Ksq Therapeutics, Inc.

Michael R Schlabach

Jason J Merkin

Gregory V Kryukov

Micah Benson

Noah Jacob Tubo

Solomon Martin Shenker

James Martin Kaberna, Ii

Andrew Alistair Wylie

Jehrod Burnett Brenneman

Elsa Beyer Krall

Anja Fides Hohmann

The present disclosure provides methods and compositions related to the modification of immune effector cells to increase therapeutic efficacy. In some embodiments, immune effector cells modified to reduce expression of one or more endogenous target genes, or to reduce one or more functions of an endogenous protein to enhance effector functions of the immune cells are provided. In some embodiments, immune effector cells further modified by introduction of transgenes conferring antigen specificity, such as exogenous T cell receptors (TCRs) or chimeric antigen receptors (CARs) are provided. Methods of treating a cell proliferative disorder, such as a cancer, using the modified immune effector cells described herein are also provided.

Gene-regulating compositions and methods for improved immunotherapy

The present disclosure provides gRNA compositions, CRISPR/Cas systems comprising the same, and methods of their use in the modification of immune effector cells. Methods of treating a cell proliferative disorder, such as a cancer, using the modified immune effector cells described herein are also provided.

Guide RNA combinations and methods of use

The invention describes a novel system for identifying optimized gRNAs for use in CRISPR/Cas9 genome editing platforms. The invention allows for the determination of specific gene alterations rendered by a particular gRNA, thereby permitting the generation of optimized gRNA libraries.

Methods for measuring and improving CRISPR reagent function

The present disclosure provides compounds having Formula I: and the pharmaceutically acceptable salts and solvates thereof, wherein X, X, X, X, R, R, R, R, R, and Rare defined as set forth in the specification. The present disclosure is also directed to the use of compounds of Formula I to inhibit a USP1 protein and/or to treat a disorder responsive to the inhibition of USP1 proteins and USP1 activity. Compounds of the present disclosure are especially useful for treating cancer.

Substituted pyrazolopyrimidines and substituted purines and their use as ubiquitin-specific-processing protease 1 (USP1) inhibitors

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Lexington, MA, United States of America

Michael R Schlabach