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EP3155098A4 - Réparation de mutations et induction de tolérance du facteur viii et adnc, compositions, procédés et systèmes associés - Google Patents

Réparation de mutations et induction de tolérance du facteur viii et adnc, compositions, procédés et systèmes associés 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
Authority
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Withdrawn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)
English (en)
Other versions
EP3155098A1 (fr
Inventor
Tom E. HOWARD
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Individual
Original Assignee
Individual
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 Individual filed Critical Individual
Publication of EP3155098A1 publication Critical patent/EP3155098A1/fr
Publication of EP3155098A4 publication Critical patent/EP3155098A4/fr
Withdrawn legal-status Critical Current

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Classifications

    • 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 [RNase]; Deoxyribonucleases [DNase]

Landscapes

  • 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 Réparation de mutations et induction de tolérance du facteur viii et adnc, compositions, procédés et systèmes associés Withdrawn EP3155098A4 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201462011019P 2014-06-11 2014-06-11
PCT/US2015/035399 WO2015191899A1 (fr) 2014-06-11 2015-06-11 Réparation de mutations et induction de tolérance du facteur viii et adnc, compositions, procédés et systèmes associés

Publications (2)

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

Family

ID=54834340

Family Applications (1)

Application Number Title Priority Date Filing Date
EP15807028.4A Withdrawn EP3155098A4 (fr) 2014-06-11 2015-06-11 Réparation de mutations et induction de tolérance du facteur viii et adnc, compositions, procédés et systèmes associés

Country Status (3)

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

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11083801B2 (en) 2012-12-07 2021-08-10 Haplomics, Inc. Factor VIII mutation repair and tolerance induction

Families Citing this family (33)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2006063031A2 (fr) 2004-12-06 2006-06-15 Haplomics Variants alleliques du facteur viii humain
WO2013066438A2 (fr) 2011-07-22 2013-05-10 President And Fellows Of Harvard College Évaluation et amélioration de la spécificité de clivage des nucléases
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
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
US12043852B2 (en) 2015-10-23 2024-07-23 President And Fellows Of Harvard College Evolved Cas9 proteins for gene editing
WO2017112895A1 (fr) * 2015-12-23 2017-06-29 Haplomics, Inc. Réparation du gène f8
KR20250103795A (ko) 2016-08-03 2025-07-07 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
CN109804066A (zh) 2016-08-09 2019-05-24 哈佛大学的校长及成员们 可编程cas9-重组酶融合蛋白及其用途
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
AU2017342543B2 (en) 2016-10-14 2024-06-27 President And Fellows Of Harvard College AAV delivery of nucleobase editors
WO2018119359A1 (fr) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Édition du gène récepteur ccr5 pour protéger contre l'infection par le vih
EP3592853A1 (fr) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression de la douleur par édition de gène
EP3592381A1 (fr) 2017-03-09 2020-01-15 President and Fellows of Harvard College Vaccin contre le cancer
KR20190127797A (ko) 2017-03-10 2019-11-13 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 시토신에서 구아닌으로의 염기 편집제
CA3057192A1 (fr) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Editeurs de nucleobase comprenant des proteines de liaison a l'adn programmable par acides nucleiques
WO2018209320A1 (fr) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Arn guides incorporés par aptazyme pour une utilisation avec crispr-cas9 dans l'édition du génome et l'activation transcriptionnelle
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 (fr) 2017-08-30 2019-07-18 President And Fellows Of Harvard College Éditeurs de bases à haut rendement comprenant une gam
CA3082251A1 (fr) 2017-10-16 2019-04-25 The Broad Institute, Inc. Utilisations d'editeurs de bases adenosine
EP3724214A4 (fr) 2017-12-15 2021-09-01 The Broad Institute Inc. Systèmes et procédés de prédiction des résultats de la réparation en ingénierie génétique
WO2019226953A1 (fr) 2018-05-23 2019-11-28 The Broad Institute, Inc. Éditeurs de bases et leurs utilisations
US12281338B2 (en) 2018-10-29 2025-04-22 The Broad Institute, Inc. Nucleobase editors comprising GeoCas9 and uses thereof
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
AU2020221340A1 (en) 2019-02-15 2021-09-16 Bayer Healthcare Llc Gene editing for hemophilia A with improved Factor VIII expression
DE112020001306T5 (de) 2019-03-19 2022-01-27 Massachusetts Institute Of Technology Verfahren und zusammensetzungen zur editierung von nukleotidsequenzen
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
JP2023525304A (ja) 2020-05-08 2023-06-15 ザ ブロード インスティテュート，インコーポレーテッド 標的二本鎖ヌクレオチド配列の両鎖同時編集のための方法および組成物

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2000017375A2 (fr) * 1998-09-23 2000-03-30 Vlaams Interuniversitair Instituut Voor Biotechnologie Vzw Procede de traitement de l'hemophilie par therapie genique in vivo a l'aide de vecteurs retroviraux

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20120149115A1 (en) * 2009-06-11 2012-06-14 Snu R&Db Foundation Targeted genomic rearrangements using site-specific nucleases
WO2012051343A1 (fr) * 2010-10-12 2012-04-19 The Children's Hospital Of Philadelphia Procédés et compositions pour traiter l'hémophilie 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 (fr) * 1998-09-23 2000-03-30 Vlaams Interuniversitair Instituut Voor Biotechnologie Vzw Procede de traitement de l'hemophilie par therapie genique in vivo a l'aide de vecteurs retroviraux

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 *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11083801B2 (en) 2012-12-07 2021-08-10 Haplomics, Inc. Factor VIII mutation repair and tolerance induction

Also Published As

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

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