SG11201706767RA - Materials and methods for treatment of hemoglobinopathies - Google Patents

Materials and methods for treatment of hemoglobinopathies

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Publication number
SG11201706767RA
SG11201706767RA SG11201706767RA SG11201706767RA SG11201706767RA SG 11201706767R A SG11201706767R A SG 11201706767RA SG 11201706767R A SG11201706767R A SG 11201706767RA SG 11201706767R A SG11201706767R A SG 11201706767RA SG 11201706767R A SG11201706767R A SG 11201706767RA
Authority
SG
Singapore
Prior art keywords
hemoglobinopathies
treatment
materials
methods
Prior art date
Application number
SG11201706767RA
Other languages
English (en)
Inventor
Matthew Hebden Porteus
Thomas James Cradick
Gang Bao
Ciaran M Lee
Original Assignee
Crispr Therapeutics Ag
Georgia Tech Res Inst
Univ Rice William M
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 Crispr Therapeutics Ag, Georgia Tech Res Inst, Univ Rice William M filed Critical Crispr Therapeutics Ag
Publication of SG11201706767RA publication Critical patent/SG11201706767RA/en

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SG11201706767RA 2015-02-23 2016-02-23 Materials and methods for treatment of hemoglobinopathies SG11201706767RA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562119754P 2015-02-23 2015-02-23
PCT/IB2016/000282 WO2016135558A2 (fr) 2015-02-23 2016-02-23 Matériels et méthodes pour le traitement des hémoglobinopathies

Publications (1)

Publication Number Publication Date
SG11201706767RA true SG11201706767RA (en) 2017-09-28

Family

ID=55795007

Family Applications (2)

Application Number Title Priority Date Filing Date
SG11201706767RA SG11201706767RA (en) 2015-02-23 2016-02-23 Materials and methods for treatment of hemoglobinopathies
SG11201706766WA SG11201706766WA (en) 2015-02-23 2016-02-23 Materials and methods for treatment of hemoglobinopathies

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SG11201706766WA SG11201706766WA (en) 2015-02-23 2016-02-23 Materials and methods for treatment of hemoglobinopathies

Country Status (8)

Country Link
US (3) US10738305B2 (fr)
EP (2) EP3262172A2 (fr)
CN (2) CN107532168A (fr)
AU (2) AU2016225178B2 (fr)
BR (2) BR112017017812A2 (fr)
CA (2) CA2977455A1 (fr)
SG (2) SG11201706767RA (fr)
WO (2) WO2016135558A2 (fr)

Families Citing this family (56)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013066438A2 (fr) 2011-07-22 2013-05-10 President And Fellows Of Harvard College Évaluation et amélioration de la spécificité de clivage des nucléases
KR20200136508A (ko) 2013-04-16 2020-12-07 리제너론 파마슈티칼스 인코포레이티드 랫트 게놈의 표적화된 변형
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
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
US9340800B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College Extended DNA-sensing GRNAS
MX2016007654A (es) 2013-12-11 2017-08-15 Regeneron Pharma Metodos y composiciones para la modificacion dirigida de un genoma.
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
WO2015148863A2 (fr) 2014-03-26 2015-10-01 Editas Medicine, Inc. Méthodes liées à crispr/cas et compositions pour le traitement de la drépanocytose
CA2956224A1 (fr) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Proteines cas9 comprenant des inteines dependant de ligands
NZ731962A (en) 2014-11-21 2022-07-01 Regeneron Pharma Methods and compositions for targeted genetic modification using paired guide rnas
EP3262172A2 (fr) 2015-02-23 2018-01-03 Crispr Therapeutics AG Matériels et méthodes pour le traitement des hémoglobinopathies
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
CN108026526B (zh) 2015-06-09 2023-05-12 爱迪塔斯医药公司 用于改善移植的crispr/cas相关方法和组合物
WO2017053729A1 (fr) 2015-09-25 2017-03-30 The Board Of Trustees Of The Leland Stanford Junior University Édition du génome à médiation par une nucléase de cellules primaires et leur enrichissement
EP4269577A3 (fr) 2015-10-23 2024-01-17 President and Fellows of Harvard College Éditeurs de nucleobases et leurs utilisations
ES2942309T3 (es) * 2015-11-04 2023-05-31 Vertex Pharma Materiales y métodos para el tratamiento de hemoglobinopatías
KR20230070331A (ko) * 2016-03-14 2023-05-22 에디타스 메디신, 인코포레이티드 베타 이상헤모글로빈증의 치료를 위한 crispr/cas-관련 방법 및 조성물
IL264565B1 (en) 2016-08-03 2024-03-01 Harvard College Adenosine nuclear base editors and their uses
EP3497214B1 (fr) 2016-08-09 2023-06-28 President and Fellows of Harvard College Protéines de fusion cas9-recombinase programmables et utilisations associées
WO2018039438A1 (fr) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation d'acides aminés non naturels dans des protéines au moyen de l'édition de bases
SG11201903089RA (en) 2016-10-14 2019-05-30 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
EP3573618B1 (fr) * 2017-01-30 2023-08-16 The Children's Hospital of Philadelphia Compositions et méthodes de production d'hémoglobine
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 (fr) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression de la douleur par édition de gène
US11542496B2 (en) 2017-03-10 2023-01-03 President And Fellows Of Harvard College Cytosine to guanine base editor
WO2018170184A1 (fr) 2017-03-14 2018-09-20 Editas Medicine, Inc. Systèmes et méthodes pour le traitement d'hémoglobinopathies
EP3601562A1 (fr) 2017-03-23 2020-02-05 President and Fellows of Harvard College Éditeurs de nucléobase comprenant des protéines de liaison à l'adn programmable par acides nucléiques
EP3622070A2 (fr) 2017-05-10 2020-03-18 Editas Medicine, Inc. Crispr/arn-guidé systèmes et procédés nucléases transgéniques
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
WO2019003193A1 (fr) * 2017-06-30 2019-01-03 Novartis Ag Méthodes pour le traitement d'une maladie à l'aide de systèmes d'édition de gènes
EP3652312A1 (fr) 2017-07-14 2020-05-20 Editas Medicine, Inc. Systèmes et procédés d'intégration ciblée et d'édition du génome et détection de celle-ci à l'aide de sites d'amorçage intégrés
EP3658573A1 (fr) 2017-07-28 2020-06-03 President and Fellows of Harvard College Procédés et compositions pour l'évolution d'éditeurs de bases à l'aide d'une évolution continue assistée par phage (pace)
WO2019139645A2 (fr) 2017-08-30 2019-07-18 President And Fellows Of Harvard College Éditeurs de bases à haut rendement comprenant une gam
WO2019079347A1 (fr) 2017-10-16 2019-04-25 The Broad Institute, Inc. Utilisations d'éditeurs de bases adénosine
US20210180091A1 (en) * 2017-10-26 2021-06-17 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
CN108103027B (zh) * 2018-02-02 2021-12-24 中国医学科学院血液病医院(血液学研究所) 高效率血细胞重编程同时实现基因编辑的方法
WO2019150203A1 (fr) 2018-02-05 2019-08-08 Crispr Therapeutics Ag Substances et méthodes pour traiter des hémoglobinopathies
EP3762496A2 (fr) * 2018-03-07 2021-01-13 Editas Medicine, Inc. Systèmes et procédés pour le traitement d'hémoglobinopathies
WO2019178416A1 (fr) * 2018-03-14 2019-09-19 Editas Medicine, Inc. Systèmes et méthodes pour le traitement d'hémoglobinopathies
EP3802826A1 (fr) * 2018-05-24 2021-04-14 CRISPR Therapeutics AG Procédés et compositions pour la délétion de gène efficace
CN110863041A (zh) * 2018-08-27 2020-03-06 深圳华大生命科学研究院 一种与地中海贫血相关的突变基因及其检测试剂和应用
SG11202104346YA (en) * 2018-10-30 2021-05-28 Crispr Therapeutics Ag Compositions and methods for nhej-mediated genome editing
KR20220013541A (ko) * 2019-02-26 2022-02-04 퍼크어시스트, 인크. 경피성 공압식 심장 보조 장치, 시스템 및 방법
WO2020191246A1 (fr) 2019-03-19 2020-09-24 The Broad Institute, Inc. Procédés et compositions pour l'édition de séquences nucléotidiques
CN111939271A (zh) * 2019-04-30 2020-11-17 博雅辑因(北京)生物科技有限公司 一种血红蛋白病治疗有效性预测方法
KR20220002565A (ko) * 2019-04-30 2022-01-06 에디진 인크. 이상헤모글로빈증 치료의 효과를 예측하는 방법
EP3983542A2 (fr) 2019-06-17 2022-04-20 CRISPR Therapeutics AG Méthodes et compositions pour la réparation dirigée par l'homologie améliorée
CN110699381A (zh) * 2019-09-17 2020-01-17 合肥瑞灵生物科技有限公司 地中海贫血病基因治疗载体构建方法及其用途
GB2614813A (en) 2020-05-08 2023-07-19 Harvard College Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
WO2022147759A1 (fr) * 2021-01-08 2022-07-14 Susheng Biotech (Hainan) Co., Ltd. Molécule d'arng ciblant l'intron i ou l'intron ii du gène hbb, son procédé de synthèse et procédé pour corriger les types de mutations du gène hbb
WO2023028469A2 (fr) * 2021-08-23 2023-03-02 The Board Of Trustees Of The Leland Stanford Junior University Intégration ciblée au niveau du locus de la bêta-globine dans des cellules souches et progénitrices hématopoïétiques humaines
CN113564248A (zh) * 2021-09-26 2021-10-29 北京贝瑞和康生物技术有限公司 同时检测hba1/2、hbb和hbd基因位点多种突变的方法和试剂盒
WO2024073751A1 (fr) 2022-09-29 2024-04-04 Vor Biopharma Inc. Procédés et compositions pour la modification et l'enrichissement de gènes

Family Cites Families (49)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FR901228A (fr) 1943-01-16 1945-07-20 Deutsche Edelstahlwerke Ag Système d'aimant à entrefer annulaire
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
AU678867B2 (en) 1993-11-09 1997-06-12 Medical College Of Ohio, The Stable cell lines capable of expressing the adeno-associated virus replication gene
EP0733103B1 (fr) 1993-11-09 2004-03-03 Targeted Genetics Corporation Production de titres eleves de vecteurs d'aav recombinants
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
AU707866B2 (en) 1994-12-06 1999-07-22 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant AAV vectors
FR2737730B1 (fr) 1995-08-10 1997-09-05 Pasteur Merieux Serums Vacc Procede de purification de virus par chromatographie
JPH11511326A (ja) 1995-08-30 1999-10-05 ジエンザイム コーポレイション アデノウィルスおよびaavの精製
US6632670B1 (en) 1995-09-08 2003-10-14 Genzyme Corporation AAV vectors for gene therapy
US5910434A (en) 1995-12-15 1999-06-08 Systemix, Inc. Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant
ES2557997T3 (es) 1997-09-05 2016-02-01 Genzyme Corporation Métodos para generar preparados de vectores AAV de título elevado sin virus auxiliar
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
AU5557501A (en) 2000-04-28 2001-11-12 Univ Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
AU2002214127B2 (en) 2000-10-27 2007-06-07 J. Craig Venter Institute, Inc. Nucleic acids and proteins from streptococcus groups A and B
ES2738980T3 (es) 2008-09-15 2020-01-28 Childrens Medical Ct Corp Modulación de BCL11A para el tratamiento de hemoglobinopatías
CA2865129A1 (fr) * 2012-02-24 2013-08-29 Fred Hutchinson Cancer Research Center Compositions et methodes pour le traitement d'hemoglobinopathies
DE102012007232B4 (de) 2012-04-07 2014-03-13 Susanne Weller Verfahren zur Herstellung von rotierenden elektrischen Maschinen
PE20190843A1 (es) 2012-05-25 2019-06-17 Emmanuelle Charpentier Arn de direccion a adn generico
RS60838B1 (sr) 2012-08-29 2020-10-30 Sangamo Therapeutics Inc Postupci i kompozicije za tretman genskog stanja
US20140085593A1 (en) 2012-09-21 2014-03-27 Shenzhen China Star Optoelectronics Technology Co., Ltd Mixture for Liquid Crystal Medium and Liquid Crystal Display Using the Same
DK2925864T3 (en) 2012-11-27 2019-02-11 Childrens Medical Ct Corp DIRECTIONAL TARGETING OF DISTANT BCL11A CONTROLS FOR FETAL HEMOGLOBIN REINUCTION
CN113528577A (zh) 2012-12-12 2021-10-22 布罗德研究所有限公司 用于序列操纵的系统、方法和优化的指导组合物的工程化
CN116083487A (zh) 2013-05-15 2023-05-09 桑格摩生物治疗股份有限公司 用于治疗遗传病状的方法和组合物
KR102551324B1 (ko) 2013-06-05 2023-07-05 듀크 유니버시티 Rna-가이드 유전자 편집 및 유전자 조절
EP3019595A4 (fr) * 2013-07-09 2016-11-30 Utilisations thérapeutiques d'édition du génome avec des systèmes crispr/cas
PT3019619T (pt) 2013-07-11 2021-11-11 Modernatx Inc Composições que compreendem polinucleótidos sintéticos que codificam proteínas relacionadas com crispr e sgarn sintéticos e métodos de utilização
WO2015021426A1 (fr) 2013-08-09 2015-02-12 Sage Labs, Inc. Nouvelle protéine de fusion à base de système crispr/cas et son application en édition de génome
WO2015026883A1 (fr) 2013-08-22 2015-02-26 E. I. Du Pont De Nemours And Company Modification du génome des plantes à l'aide de systèmes d'arn de guidage/endonucléase cas et leurs procédés d'utilisation
JP2015092462A (ja) 2013-09-30 2015-05-14 Tdk株式会社 正極及びそれを用いたリチウムイオン二次電池
US10354746B2 (en) 2014-01-27 2019-07-16 Georgia Tech Research Corporation Methods and systems for identifying CRISPR/Cas off-target sites
JP6202701B2 (ja) 2014-03-21 2017-09-27 株式会社日立国際電気 基板処理装置、半導体装置の製造方法及びプログラム
WO2015148863A2 (fr) 2014-03-26 2015-10-01 Editas Medicine, Inc. Méthodes liées à crispr/cas et compositions pour le traitement de la drépanocytose
WO2015183025A1 (fr) 2014-05-28 2015-12-03 주식회사 툴젠 Procédé permettant la détection sensible d'adn cible à l'aide d'une nucléase spécifique de cible
JP6197169B2 (ja) 2014-09-29 2017-09-20 東芝メモリ株式会社 半導体装置の製造方法
KR20240013283A (ko) 2014-12-03 2024-01-30 애질런트 테크놀로지스, 인크. 화학적 변형을 갖는 가이드 rna
WO2016135559A2 (fr) 2015-02-23 2016-09-01 Crispr Therapeutics Ag Matériaux et procédés pour le traitement de maladies génétiques humaines, y compris d'hémoglobinopathies
EP3262172A2 (fr) 2015-02-23 2018-01-03 Crispr Therapeutics AG Matériels et méthodes pour le traitement des hémoglobinopathies
EP3274454B1 (fr) 2015-03-25 2021-08-25 Editas Medicine, Inc. Procédés, compositions et constituants liés à crispr/cas
ES2884838T3 (es) 2015-04-06 2021-12-13 Univ Leland Stanford Junior ARN guía químicamente modificados para la regulación génica mediada por CRISPR/CAS
AU2016285724A1 (en) 2015-06-29 2017-11-16 Ionis Pharmaceuticals, Inc. Modified CRISPR RNA and modified single CRISPR RNA and uses thereof
ES2942309T3 (es) 2015-11-04 2023-05-31 Vertex Pharma Materiales y métodos para el tratamiento de hemoglobinopatías
CN108779462A (zh) 2015-12-28 2018-11-09 诺华股份有限公司 用于治疗血红蛋白病的组合物和方法
KR20230070331A (ko) 2016-03-14 2023-05-22 에디타스 메디신, 인코포레이티드 베타 이상헤모글로빈증의 치료를 위한 crispr/cas-관련 방법 및 조성물
LT3445388T (lt) 2016-04-18 2024-06-10 Vertex Pharmaceuticals Incorporated Hemoglobinopatijų gydymui skirtos medžiagos ir būdai
WO2017191503A1 (fr) 2016-05-05 2017-11-09 Crispr Therapeutics Ag Substances et méthodes pour le traitement d'hémoglobinopathies
EP3532079A4 (fr) 2016-10-27 2020-07-08 Intima Bioscience, Inc. Procédés viraux de thérapie par lymphocytes t
US20210180091A1 (en) 2017-10-26 2021-06-17 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
WO2019150203A1 (fr) 2018-02-05 2019-08-08 Crispr Therapeutics Ag Substances et méthodes pour traiter des hémoglobinopathies

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US20180021413A1 (en) 2018-01-25
US20210009998A1 (en) 2021-01-14
CN107532168A (zh) 2018-01-02
CN107532182A (zh) 2018-01-02
WO2016135558A3 (fr) 2016-10-20
WO2016135558A2 (fr) 2016-09-01
WO2016135557A3 (fr) 2016-10-20
AU2016225178B2 (en) 2022-05-05
WO2016135557A2 (fr) 2016-09-01
CA2977447A1 (fr) 2016-09-01
SG11201706766WA (en) 2017-09-28
AU2016225178A1 (en) 2017-08-31
BR112017017812A2 (pt) 2018-04-10
BR112017017810A2 (pt) 2018-04-10
US20180030438A1 (en) 2018-02-01
EP3262171A2 (fr) 2018-01-03
AU2016225179B2 (en) 2022-05-05
AU2016225178A9 (en) 2022-05-05
AU2016225179C1 (en) 2022-11-03
EP3262172A2 (fr) 2018-01-03
US10738305B2 (en) 2020-08-11
AU2016225179A1 (en) 2017-08-31
US20210317450A9 (en) 2021-10-14

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