SG10201809290SA - Compositions and Methods to Treating Hemoglobinopathies - Google Patents

Compositions and Methods to Treating Hemoglobinopathies

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Publication number
SG10201809290SA
SG10201809290SA SG10201809290SA SG10201809290SA SG10201809290SA SG 10201809290S A SG10201809290S A SG 10201809290SA SG 10201809290S A SG10201809290S A SG 10201809290SA SG 10201809290S A SG10201809290S A SG 10201809290SA SG 10201809290S A SG10201809290S A SG 10201809290SA
Authority
SG
Singapore
Prior art keywords
methods
compositions
hemoglobinopathies
expression
treating hemoglobinopathies
Prior art date
Application number
SG10201809290SA
Other languages
English (en)
Inventor
Michael Milsom
David A Williams
Richard Gregory
Original Assignee
Childrens Medical Ct Corp
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 Childrens Medical Ct Corp filed Critical Childrens Medical Ct Corp
Publication of SG10201809290SA publication Critical patent/SG10201809290SA/en

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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
    • C12N15/1135—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 against oncogenes or tumor suppressor genes
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    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6806—Preparing nucleic acids for analysis, e.g. for polymerase chain reaction [PCR] assay
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • 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
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
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    • 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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    • 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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    • C12N2310/141—MicroRNAs, miRNAs
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
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    • C12N2740/00—Reverse transcribing RNA viruses
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    • C12N2740/10011—Retroviridae
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/10041—Use of virus, viral particle or viral elements as a vector
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15032—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16032—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent

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  • Health & Medical Sciences (AREA)
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  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
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  • Wood Science & Technology (AREA)
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  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
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  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Virology (AREA)
  • Hematology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Cell Biology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Epidemiology (AREA)
  • Diabetes (AREA)
  • Analytical Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Oncology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
SG10201809290SA 2014-04-25 2015-04-24 Compositions and Methods to Treating Hemoglobinopathies SG10201809290SA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201461984247P 2014-04-25 2014-04-25
US201462066783P 2014-10-21 2014-10-21

Publications (1)

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SG10201809290SA true SG10201809290SA (en) 2019-01-30

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Application Number Title Priority Date Filing Date
SG10201809290SA SG10201809290SA (en) 2014-04-25 2015-04-24 Compositions and Methods to Treating Hemoglobinopathies
SG11201608482UA SG11201608482UA (en) 2014-04-25 2015-04-24 Compositions and methods to treating hemoglobinopathies

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SG11201608482UA SG11201608482UA (en) 2014-04-25 2015-04-24 Compositions and methods to treating hemoglobinopathies

Country Status (17)

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US (4) US10287588B2 (da)
EP (2) EP3134130B1 (da)
JP (2) JP6514717B2 (da)
KR (1) KR102390629B1 (da)
CN (2) CN112852812A (da)
AU (1) AU2015249381B2 (da)
BR (1) BR112016024565A2 (da)
CA (1) CA2946309C (da)
DK (1) DK3134130T3 (da)
ES (1) ES2744186T3 (da)
IL (1) IL248469B (da)
MX (1) MX2016013832A (da)
RU (1) RU2707540C2 (da)
SA (1) SA516380143B1 (da)
SG (2) SG10201809290SA (da)
WO (1) WO2015164750A2 (da)
ZA (1) ZA201607540B (da)

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK3502240T3 (da) 2012-11-27 2021-08-09 Childrens Medical Ct Corp Målretning af distale bcl11a-reguleringselementer til føtal hæmoglobinreinduktion
ES2744186T3 (es) 2014-04-25 2020-02-24 Childrens Medical Center Composiciones y métodos para tratar las hemoglobinopatías
JP7288302B2 (ja) 2015-05-08 2023-06-07 ザ 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再誘導のための、ｂｃｌ１１ａエンハンサー機能性領域を標的とする方法
EA201891532A1 (ru) * 2015-12-28 2019-01-31 Новартис Аг Композиции и способы лечения гемоглобинопатий
US11655469B2 (en) * 2016-03-07 2023-05-23 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services MicroRNAs and methods of their use
EP3526319A4 (en) 2016-10-14 2020-07-29 Children's Medical Center Corporation METHOD AND COMPOSITIONS AND FOR TREATING DISEASES AND DISORDERS OF THE CENTRAL VENTILATION SYSTEM
AU2018210853B2 (en) 2017-01-17 2023-09-28 Children's Medical Center Corporation Compositions and methods for treating lysosomal storage diseases and disorders
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
BR112019020322A2 (pt) * 2017-03-29 2020-04-28 Bluebird Bio Inc vetores e composições para o tratamento de hemoglobinopatias
US11261441B2 (en) * 2017-03-29 2022-03-01 Bluebird Bio, Inc. Vectors and compositions for treating hemoglobinopathies
US11788087B2 (en) 2017-05-25 2023-10-17 The Children's Medical Center Corporation BCL11A guide delivery
CN111108207A (zh) * 2017-06-02 2020-05-05 国家健康与医学研究院 用于遗传障碍的基因疗法的基因组编辑手段和结合病毒载体的基因疗法
US10648728B2 (en) 2017-09-29 2020-05-12 Nxp Usa, Inc. Multifunctional radio frequency systems and methods for UV sterilization, air purification, and defrost operations
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
CA3084955A1 (en) * 2017-12-05 2019-06-13 Vertex Pharmaceuticals Incorporated Crispr-cas9 modified cd34+ human hematopoietic stem and progenitor cells and uses thereof
WO2019213013A1 (en) * 2018-05-02 2019-11-07 The Children's Medical Center Corporation Improved bcl11a micrornas for treating hemoglobinopathies
WO2020193434A1 (en) * 2019-03-22 2020-10-01 INSERM (Institut National de la Santé et de la Recherche Médicale) Bifunctional vectors allowing bcl11a silencing and expression of an anti-sickling hbb and uses thereof for gene therapy of b- hemoglobinopathies
WO2021067613A1 (en) * 2019-10-01 2021-04-08 Children's Medical Center Corporation Compositions and methods for treating amyotrophic lateral sclerosis
CA3174286A1 (en) 2020-04-02 2021-10-07 Robert PLACE Targeted inhibition using engineered oligonucleotides
WO2022266366A1 (en) * 2021-06-16 2022-12-22 Duke University Compositions and methods for the prevention and treatment of hemoglobinopathies
CN114990164B (zh) * 2022-06-16 2023-12-05 北京大学 中介体复合物亚基抑制剂及其应用

Family Cites Families (49)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5061620A (en) 1990-03-30 1991-10-29 Systemix, Inc. Human hematopoietic stem cell
US5635387A (en) 1990-04-23 1997-06-03 Cellpro, Inc. Methods and device for culturing human hematopoietic cells and their precursors
CA2131620A1 (en) 1992-03-20 1993-09-30 Louis C. Smith A dna transporter system and method of use
US5460964A (en) 1992-04-03 1995-10-24 Regents Of The University Of Minnesota Method for culturing hematopoietic cells
US5409813A (en) 1993-09-30 1995-04-25 Systemix, Inc. Method for mammalian cell separation from a mixture of cell populations
US5677136A (en) 1994-11-14 1997-10-14 Systemix, Inc. Methods of obtaining compositions enriched for hematopoietic stem cells, compositions derived therefrom and methods of use thereof
US5928638A (en) 1996-06-17 1999-07-27 Systemix, Inc. Methods for gene transfer
US8101349B2 (en) 1997-12-23 2012-01-24 Novartis Vaccines And Diagnostics, Inc. Gene products differentially expressed in cancerous cells and their methods of use II
FR2777909B1 (fr) 1998-04-24 2002-08-02 Pasteur Institut Utilisation de sequences d'adn de structure triplex pour le tranfert de sequences de nucleotides dans des cellules, vecteurs recombinants contenant ces sequences triplex
AU3988799A (en) 1998-05-13 1999-11-29 Genetix Pharmaceuticals, Inc. Novel lentiviral packaging cells
SI1407044T2 (en) 2000-12-01 2018-03-30 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Small RNA molecules that mediate RNA interference
EP1560931B1 (en) 2002-11-14 2011-07-27 Dharmacon, Inc. Functional and hyperfunctional sirna
ATE474460T1 (de) 2002-12-13 2010-08-15 Genetix Pharmaceuticals Inc Therapeutische retrovirus-vektoren für gentherapie
EP1752536A4 (en) 2004-05-11 2008-04-16 Alphagen Co Ltd POLYNUCLEOTIDE CAUSING RNA INTERFERENCE AND METHOD OF REGULATING GENE EXPRESSION WITH THE USE OF THE SAME
CA2633754C (en) * 2006-01-05 2013-06-18 The Ohio State University Research Foundation Microrna-based methods and compositions for the diagnosis and treatment of solid cancers
US20080051431A1 (en) 2006-05-26 2008-02-28 Dominique Verhelle Methods and compositions using immunomodulatory compounds in combination therapy
WO2008153029A1 (ja) 2007-06-11 2008-12-18 Takara Bio Inc. 特異的遺伝子発現方法
GB0713183D0 (en) 2007-07-06 2007-08-15 King S College London Method
MX345116B (es) 2008-09-15 2017-01-17 Children's Medical Center Corp Modulacion de bcl11a para tratamiento de hemoglobinopatias.
US20110294114A1 (en) 2009-12-04 2011-12-01 Cincinnati Children's Hospital Medical Center Optimization of determinants for successful genetic correction of diseases, mediated by hematopoietic stem cells
CN102802412A (zh) 2009-12-08 2012-11-28 海玛奎斯特医药公司 用于治疗红细胞病症的方法及低剂量方案
SI2561078T1 (sl) 2010-04-23 2019-01-31 Cold Spring Harbor Laboratory Nove strukturno oblikovane shRNA
GB201012420D0 (en) * 2010-07-23 2010-09-08 Univ Erasmus Medical Ct Foetal heamoglobin inhibitor
WO2012073047A2 (en) 2010-12-03 2012-06-07 Genome Research Limited Compositions and methods
WO2012079046A2 (en) * 2010-12-10 2012-06-14 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of klf-1 and bcl11a genes
EP3492108A1 (en) 2011-06-10 2019-06-05 Bluebird Bio, Inc. Gene therapy vectors for adrenoleukodystrophy and adrenomyeloneuropathy
EP3269802B1 (en) 2011-09-30 2019-10-23 Bluebird Bio, Inc. Compounds for improved viral transduction
SG10201606959PA (en) 2012-02-24 2016-09-29 Hutchinson Fred Cancer Res Compositions and methods for the treatment of hemoglobinopathies
US10174315B2 (en) 2012-05-16 2019-01-08 The General Hospital Corporation Compositions and methods for modulating hemoglobin gene family expression
ES2728782T3 (es) 2012-05-25 2019-10-28 Univ California Métodos y composiciones para la modificación de ADN objetivo dirigida por ARN y para la modulación de la transcripción dirigida por ARN
DK3502240T3 (da) 2012-11-27 2021-08-09 Childrens Medical Ct Corp Målretning af distale bcl11a-reguleringselementer til føtal hæmoglobinreinduktion
IL307735A (en) 2012-12-12 2023-12-01 Broad Inst Inc Systems engineering, methods and optimal guiding components for sequence manipulation
US10106816B2 (en) 2012-12-14 2018-10-23 Case Western Reserve University Genomic RNA packaging enhancer element
CN105683376A (zh) 2013-05-15 2016-06-15 桑格摩生物科学股份有限公司 用于治疗遗传病状的方法和组合物
WO2015065964A1 (en) 2013-10-28 2015-05-07 The Broad Institute Inc. Functional genomics using crispr-cas systems, compositions, methods, screens and applications thereof
WO2015073683A2 (en) 2013-11-13 2015-05-21 Children's Medical Center Corporation Nuclease-mediated regulation of gene expression
US11242525B2 (en) 2014-03-26 2022-02-08 Editas Medicine, Inc. CRISPR/CAS-related methods and compositions for treating sickle cell disease
CN106536549B (zh) 2014-04-25 2020-01-17 蓝鸟生物公司 Mnd启动子嵌合抗原受体
ES2744186T3 (es) 2014-04-25 2020-02-24 Childrens Medical Center Composiciones y métodos para tratar las hemoglobinopatías
US20170049819A1 (en) 2014-04-25 2017-02-23 Bluebird Bio, Inc. Kappa/lambda chimeric antigen receptors
US10619156B2 (en) 2014-05-28 2020-04-14 The Regents Of The University Of California Hybrid tRNA/pre-miRNA molecules and methods of use
ES2750725T3 (es) 2014-12-12 2020-03-26 Bluebird Bio Inc Receptores de antígeno quiméricos BCMA
WO2016182893A1 (en) 2015-05-08 2016-11-17 Teh Broad Institute Inc. Functional genomics using crispr-cas systems for saturating mutagenesis of non-coding elements, compositions, methods, libraries and applications thereof
EP3294879A4 (en) 2015-05-14 2019-02-20 University of Southern California OPTIMIZED GENETIZATION WITH A RECOMBINANT ENDONUCLEASE SYSTEM
WO2017040529A1 (en) 2015-08-31 2017-03-09 Bluebird Bio, Inc. Anti-sialyl tn chimeric antigen receptors
GB201522243D0 (en) 2015-12-16 2016-01-27 Ucl Business Plc Treatment
SG11201806758VA (en) 2016-02-12 2018-09-27 Bluebird Bio Inc Vcn enhancer compositions and methods of using the same
US20190093128A1 (en) 2016-03-31 2019-03-28 The Regents Of The University Of California Methods for genome editing in zygotes
US11788087B2 (en) 2017-05-25 2023-10-17 The Children's Medical Center Corporation BCL11A guide delivery

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CN112852812A (zh) 2021-05-28
DK3134130T3 (da) 2019-08-26
RU2016145964A (ru) 2018-05-25
SG11201608482UA (en) 2016-11-29
MX2016013832A (es) 2017-03-09
WO2015164750A2 (en) 2015-10-29
RU2016145964A3 (da) 2019-04-23
CN106794260B (zh) 2021-02-23
US20170218372A1 (en) 2017-08-03
ZA201607540B (en) 2019-04-24
JP2017513505A (ja) 2017-06-01
CA2946309C (en) 2021-11-09
CN106794260A (zh) 2017-05-31
EP3134130A2 (en) 2017-03-01
JP2019129846A (ja) 2019-08-08
SA516380143B1 (ar) 2020-05-14
EP3134130A4 (en) 2018-04-04
WO2015164750A3 (en) 2015-12-17
US20200370048A1 (en) 2020-11-26
US20190316136A1 (en) 2019-10-17
IL248469A0 (en) 2016-12-29
BR112016024565A2 (pt) 2018-01-23
ES2744186T3 (es) 2020-02-24
NZ726792A (en) 2020-09-25
IL248469B (en) 2020-04-30
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AU2015249381A1 (en) 2016-11-10
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US20220056453A1 (en) 2022-02-24
US11124794B2 (en) 2021-09-21
EP3134130B1 (en) 2019-06-12
RU2707540C2 (ru) 2019-11-27
KR102390629B1 (ko) 2022-04-26
AU2015249381B2 (en) 2020-04-30
EP3598984A1 (en) 2020-01-29
US10662429B2 (en) 2020-05-26
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