WO2020127990A1 - Système de production de compositions pharmaceutiques à base de crispr - Google Patents

Système de production de compositions pharmaceutiques à base de crispr Download PDF

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Publication number
WO2020127990A1
WO2020127990A1 PCT/EP2019/086708 EP2019086708W WO2020127990A1 WO 2020127990 A1 WO2020127990 A1 WO 2020127990A1 EP 2019086708 W EP2019086708 W EP 2019086708W WO 2020127990 A1 WO2020127990 A1 WO 2020127990A1
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WO
WIPO (PCT)
Prior art keywords
sequence
rna
pathogen
protein
patient
Prior art date
Application number
PCT/EP2019/086708
Other languages
English (en)
Inventor
Theodore Anastasius PRAMER
Martin Ekenberg
Corinne Kay
Original Assignee
Biomedrex Ab
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 Biomedrex Ab filed Critical Biomedrex Ab
Publication of WO2020127990A1 publication Critical patent/WO2020127990A1/fr

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Classifications

    • GPHYSICS
    • G16INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16BBIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
    • G16B30/00ICT specially adapted for sequence analysis involving nucleotides or amino acids
    • G16B30/10Sequence alignment; Homology search

Abstract

L'invention concerne un système comprenant au moins un dispositif de séquençage et au moins un dispositif de synthèse d'ARN, le dispositif de séquençage comprenant un premier ordinateur et le dispositif de synthèse de nucléotides comprenant un second ordinateur, le dispositif de séquençage étant conçu pour a) recevoir au moins un échantillon biologique comprenant du matériel génétique se présentant sous la forme de polynucléotides, b) déterminer la séquence polynucléotidique du matériel génétique et la stocker sous la forme de données de séquence, c) fournir les données de séquence au dispositif de synthèse d'ARN; le dispositif de synthèse d'ARN étant conçu pour recevoir les données de séquence, le dispositif de séquençage ou le dispositif de synthèse d'ARN étant conçus pour stocker les données de séquence sous la forme d'un ensemble de données de séquence de patient représentant le génome du patient et d'un ensemble de données de séquence d'agent pathogène représentant au moins une partie du matériel génétique de l'agent pathogène, le dispositif de synthèse d'ARN étant conçu pour : d) trouver, dans l'ensemble de données de séquence d'agent pathogène, au moins une séquence polynucléotidique cible de 18 à 60 nucléotides de long, ladite séquence cible n'étant pas présente dans l'ensemble de données de séquence génétique du patient, e) synthétiser un brin antisens d'ARN pour une protéine de type CRISPR qui est complémentaire de la séquence cible de l'étape e), le brin antisens d'ARN comprenant en outre une séquence « poignée » prédéterminée qui médie la liaison à une protéine de type CRISPR.
PCT/EP2019/086708 2018-12-20 2019-12-20 Système de production de compositions pharmaceutiques à base de crispr WO2020127990A1 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
SE1851634-4 2018-12-20
SE1851634 2018-12-20

Publications (1)

Publication Number Publication Date
WO2020127990A1 true WO2020127990A1 (fr) 2020-06-25

Family

ID=69156392

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/EP2019/086708 WO2020127990A1 (fr) 2018-12-20 2019-12-20 Système de production de compositions pharmaceutiques à base de crispr

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WO (1) WO2020127990A1 (fr)

Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5846946A (en) 1996-06-14 1998-12-08 Pasteur Merieux Serums Et Vaccins Compositions and methods for administering Borrelia DNA
WO2003064026A1 (fr) 2002-01-31 2003-08-07 Nimblegen Systems Llc Substrat predessine, dispositif et procede pour la synthese optique de sondes d'adn
WO2012012779A2 (fr) 2010-07-23 2012-01-26 Beckman Coulter Inc. Système et procédé comprenant des unités analytiques
US8404658B2 (en) 2007-12-31 2013-03-26 Nanocor Therapeutics, Inc. RNA interference for the treatment of heart failure
US8454972B2 (en) 2004-07-16 2013-06-04 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Method for inducing a multiclade immune response against HIV utilizing a multigene and multiclade immunogen
US20160350476A1 (en) * 2015-05-29 2016-12-01 Agenovir Corporation Antiviral methods and compositions
US20180169658A1 (en) 2016-12-21 2018-06-21 Quandx Inc. Systems and methods for molecular diagnostics

Patent Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5846946A (en) 1996-06-14 1998-12-08 Pasteur Merieux Serums Et Vaccins Compositions and methods for administering Borrelia DNA
WO2003064026A1 (fr) 2002-01-31 2003-08-07 Nimblegen Systems Llc Substrat predessine, dispositif et procede pour la synthese optique de sondes d'adn
US8454972B2 (en) 2004-07-16 2013-06-04 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Method for inducing a multiclade immune response against HIV utilizing a multigene and multiclade immunogen
US8404658B2 (en) 2007-12-31 2013-03-26 Nanocor Therapeutics, Inc. RNA interference for the treatment of heart failure
WO2012012779A2 (fr) 2010-07-23 2012-01-26 Beckman Coulter Inc. Système et procédé comprenant des unités analytiques
US20160350476A1 (en) * 2015-05-29 2016-12-01 Agenovir Corporation Antiviral methods and compositions
US20180169658A1 (en) 2016-12-21 2018-06-21 Quandx Inc. Systems and methods for molecular diagnostics

Non-Patent Citations (17)

* Cited by examiner, † Cited by third party
Title
ABUDAYYEH ET AL., NATURE, vol. 550, 2017, pages 280 - 284
BERND ZETSCHE ET AL: "Cpf1 Is a Single RNA-Guided Endonuclease of a Class 2 CRISPR-Cas System", CELL, vol. 163, no. 3, 1 October 2015 (2015-10-01), AMSTERDAM, NL, pages 759 - 771, XP055553375, ISSN: 0092-8674, DOI: 10.1016/j.cell.2015.09.038 *
COXGOOTENBERGABUDAYYEH ET AL., SCIENCE, 2017
DE BUHR HENDRIK ET AL: "Harnessing CRISPR to combat human viral infections", CURRENT OPINION IN IMMUNOLOGY, ELSEVIER, OXFORD, GB, vol. 54, 26 July 2018 (2018-07-26), pages 123 - 129, XP085510271, ISSN: 0952-7915, DOI: 10.1016/J.COI.2018.06.002 *
DUO PENG ET AL: "EuPaGDT: a web tool tailored to design CRISPR guide RNAs for eukaryotic pathogens", MICROBIAL GENOMICS, vol. 1, no. 4, 13 October 2015 (2015-10-13), XP055681566, DOI: 10.1099/mgen.0.000033 *
FREIJE ET AL., MOLECULAR CELL, vol. 76, 2019, pages 826 - 837
GLASSLEELIXU, TRENDS IN BIOTECHNOLOGY, 2017
LEE ET AL., NATURE BIOMEDICAL ENGINEERING, vol. 1, 2017, pages 889 - 901
LI ET AL., BIOMATERIALS, vol. 178, 2018, pages 652 - 662
LIUZHANGLIUCHENG, JOURNAL OF CONTROLLED DISEASE, vol. 266, 2017, pages 17 - 26
MALI ET AL., SCIENCE, vol. 339, 2013, pages 823 - 826
MOUT ET AL., ACS NANO, vol. 11, 2017, pages 2452 - 2458
PARK ET AL., NATURE COMMUNICATIONS, vol. 9, 2018, pages 3313
SMARGONCOXPYZOCHA ET AL., MOLECULAR CELL, 2017
UEDA, MICROBIOLOGY AND IMMUNOLOGY, vol. 60, no. 7, July 2016 (2016-07-01), pages 483 - 496
WANG ET AL., PNAS, vol. 113, no. 11, 15 March 2016 (2016-03-15), pages 2868 - 2873
ZETSCHE ET AL., CELL, vol. 163, 2015, pages 759 - 771

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