COMPOSITIONS, SYSTEMS, AND METHODS FOR GENERATING GENE-EDITED CELLS

Title:

COMPOSITIONS, SYSTEMS, AND METHODS FOR GENERATING GENE-EDITED CELLS

Document Type and Number:

WIPO Patent Application WO/2021/232014

Kind Code:

A3

Abstract:

The present invention relates to compositions, systems, and methods for editing a disease/condition causing mutation region in a target gene in a cell. In certain embodiments, the following components are employed: i) mRNA encoding a Tumor Protein p53 (TP53) inhibitor, ii) an inhibiting agent that inhibits Tumor Suppressor p53-Binding Protein 1 (53BPI) (e.g., small molecule EoHR or mRNA encoding a protein that inhibits 53BPI), iii) mRNA encoding a Cas nuclease for CRISPR; iv) a guide RNA specific for a target cleavage site proximal to said disease/condition-causing mutation region; and v) a repair template comprising a region of interest configured to replace said disease/condition-causing mutation region in the target gene during homology-directed repair (HDR). In certain embodiments, the cell is a T-cell, stem cell (e.g., hematopoietic stem cell), or progenitor cell from a subject with the disease or condition (e.g., a Primary Immunodeficiency Disease (PID)). In some embodiments, the gene-edited cell is administered to the subject.

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Inventors:

MEIS RONALD (US)
DAHL GARY (US)
DE RAVIN SUK SEE (US)
BRAULT JULIE (US)
SWEENEY COLIN L (US)
MALECH HARRY L (US)

Application Number:

PCT/US2021/032782

Publication Date:

December 23, 2021

Filing Date:

May 17, 2021

Export Citation:

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Assignee:

CELLSCRIPT LLC (US)
US HEALTH (US)

International Classes:

C12N15/10; C07K14/47; C12N9/22; C12N15/113; C12N15/90

Foreign References:

US20190010196A1	2019-01-10
US20180245065A1	2018-08-30
US20090197335A1	2009-08-06

Other References:

YANG DIANE, SCAVUZZO MARISSA A, CHMIELOWIEC JOLANTA, SHARP ROBERT, BAJIC ALEKSANDAR, BOROWIAK MALGORZATA: "Enrichment of G2/M cell cycle phase in human pluripotent stem cells enhances HDR-mediated gene repair with customizable endonuclease s", SCIENTIFIC REPORTS, vol. 6, no. 21264, 18 February 2016 (2016-02-18), pages 1 - 15, XP055379621

Attorney, Agent or Firm:

BOND, Jason R. (US)

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