JP2019500043A - 異常ヘモグロビン症の治療用組成物および方法 - Google Patents
異常ヘモグロビン症の治療用組成物および方法 Download PDFInfo
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- JP2019500043A JP2019500043A JP2018534141A JP2018534141A JP2019500043A JP 2019500043 A JP2019500043 A JP 2019500043A JP 2018534141 A JP2018534141 A JP 2018534141A JP 2018534141 A JP2018534141 A JP 2018534141A JP 2019500043 A JP2019500043 A JP 2019500043A
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- C12N15/90—Stable introduction of foreign DNA into chromosome
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- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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- A61K2035/124—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells the cells being hematopoietic, bone marrow derived or blood cells
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
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- Diabetes (AREA)
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- Crystallography & Structural Chemistry (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
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Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021096653A JP2021166514A (ja) | 2015-12-28 | 2021-06-09 | 異常ヘモグロビン症の治療用組成物および方法 |
| JP2023128086A JP2023159185A (ja) | 2015-12-28 | 2023-08-04 | 異常ヘモグロビン症の治療用組成物および方法 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562271968P | 2015-12-28 | 2015-12-28 | |
| US62/271,968 | 2015-12-28 | ||
| US201662347484P | 2016-06-08 | 2016-06-08 | |
| US62/347,484 | 2016-06-08 | ||
| PCT/IB2016/058007 WO2017115268A1 (en) | 2015-12-28 | 2016-12-26 | Compositions and methods for the treatment of hemoglobinopathies |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021096653A Division JP2021166514A (ja) | 2015-12-28 | 2021-06-09 | 異常ヘモグロビン症の治療用組成物および方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2019500043A true JP2019500043A (ja) | 2019-01-10 |
| JP2019500043A5 JP2019500043A5 (enExample) | 2020-02-13 |
Family
ID=57822008
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018534141A Pending JP2019500043A (ja) | 2015-12-28 | 2016-12-26 | 異常ヘモグロビン症の治療用組成物および方法 |
| JP2021096653A Pending JP2021166514A (ja) | 2015-12-28 | 2021-06-09 | 異常ヘモグロビン症の治療用組成物および方法 |
| JP2023128086A Pending JP2023159185A (ja) | 2015-12-28 | 2023-08-04 | 異常ヘモグロビン症の治療用組成物および方法 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021096653A Pending JP2021166514A (ja) | 2015-12-28 | 2021-06-09 | 異常ヘモグロビン症の治療用組成物および方法 |
| JP2023128086A Pending JP2023159185A (ja) | 2015-12-28 | 2023-08-04 | 異常ヘモグロビン症の治療用組成物および方法 |
Country Status (16)
| Country | Link |
|---|---|
| US (2) | US20190010495A1 (enExample) |
| EP (2) | EP3397767A1 (enExample) |
| JP (3) | JP2019500043A (enExample) |
| KR (2) | KR102860636B1 (enExample) |
| CN (1) | CN108779462A (enExample) |
| AU (4) | AU2016381313B2 (enExample) |
| CA (1) | CA3009727A1 (enExample) |
| EA (1) | EA201891532A1 (enExample) |
| HK (1) | HK1258205A1 (enExample) |
| IL (2) | IL308706A (enExample) |
| MX (2) | MX2018007987A (enExample) |
| MY (1) | MY192848A (enExample) |
| NZ (4) | NZ744649A (enExample) |
| PH (1) | PH12018501378A1 (enExample) |
| SG (1) | SG11201805217XA (enExample) |
| WO (1) | WO2017115268A1 (enExample) |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US12161674B2 (en) | 2017-12-05 | 2024-12-10 | Vertex Pharmaceuticals Incorporated | CRISPR-CAS9 modified CD34+ human hematopoietic stem and progenitor cells and uses thereof |
Families Citing this family (85)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP6261500B2 (ja) | 2011-07-22 | 2018-01-17 | 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 | ヌクレアーゼ切断特異性の評価および改善 |
| US10704021B2 (en) | 2012-03-15 | 2020-07-07 | Flodesign Sonics, Inc. | Acoustic perfusion devices |
| US20150044192A1 (en) | 2013-08-09 | 2015-02-12 | 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 |
| US9737604B2 (en) | 2013-09-06 | 2017-08-22 | President And Fellows Of Harvard College | Use of cationic lipids to deliver CAS9 |
| US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
| US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
| US9068179B1 (en) | 2013-12-12 | 2015-06-30 | President And Fellows Of Harvard College | Methods for correcting presenilin point mutations |
| WO2015105955A1 (en) | 2014-01-08 | 2015-07-16 | Flodesign Sonics, Inc. | Acoustophoresis device with dual acoustophoretic chamber |
| US10077453B2 (en) | 2014-07-30 | 2018-09-18 | President And Fellows Of Harvard College | CAS9 proteins including ligand-dependent inteins |
| WO2016135558A2 (en) | 2015-02-23 | 2016-09-01 | Crispr Therapeutics Ag | Materials and methods for treatment of hemoglobinopathies |
| WO2016135559A2 (en) | 2015-02-23 | 2016-09-01 | Crispr Therapeutics Ag | Materials and methods for treatment of human genetic diseases including hemoglobinopathies |
| US11708572B2 (en) | 2015-04-29 | 2023-07-25 | Flodesign Sonics, Inc. | Acoustic cell separation techniques and processes |
| US11377651B2 (en) | 2016-10-19 | 2022-07-05 | Flodesign Sonics, Inc. | Cell therapy processes utilizing acoustophoresis |
| WO2016182917A1 (en) * | 2015-05-08 | 2016-11-17 | Children's Medical Center Corporation | Targeting bcl11a enhancer functional regions for fetal hemoglobin reinduction |
| CA2986310A1 (en) | 2015-05-11 | 2016-11-17 | Editas Medicine, Inc. | Optimized crispr/cas9 systems and methods for gene editing in stem cells |
| US10808020B2 (en) * | 2015-05-12 | 2020-10-20 | Sangamo Therapeutics, Inc. | Nuclease-mediated regulation of gene expression |
| EP3307887A1 (en) | 2015-06-09 | 2018-04-18 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for improving transplantation |
| EP3365356B1 (en) | 2015-10-23 | 2023-06-28 | 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 |
| US11214789B2 (en) | 2016-05-03 | 2022-01-04 | Flodesign Sonics, Inc. | Concentration and washing of particles with acoustics |
| 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 |
| EP3497214B1 (en) | 2016-08-09 | 2023-06-28 | President and Fellows of Harvard College | Programmable cas9-recombinase fusion proteins and uses thereof |
| WO2018039438A1 (en) | 2016-08-24 | 2018-03-01 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| US20190203230A1 (en) | 2016-09-28 | 2019-07-04 | Novartis Ag | Porous membrane-based macromolecule delivery system |
| EP3526320A1 (en) | 2016-10-14 | 2019-08-21 | 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 |
| 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 |
| WO2018165504A1 (en) | 2017-03-09 | 2018-09-13 | 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 |
| 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 |
| KR20240116572A (ko) | 2017-03-23 | 2024-07-29 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제 |
| US20210380937A1 (en) | 2017-04-04 | 2021-12-09 | The Board Of Trustees Of The Leland Stanford Junior University | Preparation , expansion, and uses of adult pluripotent stem cells |
| 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 |
| US11788087B2 (en) * | 2017-05-25 | 2023-10-17 | The Children's Medical Center Corporation | BCL11A guide delivery |
| WO2019003193A1 (en) | 2017-06-30 | 2019-01-03 | Novartis Ag | METHODS FOR TREATING DISEASES USING GENE EDITING SYSTEMS |
| 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 |
| WO2019023680A1 (en) | 2017-07-28 | 2019-01-31 | President And Fellows Of Harvard College | METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE) |
| EP3676376B1 (en) | 2017-08-30 | 2025-01-15 | President and Fellows of Harvard College | High efficiency base editors comprising gam |
| JP2020537541A (ja) * | 2017-09-29 | 2020-12-24 | インテリア セラピューティクス,インコーポレーテッド | 脂質ナノ粒子を用いるインビトロでのmrnaの送達方法 |
| CN109706148A (zh) * | 2017-09-30 | 2019-05-03 | 广东赤萌医疗科技有限公司 | 一种用于敲除BCL11A基因或者BCL11A基因增强子的gRNA、gRNA组合物以及电转方法 |
| CN111757937A (zh) | 2017-10-16 | 2020-10-09 | 布罗德研究所股份有限公司 | 腺苷碱基编辑器的用途 |
| MA50849A (fr) * | 2017-10-26 | 2020-09-02 | Vertex Pharma | Substances et procédés pour le traitement d'hémoglobinopathies |
| 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 |
| US20210180053A1 (en) | 2017-11-01 | 2021-06-17 | Novartis Ag | Synthetic rnas and methods of use |
| WO2019098759A2 (ko) * | 2017-11-16 | 2019-05-23 | 재단법인 목암생명과학연구소 | 형질전환된 인간세포 및 이의 용도 |
| WO2019106522A1 (en) * | 2017-11-28 | 2019-06-06 | Novartis Ag | Pooled crispr/cas9 screening in primary cells using guide swap technology |
| BR112020009889A2 (pt) | 2017-12-14 | 2020-11-03 | Flodesign Sonics, Inc. | acionador e controlador de transdutor acústico |
| WO2019118949A1 (en) | 2017-12-15 | 2019-06-20 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| CN111902411A (zh) * | 2018-01-03 | 2020-11-06 | 美真达治疗公司 | 用于扩增造血干细胞和祖细胞以及治疗遗传性代谢紊乱的组合物和方法 |
| WO2019147302A1 (en) * | 2018-01-26 | 2019-08-01 | Bauer Daniel E | Targeting bcl11a distal regulatory elements with a cas9-cas9 fusion for fetal hemoglobin reinduction |
| US11566236B2 (en) | 2018-02-05 | 2023-01-31 | Vertex Pharmaceuticals Incorporated | Materials and methods for treatment of hemoglobinopathies |
| EP3749768A1 (en) * | 2018-02-05 | 2020-12-16 | Vertex Pharmaceuticals Incorporated | Materials and methods for treatment of hemoglobinopathies |
| 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 |
| WO2019213273A1 (en) * | 2018-05-01 | 2019-11-07 | The Children's Medical Center Corporation | Enhanced bcl11a rnp / crispr delivery & editing using a 3xnls-cas9 |
| EP3787600A4 (en) | 2018-05-02 | 2022-02-16 | The Children's Medical Center Corporation | ENHANCED BCL11A MICRORNAS FOR THE TREATMENT OF HEMOGLOBINOPATHIES |
| CN112105732A (zh) * | 2018-05-10 | 2020-12-18 | 先正达参股股份有限公司 | 用于多核苷酸的靶向编辑的方法和组合物 |
| CN112654710A (zh) | 2018-05-16 | 2021-04-13 | 辛瑟高公司 | 用于指导rna设计和使用的方法和系统 |
| US12157760B2 (en) | 2018-05-23 | 2024-12-03 | The Broad Institute, Inc. | Base editors and uses thereof |
| CA3113255A1 (en) | 2018-09-21 | 2020-03-26 | APstem Therapeutics, Inc. | Human pluripotent adult stem cells |
| US12281338B2 (en) | 2018-10-29 | 2025-04-22 | The Broad Institute, Inc. | Nucleobase editors comprising GeoCas9 and uses thereof |
| US20220047637A1 (en) * | 2018-11-29 | 2022-02-17 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
| WO2020154500A1 (en) | 2019-01-23 | 2020-07-30 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| MX2021011426A (es) | 2019-03-19 | 2022-03-11 | Broad Inst Inc | Metodos y composiciones para editar secuencias de nucleótidos. |
| EP3956349A1 (en) | 2019-04-17 | 2022-02-23 | 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 |
| WO2020221291A1 (zh) * | 2019-04-30 | 2020-11-05 | 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 |
| US20220380756A1 (en) * | 2019-06-28 | 2022-12-01 | Asc Therapeutics Inc. | Methods and compositions for treating thalassemia or sickle cell disease |
| CN114364799B (zh) * | 2019-08-28 | 2024-07-19 | 甘李药业股份有限公司 | 编辑造血干/祖细胞中bcl11a基因的方法 |
| US20220380757A1 (en) * | 2019-09-30 | 2022-12-01 | The Children's Medical Center Corporation | Bcl11a guide and base editor delivery |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| MX2022014008A (es) | 2020-05-08 | 2023-02-09 | Broad Inst Inc | Métodos y composiciones para la edición simultánea de ambas cadenas de una secuencia de nucleótidos de doble cadena objetivo. |
| CN114149990A (zh) * | 2020-09-08 | 2022-03-08 | 甘李药业股份有限公司 | 编辑造血干/祖细胞中bcl11a基因的方法 |
| US12079386B2 (en) | 2020-09-14 | 2024-09-03 | Zebra Technologies Corporation | Devices having protective screens selectively positionable to switch devices between user interface modes and methods of operating the same |
| US20240245725A1 (en) * | 2021-05-13 | 2024-07-25 | The Children's Medical Center Corporation | Methods for stratifying subjects for fetal hemoglobin reinduction |
| CN113584167B (zh) * | 2021-07-07 | 2023-10-03 | 武汉大学中南医院 | 一种用于检测FLT3-F691L突变的crRNA、等温扩增引物和试剂盒 |
| EP4273242A1 (en) * | 2022-05-03 | 2023-11-08 | ETH Zurich | Crispr-based modification of human hbd gene |
| WO2023214512A1 (ja) | 2022-05-06 | 2023-11-09 | 学校法人常翔学園 | 人工rna分子 |
| EP4561639A2 (en) * | 2022-07-25 | 2025-06-04 | Celyntra Therapeutics SA | Compositions and methods for genome editing |
| 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 |
| 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 (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2010059401A2 (en) * | 2008-10-30 | 2010-05-27 | Irm Llc | Compounds that expand hematopoietic stem cells |
| WO2013110198A1 (en) * | 2012-01-27 | 2013-08-01 | Université de Montréal | Pyrimido[4,5-b]indole derivatives and use thereof in the expansion of hematopoietic stem cells |
| 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 |
| 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 |
| JP2015536657A (ja) * | 2012-11-27 | 2015-12-24 | 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の再誘導のためのbcl11a遠位調節エレメントの標的化 |
| WO2016182917A1 (en) * | 2015-05-08 | 2016-11-17 | Children's Medical Center Corporation | Targeting bcl11a enhancer functional regions for fetal hemoglobin reinduction |
Family Cites Families (26)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| SG181904A1 (en) | 2009-12-23 | 2012-07-30 | Novartis Ag | Lipids, lipid compositions, and methods of using them |
| ES2557382T3 (es) | 2010-07-06 | 2016-01-25 | Glaxosmithkline Biologicals Sa | Liposomas con lípidos que tienen un valor de pKa ventajoso para el suministro de ARN |
| EP3542789A3 (en) | 2010-08-31 | 2020-01-01 | GlaxoSmithKline Biologicals SA | Lipids suitable for liposomal delivery of protein-coding rna |
| EP4115875A1 (en) | 2011-07-06 | 2023-01-11 | GlaxoSmithKline Biologicals S.A. | Liposomes having useful n:p ratio for delivery of rna molecules |
| BR112014009346B1 (pt) | 2011-10-17 | 2020-09-15 | Massachusetts Institute Of Technology | Entrega intracelular |
| 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 |
| SG11201405103SA (en) * | 2012-02-24 | 2014-09-26 | Hutchinson Fred Cancer Res | Compositions and methods for the treatment of hemoglobinopathies |
| US8697359B1 (en) * | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
| WO2014136086A1 (en) | 2013-03-08 | 2014-09-12 | Novartis Ag | Lipids and lipid compositions for the delivery of active agents |
| EP2970948B1 (en) | 2013-03-15 | 2018-12-26 | GlaxoSmithKline Biologicals SA | Rna purification methods |
| KR102210319B1 (ko) * | 2013-03-15 | 2021-02-01 | 더 제너럴 하스피탈 코포레이션 | 특정 게놈 좌위에 대한 유전적 및 후성적 조절 단백질의 rna-안내 표적화 |
| AU2014265331B2 (en) * | 2013-05-15 | 2019-12-05 | Sangamo Therapeutics, Inc. | Methods and compositions for treatment of a genetic condition |
| 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 |
| KR102243597B1 (ko) | 2013-08-16 | 2021-04-26 | 메사추세츠 인스티튜트 오브 테크놀로지 | 세포로의 선택적 물질 전달 |
| PT3492593T (pt) * | 2013-11-13 | 2021-10-18 | Childrens Medical Center | Regulação da expressão de genes mediada por nucleases |
| WO2015079057A2 (en) * | 2013-11-28 | 2015-06-04 | Haplogen Genomics Gmbh | Somatic haploid human cell line |
| US10426737B2 (en) | 2013-12-19 | 2019-10-01 | Novartis Ag | Lipids and lipid compositions for the delivery of active agents |
| EP3083556B1 (en) | 2013-12-19 | 2019-12-25 | Novartis AG | Lipids and lipid compositions for the delivery of active agents |
| WO2015158860A1 (de) * | 2014-04-16 | 2015-10-22 | Sika Technology Ag | Schnell härtende migrationsfreie zusammensetzung auf basis von silangruppen-haltigen polymeren |
| JP6514717B2 (ja) * | 2014-04-25 | 2019-05-15 | ザ チルドレンズ メディカル センター コーポレーション | 異常ヘモグロビン症を治療するための組成物および方法 |
| CN104480144B (zh) * | 2014-12-12 | 2017-04-12 | 武汉大学 | 用于艾滋病基因治疗的CRISPR/Cas9重组慢病毒载体及其慢病毒 |
| CN109706148A (zh) * | 2017-09-30 | 2019-05-03 | 广东赤萌医疗科技有限公司 | 一种用于敲除BCL11A基因或者BCL11A基因增强子的gRNA、gRNA组合物以及电转方法 |
| 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 |
| 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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-
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- 2022-10-13 US US17/965,366 patent/US20240002843A1/en active Pending
-
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- 2023-08-04 JP JP2023128086A patent/JP2023159185A/ja active Pending
-
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- 2025-01-17 AU AU2025200366A patent/AU2025200366A1/en active Pending
Patent Citations (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2010059401A2 (en) * | 2008-10-30 | 2010-05-27 | Irm Llc | Compounds that expand hematopoietic stem cells |
| WO2013110198A1 (en) * | 2012-01-27 | 2013-08-01 | Université de Montréal | Pyrimido[4,5-b]indole derivatives and use thereof in the expansion of hematopoietic stem cells |
| JP2015536657A (ja) * | 2012-11-27 | 2015-12-24 | 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の再誘導のためのbcl11a遠位調節エレメントの標的化 |
| 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 |
| 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 |
| WO2016182917A1 (en) * | 2015-05-08 | 2016-11-17 | Children's Medical Center Corporation | Targeting bcl11a enhancer functional regions for fetal hemoglobin reinduction |
Non-Patent Citations (2)
| Title |
|---|
| CANVER M. C. ET AL., NATURE, vol. Vol.527 (2015 Nov), JPN6020047977, pages 192 - 197, ISSN: 0004407010 * |
| WANG T. ET AL., SCIENCE, vol. Vol.343 (2014), JPN6020047976, pages 80 - 84, ISSN: 0004407011 * |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US12161674B2 (en) | 2017-12-05 | 2024-12-10 | Vertex Pharmaceuticals Incorporated | CRISPR-CAS9 modified CD34+ human hematopoietic stem and progenitor cells and uses thereof |
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