2015-08-28 2022-03-23 The General Hospital Corporation Engineered crispr-cas9 nucleases
US9512446B1 (en) 2015-08-28 2016-12-06 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
SG11201803173VA (en) 2015-10-23 2018-05-30 Harvard College Nucleobase editors and uses thereof
ES2908041T3 (es) * 2015-10-27 2022-04-27 Childrens Hospital Med Ct Uso de inhibidores de MAPK para reducir la pérdida de células madre hematopoyéticas durante el cultivo ex vivo y la manipulación genética
HK1252878A1 (zh) 2015-11-03 2019-06-06 President And Fellows Of Harvard College 用於含有基质的三维核酸的体积成像的方法和设备
EP3371306B8 (en) * 2015-11-04 2023-02-22 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
PT3371314T (pt) 2015-11-04 2023-08-31 Fate Therapeutics Inc Modificação genómica de células pluripotentes
CA3003152A1 (en) 2015-11-04 2017-05-11 Fate Therapeutics, Inc. Methods and compositions for inducing hematopoietic cell differentiation
US20190010495A1 (en) * 2015-12-28 2019-01-10 Novartis Ag Compositions and methods for the treatment of hemoglobinopathies
RU2021114137A (ru) 2016-01-27 2021-05-25 Онкорус, Инк. Онколитические вирусные векторы и их применение
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
EP3430142A1 (en) * 2016-03-14 2019-01-23 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
JP7259182B2 (ja) 2016-04-25 2023-04-18 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 ｉｎ ｓｉｔｕ分子検出のためのハイブリダイゼーション連鎖反応法
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
AU2017301609A1 (en) * 2016-07-25 2019-02-21 Bluebird Bio, Inc. BCL11A homing endonuclease variants, compositions, and methods of use
US11078481B1 (en) 2016-08-03 2021-08-03 KSQ Therapeutics, Inc. Methods for screening for cancer targets
JP7231935B2 (ja) 2016-08-03 2023-03-08 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 アデノシン核酸塩基編集因子およびそれらの使用
CA3033327A1 (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
WO2018039438A1 (en) 2016-08-24 2018-03-01 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
CA3035455A1 (en) 2016-09-08 2018-03-15 Bluebird Bio, Inc. Pd-1 homing endonuclease variants, compositions, and methods of use
WO2018071868A1 (en) 2016-10-14 2018-04-19 President And Fellows Of Harvard College Aav delivery of nucleobase editors
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
JP7175277B2 (ja) 2017-01-27 2022-11-18 チルドレンズ ホスピタル メディカル センター 造血幹細胞の生着活性を向上させる方法
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
US12390514B2 (en) 2017-03-09 2025-08-19 President And Fellows Of Harvard College Cancer vaccine
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
JP2020510439A (ja) 2017-03-10 2020-04-09 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 シトシンからグアニンへの塩基編集因子
WO2018170184A1 (en) 2017-03-14 2018-09-20 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
CN111031790A (zh) 2017-04-20 2020-04-17 E开创生物技术股份有限公司 产生基因修改的动物的方法
WO2018195545A2 (en) 2017-04-21 2018-10-25 The General Hospital Corporation Variants of cpf1 (cas12a) with altered pam specificity
WO2018200597A1 (en) * 2017-04-24 2018-11-01 Seattle Children's Hospital (dba Seattle Children's Research Institute) Homology directed repair compositions for the treatment of hemoglobinopathies
WO2018209158A2 (en) 2017-05-10 2018-11-15 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
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
CA3063449A1 (en) 2017-05-25 2018-11-29 The General Hospital Corporation Using split deaminases to limit unwanted off-target base editor deamination
US11788087B2 (en) 2017-05-25 2023-10-17 The Children's Medical Center Corporation BCL11A guide delivery
US12139720B2 (en) * 2017-06-02 2024-11-12 Institut National De La Sante Et De La Recherche Medicale (Inserm) Recombinant lentiviral vector for stem cell- based gene therapy of sickle cell disorder
US11866726B2 (en) 2017-07-14 2024-01-09 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
US11612625B2 (en) 2017-07-26 2023-03-28 Oncorus, Inc. Oncolytic viral vectors and uses thereof
EP3658573A1 (en) 2017-07-28 2020-06-03 President and Fellows of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (pace)
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
WO2019070974A1 (en) 2017-10-04 2019-04-11 Bluebird Bio, Inc. PCSK9 ENDONUCLEASE VARIANTS, COMPOSITIONS AND METHODS OF USE
KR20200086670A (ko) 2017-10-13 2020-07-17 셀렉타 바이오사이언시즈, 인크. 항바이러스 전달 벡터 IgM 반응을 약화시키기 위한 방법 및 조성물
AU2018352592C1 (en) 2017-10-16 2025-09-25 Beam Therapeutics, Inc. Uses of adenosine base editors
US20210180091A1 (en) * 2017-10-26 2021-06-17 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
CN109722414A (zh) * 2017-10-27 2019-05-07 博雅辑因（北京）生物科技有限公司 一种高效制备成熟红细胞的方法以及用于制备成熟红细胞的培养基
MX2020005849A (es) * 2017-12-05 2020-11-06 Vertex Pharma Células madre y progenitoras hematopoyéticas humanas cd34+ modificadas con crispr-cas9 y sus usos.
EP3724214A4 (en) 2017-12-15 2021-09-01 The Broad Institute Inc. SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING
EP3749768A1 (en) 2018-02-05 2020-12-16 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
WO2019150196A1 (en) 2018-02-05 2019-08-08 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
KR20200139679A (ko) 2018-02-26 2020-12-14 안톨알엑스, 인크. 면역관용성 리포솜 및 그의 사용 방법
WO2019178426A1 (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
WO2019204226A1 (en) 2018-04-16 2019-10-24 University Of Massachusetts Compositions and methods for improved gene editing
WO2019210216A2 (en) * 2018-04-27 2019-10-31 Seattle Children's Hospital D/B/A Seattle Children's Research Institute Talen-based and crispr/cas-based gene editing for bruton's tyrosine kinase
US12350284B2 (en) 2018-05-02 2025-07-08 The Children's Medical Center Corporation BCL11A microRNAs for treating hemoglobinopathies
CA3100019A1 (en) 2018-05-11 2019-11-14 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
AU2019287720B2 (en) 2018-06-14 2025-08-28 Regeneron Pharmaceuticals, Inc. CD79A chimeric antigen receptors
SG11202101576UA (en) 2018-08-18 2021-03-30 Harvard College In situ gene editing
US12421507B2 (en) 2018-08-20 2025-09-23 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
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
US12404500B2 (en) 2018-12-10 2025-09-02 Novo Nordisk A/S Homing endonuclease 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
US12264323B2 (en) 2018-12-17 2025-04-01 The Broad Institute, Inc. CRISPR CPF1 direct repeat variants
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
AU2020223060B2 (en) * 2019-02-13 2023-04-13 Beam Therapeutics Inc. Compositions and methods for treating hemoglobinopathies
GB2601617B (en) 2019-03-19 2024-02-21 Broad Inst 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
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
JP2022531269A (ja) * 2019-04-30 2022-07-06 博雅▲輯▼因（北京）生物科技有限公司 異常ヘモグロビン症の治療の有効性を予測するための方法
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
CN114206396A (zh) 2019-05-28 2022-03-18 西莱克塔生物科技公司 用于减弱抗病毒转移载体免疫应答的方法和组合物
CN114072518B (zh) * 2019-06-28 2024-06-21 Asc治疗公司 用于治疗地中海贫血或镰状细胞病的方法和组合物
EP4038190A1 (en) 2019-10-03 2022-08-10 Artisan Development Labs, Inc. 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
WO2021077000A1 (en) 2019-10-16 2021-04-22 The Broad Institute, Inc. Engineered muscle targeting compositions
EP4058586A4 (en) * 2019-11-15 2024-04-10 The Board of Trustees of the Leland Stanford Junior University Targeted integration at alpha-globin locus in human hematopoietic stem and progenitor cells
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
US12091681B2 (en) 2020-03-27 2024-09-17 Mendus B.V. Ex vivo use of modified cells of leukemic origin for enhancing the efficacy of adoptive cell therapy
DE112021002672T5 (de) 2020-05-08 2023-04-13 President And Fellows Of Harvard College Vefahren und zusammensetzungen zum gleichzeitigen editieren beider stränge einer doppelsträngigen nukleotid-zielsequenz
JP2023526007A (ja) * 2020-05-13 2023-06-20 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル β－ヘモグロビン異常症の処置のための塩基編集アプローチ
CN116157514A (zh) * 2020-06-04 2023-05-23 埃门多生物公司 新颖omni-59、61、67、76、79、80、81和82crispr核酸酶
WO2022003568A1 (en) 2020-06-30 2022-01-06 Dcprime B.V. Use of leukemia-derived cells in ovarian cancer vaccines
US20220049303A1 (en) 2020-08-17 2022-02-17 Readcoor, Llc Methods and systems for spatial mapping of genetic variants
CN116157517A (zh) * 2020-09-04 2023-05-23 国立大学法人广岛大学 目标dna的编辑方法、编辑了目标dna的细胞的制备方法及用于它们的dna编辑系统
JP2024503249A (ja) * 2020-12-21 2024-01-25 ２セブンティ バイオ インコーポレイテッド 部位特異的変異導入のための組成物及び方法
AU2022227650A1 (en) 2021-02-25 2023-10-12 Celyntra Therapeutics Sa Compositions and methods for targeting, editing, or modifying genes
EP4304633A1 (en) 2021-03-12 2024-01-17 Mendus B.V. Methods of vaccination and use of cd47 blockade
US20250179481A1 (en) 2021-06-01 2025-06-05 Celyntra Therapeutics Sa 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
JP2024534945A (ja) 2021-09-10 2024-09-26 アジレント・テクノロジーズ・インク 化学修飾を有するプライム編集のためのガイドｒｎａ
US20230141563A1 (en) 2021-10-12 2023-05-11 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
US20060153826A1 (en) 2003-01-28 2006-07-13 Sylvain Arnould Use of meganucleases for inducing homologous recombination ex vivo and in toto in vertebrate somatic tissues and application thereof
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
US20100086533A1 (en) 2007-02-19 2010-04-08 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
EP2334794B8 (en) * 2008-09-15 2017-04-19 The Children's Medical Center Corporation 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
WO2012118717A2 (en) * 2011-02-28 2012-09-07 Seattle Children's Research Institute Coupling endonucleases with end-processing enzymes drive high efficiency gene disruption
RU2018109507A (ru) 2012-02-24 2019-02-28 Фред Хатчинсон Кэнсер Рисерч Сентер Композиции и способы лечения гемоглобинопатии
SG11201500852WA (en) * 2012-08-29 2015-04-29 Sangamo Biosciences Inc Methods and compositions for treatment of a genetic condition

Also Published As

Publication number Publication date
AU2017201235C1 (en) 2018-11-01
NZ630900A (en) 2016-10-28
EP2836226B1 (en) 2017-06-28
SG11201405103SA (en) 2014-09-26
AU2017201235A1 (en) 2017-03-16
EP2836226A4 (en) 2016-04-13
AU2013222170B2 (en) 2017-01-05
AU2013222170A1 (en) 2014-10-02
US20150166969A1 (en) 2015-06-18
ES2641840T3 (es) 2017-11-14
KR20180009383A (ko) 2018-01-26
RU2018109507A (ru) 2019-02-28
US20170298329A1 (en) 2017-10-19
IN2014DN07853A (cg-RX-API-DMAC7.html) 2015-04-24
JP6170080B2 (ja) 2017-07-26
RU2650811C2 (ru) 2018-04-17
EP3272356A1 (en) 2018-01-24
EP2836226A1 (en) 2015-02-18
JP2015516146A (ja) 2015-06-11
ZA201406187B (en) 2018-05-30
WO2013126794A1 (en) 2013-08-29
AU2017201235B2 (en) 2018-08-02
RU2014138491A (ru) 2016-04-10
US10619140B2 (en) 2020-04-14
CN104284669A (zh) 2015-01-14
SG10201606959PA (en) 2016-09-29
JP2017148056A (ja) 2017-08-31
BR112014020625A2 (pt) 2017-07-04
DK2836226T3 (en) 2017-09-18
IL266582A (en) 2019-07-31
KR20140128453A (ko) 2014-11-05
HK1249861A1 (en) 2018-11-16
KR101833589B1 (ko) 2018-03-02
CA2865129A1 (en) 2013-08-29

Similar Documents

Publication Publication Date Title
IL266582A (en) Preparations and methods for the treatment of hemoglobin diseases
IL233674B (en) Preparations and methods for using phorbol esters for stroke treatment
EP2847152A4 (en) COMPOSITIONS AND METHOD FOR THE TREATMENT OF METABOLIC SYNDROME
HUE041553T2 (hu) Lizoszomális tárolási betegségek (LSD) kezelése és génterápia
IL225239A0 (en) Compounds for the treatment of acne and related diseases
IL238416A0 (en) Preparations and methods for the treatment of diseases caused by protein pathology
IL238177A0 (en) Methods and preparations for the treatment of cancer
EP2675469A4 (en) COMPOSITIONS, DEVICES AND METHODS OF USE THEREOF FOR THE TREATMENT OF CANCER
DK3292875T3 (en) Compositions and methods for treating diseases
ZA201408055B (en) Compositions and methods for the treatment of local pain
ZA201408056B (en) Compositions and methods for the treatment of mucositis
IL239374A0 (en) Preparations and methods for treating brain tumors
PL2968478T3 (pl) Kompozycje i sposoby do leczenia karboksyhemoglobinemii
IL238126B (en) Compositions for the treatment and prevention of obesity
ZA201408062B (en) Compositions and methods for the treatment of multiple sclerosis
ZA201408060B (en) Compositions and methods for the treatment of neurological disorders
IL235272B (en) Alfentanil preparation for the treatment of acute pain
ZA201408057B (en) Compositions and methods for the treatment of moderate to severe pain
IL233961A0 (en) The composition of a phytogenic supplement and methods of its use
ZA201408058B (en) Compositions and methods for the treatment of epilepsy
ZA201408065B (en) Compositions and methods for the treatment of diabetes
ZA201408061B (en) Compositions and methods for the treatment of neurological disorders
ZA201408053B (en) Compositions and methods for the treatment of metabolic syndrome
GB201207064D0 (en) Pharmaceutical compositions comprising 15-hetre and methods of using the same
AU2012902982A0 (en) Compositions and methods for the treatment of tuberculosis

Legal Events

Date Code Title Description
FF Patent granted
KB Patent renewed