Title:
CRISPR/CAS-RELATED METHODS AND COMPOSITIONS FOR KNOCKING OUT C5
Document Type and Number:
WIPO Patent Application WO/2023/077053
Kind Code:
A3
Abstract:
Guide RNAs and CRISPR/Cas systems targeting a C5 locus or gene, lipid nanoparticles or viral vectors comprising such guide RNAs or CRISPR/Cas systems, and cells or animals comprising such guide RNAs or systems are provided. Methods of modifying or knocking down or knocking out a C5 locus or gene using the CRISPR/Cas systems are also provided, as well as use of the CRISPR/Cas systems in prophylactic and therapeutic applications for treatment and/or prevention of a disease, disorder, or condition associated with C5 and/or for ameliorating at least one symptom associated with such disease, disorder, or condition.
Inventors:
DEVALARAJA-NARASHIMHA KISHOR (US)
MORTON LORI (US)
PEFANIS EVANGELOS (US)
HARTFORD SUZANNE (US)
KANJOLIA ARTI (US)
HESSE SARAH (US)
MORTON LORI (US)
PEFANIS EVANGELOS (US)
HARTFORD SUZANNE (US)
KANJOLIA ARTI (US)
HESSE SARAH (US)
Application Number:
PCT/US2022/078855
Publication Date:
June 15, 2023
Filing Date:
October 28, 2022
Export Citation:
Assignee:
REGENERON PHARMA (US)
International Classes:
A61K31/712; C12N15/113; A61P7/00
Domestic Patent References:
| WO2017201476A1 | 2017-11-23 | |||
| WO2015088643A1 | 2015-06-18 | |||
| WO2016040589A1 | 2016-03-17 | |||
| WO2021050940A1 | 2021-03-18 | |||
| WO2016081923A2 | 2016-05-26 |
Foreign References:
| KR20200121080A | 2020-10-23 | |||
| US20160237438A1 | 2016-08-18 |
Other References:
JOHN G DOENCH ET AL: "Optimized sgRNA design to maximize activity and minimize off-target effects of CRISPR-Cas9", NATURE BIOTECHNOLOGY, vol. 34, no. 2, 18 January 2016 (2016-01-18), New York, pages 184 - 191, XP055551151, ISSN: 1087-0156, DOI: 10.1038/nbt.3437
ORT MARION ET AL: "Treatment of Rare Inflammatory Kidney Diseases: Drugs Targeting the Terminal Complement Pathway", FRONTIERS IN IMMUNOLOGY, vol. 11, 10 December 2020 (2020-12-10), XP055858384, Retrieved from the Internet DOI: 10.3389/fimmu.2020.599417
GIORGIO CRISTINA ET AL: "Emerging Role of C5 Complement Pathway in Peripheral Neuropathies: Current Treatments and Future Perspectives", BIOMEDICINES, vol. 9, no. 4, 7 April 2021 (2021-04-07), pages 399, XP093018108, DOI: 10.3390/biomedicines9040399
SANSON KENDALL R. ET AL: "Optimized libraries for CRISPR-Cas9 genetic screens with multiple modalities", NATURE COMMUNICATIONS, vol. 9, no. 1, 21 December 2018 (2018-12-21), pages 5416, XP093034521, Retrieved from the Internet DOI: 10.1038/s41467-018-07901-8
LÓPEZ-LERA ALBERTO ET AL: "Molecular characterization of three new mutations causing C5 deficiency in two non-related families", MOLECULAR IMMUNOLOGY, vol. 46, no. 11-12, 2 May 2009 (2009-05-02), GB, pages 2340 - 2347, XP093034501, ISSN: 0161-5890, DOI: 10.1016/j.molimm.2009.03.026
LAPPEGÅRD KNUT TORE ET AL: "Human genetic deficiencies reveal the roles of complement in the inflammatory network: Lessons from nature", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES, vol. 106, no. 37, 15 September 2009 (2009-09-15), pages 15861 - 15866, XP093034462, ISSN: 0027-8424, Retrieved from the Internet DOI: 10.1073/pnas.0903613106
EL SISSY CARINE ET AL: "Clinical and Genetic Spectrum of a Large Cohort With Total and Sub-total Complement Deficiencies", FRONTIERS IN IMMUNOLOGY, vol. 10, 8 August 2019 (2019-08-08), XP093034466, DOI: 10.3389/fimmu.2019.01936
ORT MARION ET AL: "Treatment of Rare Inflammatory Kidney Diseases: Drugs Targeting the Terminal Complement Pathway", FRONTIERS IN IMMUNOLOGY, vol. 11, 10 December 2020 (2020-12-10), XP055858384, Retrieved from the Internet
GIORGIO CRISTINA ET AL: "Emerging Role of C5 Complement Pathway in Peripheral Neuropathies: Current Treatments and Future Perspectives", BIOMEDICINES, vol. 9, no. 4, 7 April 2021 (2021-04-07), pages 399, XP093018108, DOI: 10.3390/biomedicines9040399
SANSON KENDALL R. ET AL: "Optimized libraries for CRISPR-Cas9 genetic screens with multiple modalities", NATURE COMMUNICATIONS, vol. 9, no. 1, 21 December 2018 (2018-12-21), pages 5416, XP093034521, Retrieved from the Internet
LÓPEZ-LERA ALBERTO ET AL: "Molecular characterization of three new mutations causing C5 deficiency in two non-related families", MOLECULAR IMMUNOLOGY, vol. 46, no. 11-12, 2 May 2009 (2009-05-02), GB, pages 2340 - 2347, XP093034501, ISSN: 0161-5890, DOI: 10.1016/j.molimm.2009.03.026
LAPPEGÅRD KNUT TORE ET AL: "Human genetic deficiencies reveal the roles of complement in the inflammatory network: Lessons from nature", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES, vol. 106, no. 37, 15 September 2009 (2009-09-15), pages 15861 - 15866, XP093034462, ISSN: 0027-8424, Retrieved from the Internet
EL SISSY CARINE ET AL: "Clinical and Genetic Spectrum of a Large Cohort With Total and Sub-total Complement Deficiencies", FRONTIERS IN IMMUNOLOGY, vol. 10, 8 August 2019 (2019-08-08), XP093034466, DOI: 10.3389/fimmu.2019.01936
Attorney, Agent or Firm:
SCHILLING, Stephen, H. et al. (US)
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