MX2018011114A - Métodos y composiciones relacionadas con crispr/cas para el tratamiento de beta hemoglobinopatías. - Google Patents
Métodos y composiciones relacionadas con crispr/cas para el tratamiento de beta hemoglobinopatías.Info
- Publication number
- MX2018011114A MX2018011114A MX2018011114A MX2018011114A MX2018011114A MX 2018011114 A MX2018011114 A MX 2018011114A MX 2018011114 A MX2018011114 A MX 2018011114A MX 2018011114 A MX2018011114 A MX 2018011114A MX 2018011114 A MX2018011114 A MX 2018011114A
- Authority
- MX
- Mexico
- Prior art keywords
- crispr
- cas
- compositions
- related methods
- treating beta
- Prior art date
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- 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
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- 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
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- 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
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- 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
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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- 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
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- 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
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
- C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
- C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
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- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0634—Cells from the blood or the immune system
- C12N5/0641—Erythrocytes
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- 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]
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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]
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
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- C12N2510/00—Genetically modified cells
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- C12N2800/00—Nucleic acids vectors
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
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- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
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- Biochemistry (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
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- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Cell Biology (AREA)
- Epidemiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Mycology (AREA)
- Immunology (AREA)
- Crystallography & Structural Chemistry (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Abstract
Se describen composiciones y métodos relacionados con CRISPR/CAS para el tratamiento de ß hemoglobinopatías.
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662308190P | 2016-03-14 | 2016-03-14 | |
| US201762456615P | 2017-02-08 | 2017-02-08 | |
| PCT/US2017/022377 WO2017160890A1 (en) | 2016-03-14 | 2017-03-14 | Crispr/cas-related methods and compositions for treating beta hemoglobinopathies |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2018011114A true MX2018011114A (es) | 2019-02-20 |
Family
ID=58413206
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2018011114A MX2018011114A (es) | 2016-03-14 | 2017-03-14 | Métodos y composiciones relacionadas con crispr/cas para el tratamiento de beta hemoglobinopatías. |
| MX2024012899A MX2024012899A (es) | 2016-03-14 | 2018-09-13 | Metodos y composiciones relacionadas con crispr/cas para el tratamiento de beta hemoglobinopatias |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2024012899A MX2024012899A (es) | 2016-03-14 | 2018-09-13 | Metodos y composiciones relacionadas con crispr/cas para el tratamiento de beta hemoglobinopatias |
Country Status (11)
| Country | Link |
|---|---|
| US (2) | US20200255857A1 (es) |
| EP (1) | EP3430142A1 (es) |
| JP (2) | JP2019508051A (es) |
| KR (2) | KR102532663B1 (es) |
| CN (4) | CN117821458A (es) |
| AU (2) | AU2017235333B2 (es) |
| CA (1) | CA3017956A1 (es) |
| IL (2) | IL313038A (es) |
| MX (2) | MX2018011114A (es) |
| SG (1) | SG11201807859WA (es) |
| WO (1) | WO2017160890A1 (es) |
Families Citing this family (67)
| 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 |
| US9163284B2 (en) | 2013-08-09 | 2015-10-20 | President And Fellows Of Harvard College | Methods for identifying a target site of a Cas9 nuclease |
| US9359599B2 (en) | 2013-08-22 | 2016-06-07 | President And Fellows Of Harvard College | Engineered transcription activator-like effector (TALE) domains and uses thereof |
| US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
| US9737604B2 (en) | 2013-09-06 | 2017-08-22 | President And Fellows Of Harvard College | Use of cationic lipids to deliver CAS9 |
| US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins 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 |
| CA2956224A1 (en) | 2014-07-30 | 2016-02-11 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| WO2016135559A2 (en) | 2015-02-23 | 2016-09-01 | Crispr Therapeutics Ag | Materials and methods for treatment of human genetic diseases including hemoglobinopathies |
| SG11201706767RA (en) | 2015-02-23 | 2017-09-28 | Crispr Therapeutics Ag | Materials and methods for treatment of hemoglobinopathies |
| US11390884B2 (en) | 2015-05-11 | 2022-07-19 | 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 |
| AU2016342380B2 (en) | 2015-10-23 | 2022-04-07 | President And Fellows Of Harvard College | Nucleobase editors and uses thereof |
| EP3371306B8 (en) | 2015-11-04 | 2023-02-22 | Vertex Pharmaceuticals Incorporated | Materials and methods for treatment of hemoglobinopathies |
| IL308426B1 (en) | 2016-08-03 | 2025-11-01 | Harvard College | Adenosine nucleobase editors and uses thereof |
| JP7201153B2 (ja) | 2016-08-09 | 2023-01-10 | 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 | プログラム可能cas9-リコンビナーゼ融合タンパク質およびその使用 |
| WO2018039438A1 (en) | 2016-08-24 | 2018-03-01 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| KR20240007715A (ko) | 2016-10-14 | 2024-01-16 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵염기 에디터의 aav 전달 |
| WO2018083071A1 (en) * | 2016-11-02 | 2018-05-11 | Universität Basel | Immunologically discernible cell surface variants for use in cell therapy |
| US10745677B2 (en) | 2016-12-23 | 2020-08-18 | 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 |
| US12390514B2 (en) | 2017-03-09 | 2025-08-19 | President And Fellows Of Harvard College | Cancer vaccine |
| US11898179B2 (en) | 2017-03-09 | 2024-02-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
| KR20190127797A (ko) | 2017-03-10 | 2019-11-13 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 시토신에서 구아닌으로의 염기 편집제 |
| EP3596217A1 (en) | 2017-03-14 | 2020-01-22 | 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 |
| 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 |
| US20200140896A1 (en) * | 2017-06-30 | 2020-05-07 | Novartis Ag | Methods for the treatment of disease with gene editing systems |
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| 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 |
| US11319532B2 (en) | 2017-08-30 | 2022-05-03 | President And Fellows Of Harvard College | High efficiency base editors comprising Gam |
| KR20200121782A (ko) * | 2017-10-16 | 2020-10-26 | 더 브로드 인스티튜트, 인코퍼레이티드 | 아데노신 염기 편집제의 용도 |
| EP3701029A1 (en) * | 2017-10-26 | 2020-09-02 | Vertex Pharmaceuticals Incorporated | Materials and methods for treatment of hemoglobinopathies |
| CN111556893A (zh) * | 2017-11-06 | 2020-08-18 | 爱迪塔斯医药股份有限公司 | 免疫疗法t细胞中cblb的crispr-cas9编辑的方法、组合物和组分 |
| MA51107A (fr) | 2017-12-05 | 2020-10-14 | Vertex Pharma | Cellules souches et progénitrices hématopoïétiques humaines cd34+ modifiées par crispr-cas9 et utilisations associées |
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| JP7706888B2 (ja) * | 2018-03-14 | 2025-07-14 | エディタス・メディシン、インコーポレイテッド | 異常ヘモグロビン症の治療のためのシステム及び方法 |
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| JP7642531B2 (ja) | 2018-05-11 | 2025-03-10 | ビーム セラピューティクス インク. | プログラム可能塩基エディターシステムを用いて病原性アミノ酸を置換する方法 |
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| WO2020053224A1 (en) * | 2018-09-11 | 2020-03-19 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods for increasing fetal hemoglobin content in eukaryotic cells and uses thereof for the treatment of hemoglobinopathies |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
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| JP7428712B2 (ja) * | 2018-12-05 | 2024-02-06 | フレッド ハッチンソン キャンサー センター | 低/最小操作による遺伝子改変細胞の製造 |
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| CN114096666B (zh) | 2019-02-13 | 2025-03-18 | 比姆医疗股份有限公司 | 治疗血红素病变的组合物和方法 |
| DE112020001342T5 (de) | 2019-03-19 | 2022-01-13 | President and Fellows of Harvard College | Verfahren und Zusammensetzungen zum Editing von Nukleotidsequenzen |
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| CN112011576A (zh) * | 2019-05-31 | 2020-12-01 | 华东师范大学 | Crispr基因编辑技术在治疗地中海贫血中的应用 |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| 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 |
| EP4146804A1 (en) | 2020-05-08 | 2023-03-15 | The Broad Institute Inc. | Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence |
| CN111876416B (zh) * | 2020-07-01 | 2021-09-03 | 广州瑞风生物科技有限公司 | 激活γ-珠蛋白基因表达的方法和组合物 |
| AU2022381552A1 (en) * | 2021-11-02 | 2024-05-09 | Incisive Genetics, Inc. | Compositions and methods for preventing, ameliorating, or treating sickle cell disease |
| CN114848851A (zh) * | 2022-04-29 | 2022-08-05 | 广州医科大学附属第三医院(广州重症孕产妇救治中心、广州柔济医院) | 治疗β-地中海贫血的药物 |
| WO2024073751A1 (en) | 2022-09-29 | 2024-04-04 | Vor Biopharma Inc. | Methods and compositions for gene modification and enrichment |
| WO2025038164A1 (en) * | 2023-08-15 | 2025-02-20 | University Of Massachusetts | Nick resection in cancer |
| 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 |
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| 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 |
| KR20180009383A (ko) * | 2012-02-24 | 2018-01-26 | 프레드 헛친슨 켄서 리서치 센터 | 이상혈색소증 치료를 위한 조성물 및 방법 |
| US9963715B2 (en) * | 2012-08-29 | 2018-05-08 | Sangamo Therapeutics, Inc. | Methods and compositions for treatment of a genetic condition |
| SG11201504038XA (en) * | 2012-11-27 | 2015-06-29 | Childrens Medical Center | Targeting bcl11a distal regulatory elements for fetal hemoglobin reinduction |
| JP2016521975A (ja) * | 2013-05-15 | 2016-07-28 | サンガモ バイオサイエンシーズ, インコーポレイテッド | 遺伝的状態の処置のための方法および組成物 |
| KR102307280B1 (ko) * | 2013-06-05 | 2021-10-01 | 듀크 유니버시티 | Rna-가이드 유전자 편집 및 유전자 조절 |
| EP3066201B1 (en) * | 2013-11-07 | 2018-03-07 | Editas Medicine, Inc. | Crispr-related methods and compositions with governing grnas |
| 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) |
| EP3981876A1 (en) * | 2014-03-26 | 2022-04-13 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating sickle cell disease |
| US11680268B2 (en) | 2014-11-07 | 2023-06-20 | Editas Medicine, Inc. | Methods for improving CRISPR/Cas-mediated genome-editing |
| SG11201706767RA (en) * | 2015-02-23 | 2017-09-28 | Crispr Therapeutics Ag | Materials and methods for treatment of hemoglobinopathies |
| US11390884B2 (en) | 2015-05-11 | 2022-07-19 | Editas Medicine, Inc. | Optimized CRISPR/cas9 systems and methods for gene editing in stem cells |
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2017
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- 2017-03-14 WO PCT/US2017/022377 patent/WO2017160890A1/en not_active Ceased
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| JP2023075166A (ja) | 2023-05-30 |
| CN109153994A (zh) | 2019-01-04 |
| JP2019508051A (ja) | 2019-03-28 |
| CN118127006A (zh) | 2024-06-04 |
| EP3430142A1 (en) | 2019-01-23 |
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| KR102723347B1 (ko) | 2024-10-28 |
| KR20230070331A (ko) | 2023-05-22 |
| CN117821458A (zh) | 2024-04-05 |
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| AU2017235333A1 (en) | 2018-10-04 |
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| AU2017235333B2 (en) | 2023-08-24 |
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| IL261714A (en) | 2018-10-31 |
| CA3017956A1 (en) | 2017-09-21 |
| KR102532663B1 (ko) | 2023-05-16 |
| KR20180120752A (ko) | 2018-11-06 |
| US20240360471A1 (en) | 2024-10-31 |
| SG11201807859WA (en) | 2018-10-30 |
| IL261714B1 (en) | 2024-06-01 |
| MX2024012899A (es) | 2024-11-08 |
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