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 PDFInfo
- 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- Withdrawn
Links
- 108020004635 Complementary DNA Proteins 0.000 title 1
- 229960000301 factor viii Drugs 0.000 title 1
- 239000000203 mixture Substances 0.000 title 1
- 230000035772 mutation Effects 0.000 title 1
- 230000024664 tolerance induction Effects 0.000 title 1
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 RNAses, DNAses
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)
- Epidemiology (AREA)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Crystallography & Structural Chemistry (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
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)
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 |
CA2853829C (en) | 2011-07-22 | 2023-09-26 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
BR112015013311A2 (en) | 2012-12-07 | 2017-11-14 | Haplomics Inc | tolerance induction and factor 8 mutation repair |
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 |
US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
US9737604B2 (en) | 2013-09-06 | 2017-08-22 | President And Fellows Of Harvard College | Use of cationic lipids to deliver CAS9 |
US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
US9068179B1 (en) | 2013-12-12 | 2015-06-30 | President And Fellows Of Harvard College | Methods for correcting presenilin point mutations |
EP4079847A1 (en) | 2014-07-30 | 2022-10-26 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
IL258821B (en) | 2015-10-23 | 2022-07-01 | Harvard College | Nucleobase editors and uses thereof |
WO2017112895A1 (en) * | 2015-12-23 | 2017-06-29 | Haplomics, Inc. | F8 gene repair |
KR102547316B1 (en) | 2016-08-03 | 2023-06-23 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | Adenosine nucleobase editing agents and uses thereof |
AU2017308889B2 (en) | 2016-08-09 | 2023-11-09 | President And Fellows Of Harvard College | Programmable Cas9-recombinase fusion proteins and uses thereof |
US11542509B2 (en) | 2016-08-24 | 2023-01-03 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
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 |
EP3592853A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Suppression of pain by gene editing |
US11542496B2 (en) | 2017-03-10 | 2023-01-03 | President And Fellows Of Harvard College | Cytosine to guanine base editor |
KR102687373B1 (en) | 2017-03-23 | 2024-07-23 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | Nucleobase editing agent comprising a nucleic acid programmable DNA binding protein |
WO2018209320A1 (en) | 2017-05-12 | 2018-11-15 | President And Fellows Of Harvard College | Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation |
CN111801345A (en) | 2017-07-28 | 2020-10-20 | 哈佛大学的校长及成员们 | Methods and compositions using an evolved base editor for 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 |
US11795443B2 (en) | 2017-10-16 | 2023-10-24 | The Broad Institute, Inc. | Uses of adenosine base editors |
AU2020221340A1 (en) * | 2019-02-15 | 2021-09-16 | Bayer Healthcare Llc | Gene editing for hemophilia A with improved Factor VIII expression |
JP2022526908A (en) | 2019-03-19 | 2022-05-27 | ザ ブロード インスティテュート,インコーポレーテッド | Editing Methods and Compositions for Editing Nucleotide Sequences |
AU2021267940A1 (en) | 2020-05-08 | 2022-12-08 | President And Fellows Of Harvard College | Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence |
Citations (1)
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)
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 |
CA2814143C (en) * | 2010-10-12 | 2020-09-08 | The Children's Hospital Of Philadelphia | Methods and compositions for treating hemophilia b |
BR112015013311A2 (en) * | 2012-12-07 | 2017-11-14 | Haplomics Inc | tolerance induction and factor 8 mutation repair |
-
2015
- 2015-06-11 WO PCT/US2015/035399 patent/WO2015191899A1/en active Application Filing
- 2015-06-11 EP EP15807028.4A patent/EP3155098A4/en not_active Withdrawn
- 2015-06-11 CA CA2951882A patent/CA2951882A1/en not_active Abandoned
Patent Citations (1)
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)
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 |
---|---|
WO2015191899A9 (en) | 2017-05-11 |
CA2951882A1 (en) | 2015-12-17 |
EP3155098A1 (en) | 2017-04-19 |
WO2015191899A1 (en) | 2015-12-17 |
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PUAI | Public reference made under article 153(3) epc to a published international application that has entered the european phase |
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17P | Request for examination filed |
Effective date: 20170110 |
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AK | Designated contracting states |
Kind code of ref document: A1 Designated state(s): AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR |
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AX | Request for extension of the european patent |
Extension state: BA ME |
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DAV | Request for validation of the european patent (deleted) | ||
DAX | Request for extension of the european patent (deleted) | ||
A4 | Supplementary search report drawn up and despatched |
Effective date: 20171130 |
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RIC1 | Information provided on ipc code assigned before grant |
Ipc: C12N 15/12 20060101ALI20171124BHEP Ipc: C07K 14/755 20060101ALI20171124BHEP Ipc: C12N 15/09 20060101AFI20171124BHEP Ipc: C12N 9/10 20060101ALI20171124BHEP Ipc: C12N 9/16 20060101ALI20171124BHEP Ipc: A61P 7/00 20060101ALI20171124BHEP Ipc: A61K 48/00 20060101ALI20171124BHEP |
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STAA | Information on the status of an ep patent application or granted ep patent |
Free format text: STATUS: THE APPLICATION IS DEEMED TO BE WITHDRAWN |
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18D | Application deemed to be withdrawn |
Effective date: 20180703 |