HK1258205A1 - 用於治療血紅蛋白病的組合物和方法 - Google Patents

用於治療血紅蛋白病的組合物和方法

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Publication number
HK1258205A1
HK1258205A1 HK19100562.7A HK19100562A HK1258205A1 HK 1258205 A1 HK1258205 A1 HK 1258205A1 HK 19100562 A HK19100562 A HK 19100562A HK 1258205 A1 HK1258205 A1 HK 1258205A1
Authority
HK
Hong Kong
Prior art keywords
hemoglobinopathies
compositions
treatment
methods
Prior art date
Application number
HK19100562.7A
Other languages
English (en)
Inventor
Anthony Edward Boitano
Michael Cooke
Lloyd B Klickstein
Reynald Lescarbeau
Craig Stephen Mickanin
Kabungo Mulumba
Seshidhar Reddy Police
Jennifer Snead
Susan C Stevenson
Morag Stewart
Yi Yang
Original Assignee
Novartis Ag
Intellia Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Novartis Ag, Intellia Therapeutics Inc filed Critical Novartis Ag
Publication of HK1258205A1 publication Critical patent/HK1258205A1/zh

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    • 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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    • C12N15/90—Stable introduction of foreign DNA into chromosome
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    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
HK19100562.7A 2015-12-28 2019-01-14 用於治療血紅蛋白病的組合物和方法 HK1258205A1 (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562271968P 2015-12-28 2015-12-28
US201662347484P 2016-06-08 2016-06-08
PCT/IB2016/058007 WO2017115268A1 (en) 2015-12-28 2016-12-26 Compositions and methods for the treatment of hemoglobinopathies

Publications (1)

Publication Number Publication Date
HK1258205A1 true HK1258205A1 (zh) 2019-11-08

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Country Status (16)

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US (2) US20190010495A1 (zh)
EP (2) EP3397767A1 (zh)
JP (3) JP2019500043A (zh)
KR (1) KR20180103923A (zh)
CN (1) CN108779462A (zh)
AU (3) AU2016381313B2 (zh)
BR (1) BR112018013065A2 (zh)
CA (1) CA3009727A1 (zh)
EA (1) EA201891532A1 (zh)
HK (1) HK1258205A1 (zh)
IL (2) IL260257B1 (zh)
MX (2) MX2018007987A (zh)
MY (1) MY192848A (zh)
PH (1) PH12018501378A1 (zh)
SG (1) SG11201805217XA (zh)
WO (1) WO2017115268A1 (zh)

Families Citing this family (64)

* Cited by examiner, † Cited by third party
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
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
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9340800B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College Extended DNA-sensing GRNAS
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
CA2935960C (en) 2014-01-08 2023-01-10 Bart Lipkens 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
US20180030438A1 (en) 2015-02-23 2018-02-01 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
US11377651B2 (en) 2016-10-19 2022-07-05 Flodesign Sonics, Inc. Cell therapy processes utilizing acoustophoresis
US11708572B2 (en) 2015-04-29 2023-07-25 Flodesign Sonics, Inc. Acoustic cell separation techniques and processes
US11572543B2 (en) * 2015-05-08 2023-02-07 The Children's Medical Center. Corporation Targeting BCL11A enhancer functional regions for fetal hemoglobin reinduction
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
BR112017024115A2 (pt) * 2015-05-12 2018-08-07 Sangamo Therapeutics Inc regulação de expressão genética mediada por nuclease
AU2016276702B2 (en) 2015-06-09 2022-07-28 Editas Medicine, Inc. CRISPR/CAS-related methods and compositions for improving transplantation
WO2017070632A2 (en) 2015-10-23 2017-04-27 President And Fellows Of Harvard College Nucleobase editors and uses thereof
US11214789B2 (en) 2016-05-03 2022-01-04 Flodesign Sonics, Inc. Concentration and washing of particles with acoustics
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
CA3033327A1 (en) 2016-08-09 2018-02-15 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
WO2018064387A1 (en) 2016-09-28 2018-04-05 Novartis Ag Porous membrane-based macromolecule delivery system
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
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
WO2018165629A1 (en) 2017-03-10 2018-09-13 President And Fellows Of Harvard College Cytosine to guanine base editor
EP3596217A1 (en) 2017-03-14 2020-01-22 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
KR20190130613A (ko) 2017-03-23 2019-11-22 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
US20210222201A1 (en) * 2017-04-24 2021-07-22 Seattie 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
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
WO2018218135A1 (en) * 2017-05-25 2018-11-29 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
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
CN111630177A (zh) * 2017-09-29 2020-09-04 英特利亚治疗股份有限公司 使用脂质纳米粒子的体外mrna递送方法
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
WO2019079347A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. USES OF BASIC EDITORS ADENOSINE
EP3701029A1 (en) * 2017-10-26 2020-09-02 Vertex Pharmaceuticals Incorporated Materials and methods for 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
EP3704245A1 (en) 2017-11-01 2020-09-09 Novartis AG Synthetic rnas and methods of use
US20200407713A1 (en) * 2017-11-16 2020-12-31 Mogam Institute For Biomedical Research Transformed human cell and use thereof
WO2019106522A1 (en) * 2017-11-28 2019-06-06 Novartis Ag Pooled crispr/cas9 screening in primary cells using guide swap technology
CN111629747A (zh) * 2017-12-05 2020-09-04 沃泰克斯药物股份有限公司 Crispr-cas9修饰的cd34+人血色素干细胞和祖细胞及其用途
SG11202003907WA (en) 2017-12-14 2020-05-28 Flodesign Sonics Inc Acoustic transducer drive and controller
CA3087527A1 (en) * 2018-01-03 2019-07-11 Magenta Therapeutics, Inc. Compositions and methods for the expansion of hematopoietic stem and progenitor cells and treatment of inherited metabolic disorders
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
EP3749767A1 (en) * 2018-02-05 2020-12-16 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
WO2019150203A1 (en) * 2018-02-05 2019-08-08 Crispr Therapeutics Ag Materials and methods for 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
KR20210045360A (ko) * 2018-05-16 2021-04-26 신테고 코포레이션 가이드 rna 설계 및 사용을 위한 방법 및 시스템
CA3113255A1 (en) 2018-09-21 2020-03-26 APstem Therapeutics, Inc. Human pluripotent adult stem cells
KR20210143230A (ko) 2019-03-19 2021-11-26 더 브로드 인스티튜트, 인코퍼레이티드 뉴클레오티드 서열을 편집하기 위한 방법 및 조성물
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
EA202192931A1 (ru) * 2019-04-30 2022-02-22 Эдиджен Инк. Способ прогнозирования эффективности лечения гемоглобинопатии
CN114364799A (zh) * 2019-08-28 2022-04-15 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
CN116096873A (zh) 2020-05-08 2023-05-09 布罗德研究所股份有限公司 同时编辑靶标双链核苷酸序列的两条链的方法和组合物
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
WO2022240787A1 (en) * 2021-05-13 2022-11-17 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

Family Cites Families (32)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1709195B1 (en) 2003-12-19 2014-01-22 Novartis Vaccines and Diagnostics, Inc. Cell transfecting formulations of small interfering rna, related compositions and methods of making and use
PE20100362A1 (es) * 2008-10-30 2010-05-27 Irm Llc Derivados de purina que expanden las celulas madre hematopoyeticas
JP2013515693A (ja) 2009-12-23 2013-05-09 ノバルティス アーゲー 脂質、脂質組成物およびそれらの使用方法
US20130171241A1 (en) 2010-07-06 2013-07-04 Novartis Ag Liposomes with lipids having an advantageous pka-value for rna delivery
EP2611420B1 (en) 2010-08-31 2019-03-27 GlaxoSmithKline Biologicals SA Lipids suitable for liposomal delivery of protein-coding rna
EP4014966A1 (en) 2011-07-06 2022-06-22 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
SI2807165T1 (sl) * 2012-01-27 2019-08-30 Universite De Montreal Pirimido(4,5-B)indol derivati in njihova uporaba pri ekspanziji hematopoetskih matičnih celic
DK2836226T3 (en) * 2012-02-24 2017-09-18 Hutchinson Fred Cancer Res COMPOSITIONS AND PROCEDURES FOR TREATING HEMOGLOBINOPATHY
ES2884925T3 (es) * 2012-11-27 2021-12-13 Childrens Medical Ct Corp Elementos reguladores distales de BCL11A como dianas para la reinducción de la hemoglobina fetal
US8697359B1 (en) * 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
US10124065B2 (en) 2013-03-08 2018-11-13 Novartis Ag Lipids and lipid compositions for the delivery of active agents
PL2970948T3 (pl) 2013-03-15 2019-06-28 Glaxosmithkline Biologicals Sa Sposoby oczyszczania rna
EP3865586A1 (en) * 2013-03-15 2021-08-18 The General Hospital Corporation Increasing specificity for rna-guided genome editing
CA2910489A1 (en) * 2013-05-15 2014-11-20 Sangamo Biosciences, Inc. Methods and compositions for treatment of a genetic condition
KR20160034901A (ko) * 2013-06-17 2016-03-30 더 브로드 인스티튜트, 인코퍼레이티드 서열 조작에 최적화된 ｃｒｉｓｐｒ-ｃａｓ 이중 닉카아제 시스템, 방법 및 조성물
ES2865107T3 (es) 2013-08-16 2021-10-15 Massachusetts Inst Technology Administración selectiva de material a células
RS62559B1 (sr) * 2013-11-13 2021-12-31 Childrens Medical Center Nukleazom posredovana regulacija eksprimiranja gena
CN106103699B (zh) * 2013-11-28 2019-11-26 地平线探索有限公司 体细胞单倍体人类细胞系
ES2774968T3 (es) 2013-12-19 2020-07-23 Novartis Ag Lípidos y composiciones lipídicas para la administración de agentes activos
ES2908827T3 (es) 2013-12-19 2022-05-04 Novartis Ag Lípidos y composiciones lipídicas para el suministro de agentes activos
WO2015148863A2 (en) * 2014-03-26 2015-10-01 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
WO2015148860A1 (en) * 2014-03-26 2015-10-01 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating beta-thalassemia
WO2015152305A1 (ja) * 2014-03-31 2015-10-08 株式会社サイエンス・ラスター 造血細胞の増殖ペプチドおよびその用途
SG10201809290SA (en) * 2014-04-25 2019-01-30 Childrens Medical Ct Corp Compositions and Methods to Treating Hemoglobinopathies
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
US11572543B2 (en) * 2015-05-08 2023-02-07 The Children's Medical Center. Corporation Targeting BCL11A enhancer functional regions for fetal hemoglobin reinduction
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
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
CN112020558A (zh) * 2018-03-14 2020-12-01 爱迪塔斯医药公司 治疗血红蛋白病的系统和方法

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