Title:
OPTIMIZED mRNA ENCODING CAS9 FOR USE IN LNPs
Document Type and Number:
WIPO Patent Application WO/2020/112908
Kind Code:
A3
Abstract:
The present disclosure relates generally to novel lipid nanoparticle (LNP)-based compositions useful for, e.g., the delivery of a site-specific endonuclease or a nucleic acid molecule encoding same, into a target cell. Some embodiments of the disclosure relate to compositions and methods for editing the genome of a cell, which involve contacting the cell with an LNP composition as described herein.
Inventors:
CHENG CHRISTOPHER J (US)
SCHARENBERG ANDREW (US)
WANG KUI (US)
SANE SHAILENDRA (US)
SCHARENBERG ANDREW (US)
WANG KUI (US)
SANE SHAILENDRA (US)
Application Number:
PCT/US2019/063456
Publication Date:
August 20, 2020
Filing Date:
November 26, 2019
Export Citation:
Assignee:
CRISPR THERAPEUTICS AG (CH)
BAYER HEALTHCARE LLC (US)
BAYER HEALTHCARE LLC (US)
International Classes:
A61K9/127; C12N9/22; A61K31/7088; C12N15/11; C12N15/88
Domestic Patent References:
| WO2018058064A1 | 2018-03-29 | |||
| WO2017144630A1 | 2017-08-31 | |||
| WO2018107026A1 | 2018-06-14 | |||
| WO2015089419A2 | 2015-06-18 | |||
| WO2019183150A1 | 2019-09-26 |
Other References:
MILLER JEFFREY C ET AL: "An improved zinc-finger nuclease architecture for highly specific genome editing", NATURE BIOTECHNOLOGY, GALE GROUP INC, NEW YORK, vol. 25, no. 7, 1 July 2007 (2007-07-01), pages 778 - 785, XP002465119, ISSN: 1087-0156, DOI: 10.1038/NBT1319
JONATHAN D. FINN ET AL: "A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing", CELL REPORTS, vol. 22, no. 9, 27 February 2018 (2018-02-27), US, pages 2227 - 2235, XP055527484, ISSN: 2211-1247, DOI: 10.1016/j.celrep.2018.02.014
KHALID A. HAJJ ET AL: "Tools for translation: non-viral materials for therapeutic mRNA delivery", NATURE REVIEWS MATERIALS, vol. 2, no. 10, 12 September 2017 (2017-09-12), XP055657784, DOI: 10.1038/natrevmats.2017.56
F. ANN RAN ET AL: "In vivo genome editing using Staphylococcus aureus Cas9", NATURE, vol. 520, no. 7546, 9 April 2015 (2015-04-09), pages 186 - 191, XP055206733, ISSN: 0028-0836, DOI: 10.1038/nature14299
EUNJI KIM ET AL: "In vivo genome editing with a small Cas9 orthologue derived from Campylobacter jejuni", NATURE COMMUNICATIONS, vol. 8, 21 February 2017 (2017-02-21), pages 1 - 12, XP055435850, DOI: 10.1038/ncomms14500
JONATHAN D. FINN ET AL: "A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing", CELL REPORTS, vol. 22, no. 9, 27 February 2018 (2018-02-27), US, pages 2227 - 2235, XP055527484, ISSN: 2211-1247, DOI: 10.1016/j.celrep.2018.02.014
KHALID A. HAJJ ET AL: "Tools for translation: non-viral materials for therapeutic mRNA delivery", NATURE REVIEWS MATERIALS, vol. 2, no. 10, 12 September 2017 (2017-09-12), XP055657784, DOI: 10.1038/natrevmats.2017.56
F. ANN RAN ET AL: "In vivo genome editing using Staphylococcus aureus Cas9", NATURE, vol. 520, no. 7546, 9 April 2015 (2015-04-09), pages 186 - 191, XP055206733, ISSN: 0028-0836, DOI: 10.1038/nature14299
EUNJI KIM ET AL: "In vivo genome editing with a small Cas9 orthologue derived from Campylobacter jejuni", NATURE COMMUNICATIONS, vol. 8, 21 February 2017 (2017-02-21), pages 1 - 12, XP055435850, DOI: 10.1038/ncomms14500
Attorney, Agent or Firm:
MANDRAGOURAS, Amy, E. (US)
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