EP3155098A4 - FACTOR VIII MUTATION REPAIR AND TOLERANCE INDUCTION AND RELATED CDNAs, COMPOSITIONS, METHODS AND SYSTEMS - Google Patents

FACTOR VIII MUTATION REPAIR AND TOLERANCE INDUCTION AND RELATED CDNAs, COMPOSITIONS, METHODS AND SYSTEMS Download PDF

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Publication number
EP3155098A4
EP3155098A4 EP15807028.4A EP15807028A EP3155098A4 EP 3155098 A4 EP3155098 A4 EP 3155098A4 EP 15807028 A EP15807028 A EP 15807028A EP 3155098 A4 EP3155098 A4 EP 3155098A4
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EP
European Patent Office
Prior art keywords
compositions
systems
methods
factor viii
tolerance induction
Prior art date
Legal status (The legal status 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 status listed.)
Withdrawn
Application number
EP15807028.4A
Other languages
German (de)
French (fr)
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EP3155098A1 (en
Inventor
Tom E. HOWARD
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Individual
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Publication of EP3155098A1 publication Critical patent/EP3155098A1/en
Publication of EP3155098A4 publication Critical patent/EP3155098A4/en
Withdrawn legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • 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
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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 RNAses, DNAses

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Hematology (AREA)
  • Physics & Mathematics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Cell Biology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Mycology (AREA)
  • Diabetes (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
EP15807028.4A 2014-06-11 2015-06-11 FACTOR VIII MUTATION REPAIR AND TOLERANCE INDUCTION AND RELATED CDNAs, COMPOSITIONS, METHODS AND SYSTEMS Withdrawn EP3155098A4 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201462011019P 2014-06-11 2014-06-11
PCT/US2015/035399 WO2015191899A1 (en) 2014-06-11 2015-06-11 FACTOR VIII MUTATION REPAIR AND TOLERANCE INDUCTION AND RELATED CDNAs, COMPOSITIONS, METHODS AND SYSTEMS

Publications (2)

Publication Number Publication Date
EP3155098A1 EP3155098A1 (en) 2017-04-19
EP3155098A4 true EP3155098A4 (en) 2018-01-03

Family

ID=54834340

Family Applications (1)

Application Number Title Priority Date Filing Date
EP15807028.4A Withdrawn EP3155098A4 (en) 2014-06-11 2015-06-11 FACTOR VIII MUTATION REPAIR AND TOLERANCE INDUCTION AND RELATED CDNAs, COMPOSITIONS, METHODS AND SYSTEMS

Country Status (3)

Country Link
EP (1) EP3155098A4 (en)
CA (1) CA2951882A1 (en)
WO (1) WO2015191899A1 (en)

Families Citing this family (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100256062A1 (en) 2004-12-06 2010-10-07 Howard Tommy E Allelic Variants of Human Factor VIII
EP3613852A3 (en) 2011-07-22 2020-04-22 President and Fellows of Harvard College Evaluation and improvement of nuclease cleavage specificity
US10272163B2 (en) 2012-12-07 2019-04-30 The Regents Of The University Of California Factor VIII mutation repair and tolerance induction
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
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
US9068179B1 (en) 2013-12-12 2015-06-30 President And Fellows Of Harvard College Methods for correcting presenilin point mutations
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
CN108513575A (en) 2015-10-23 2018-09-07 哈佛大学的校长及成员们 Nucleobase editing machine and application thereof
WO2017112895A1 (en) * 2015-12-23 2017-06-29 Haplomics, Inc. F8 gene repair
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
KR20240007715A (en) 2016-10-14 2024-01-16 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 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
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
JP2020510439A (en) 2017-03-10 2020-04-09 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 Base-editing factor from cytosine to guanine
SG11201908658TA (en) 2017-03-23 2019-10-30 Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
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
US11732274B2 (en) 2017-07-28 2023-08-22 President And Fellows Of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (PACE)
US11319532B2 (en) 2017-08-30 2022-05-03 President And Fellows Of Harvard College High efficiency base editors comprising Gam
CN111757937A (en) 2017-10-16 2020-10-09 布罗德研究所股份有限公司 Use of adenosine base editor
WO2020168362A1 (en) * 2019-02-15 2020-08-20 Crispr Therapeutics Ag Gene editing for hemophilia a with improved factor viii expression
DE112020001342T5 (en) 2019-03-19 2022-01-13 President and Fellows of Harvard College Methods and compositions for editing nucleotide sequences
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

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2000017375A2 (en) * 1998-09-23 2000-03-30 Vlaams Interuniversitair Instituut Voor Biotechnologie Vzw Method to treat haemophilia by in vivo gene therapy with retroviral vectors

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20100133319A (en) * 2009-06-11 2010-12-21 주식회사 툴젠 Targeted genomic rearrangements using site-specific nucleases
JP6018069B2 (en) * 2010-10-12 2016-11-02 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアＴｈｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ Ｏｆ Ｐｈｉｌａｄｅｌｐｈｉａ Methods and compositions for treating hemophilia B
US10272163B2 (en) * 2012-12-07 2019-04-30 The Regents Of The University Of California Factor VIII mutation repair and tolerance induction

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2000017375A2 (en) * 1998-09-23 2000-03-30 Vlaams Interuniversitair Instituut Voor Biotechnologie Vzw Method to treat haemophilia by in vivo gene therapy with retroviral vectors

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
C.-Y. PARK ET AL: "Targeted inversion and reversion of the blood coagulation factor 8 gene in human iPS cells using TALENs", PROCEEDINGS NATIONAL ACADEMY OF SCIENCES PNAS, vol. 111, no. 25, 9 June 2014 (2014-06-09), US, pages 9253 - 9258, XP055277137, ISSN: 0027-8424, DOI: 10.1073/pnas.1323941111 *
LIN YI ET AL: "Use of blood outgrowth endothelial cells for gene therapy for hemophilia A", BLOOD, AMERICAN SOCIETY OF HEMATOLOGY, US, vol. 99, no. 2, 15 January 2002 (2002-01-15), pages 457 - 462, XP002430423, ISSN: 0006-4971, DOI: 10.1182/BLOOD.V99.2.457 *
MATSUI HIDETO ET AL: "Ex vivo gene therapy for hemophilia a that enhances safe delivery and sustained in vivo factor VIII expression from lentivirally engineered endothelial progenitors", STEM CELLS (MIAMISBURG), vol. 25, no. 10, 2007, pages 2660 - 2669, XP055426660, ISSN: 1066-5099 *
See also references of WO2015191899A1 *
WALSH C E: "GENE THERAPY PROGRESS AND PROSPECTS: GENE THERAPY FOR THE HEMOPHILIAS", GENE THERAPY, NATURE PUBLISHING GROUP, GB, vol. 10, no. 12, 1 June 2003 (2003-06-01), pages 999 - 1003, XP001155785, ISSN: 0969-7128, DOI: 10.1038/SJ.GT.3302024 *

Also Published As

Publication number Publication date
CA2951882A1 (en) 2015-12-17
WO2015191899A1 (en) 2015-12-17
WO2015191899A9 (en) 2017-05-11
EP3155098A1 (en) 2017-04-19

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