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KSQ Therapeutics Announces Discovery of Gene Targets with Potential Activity Superior to PD-1 for Development of Engineered Tumor Infiltrating Lymphocyte (eTIL™) Therapies for Solid Tumors

  • Data demonstrate potential to increase efficacy of TIL adoptive cell therapy in preclinical models

CAMBRIDGE, Mass., February 9, 2020 – KSQ Therapeutics, a biotechnology company using its proprietary CRISPRomics® discovery platform to systematically screen the whole genome to identify optimal gene targets for oncology and autoimmune disease, today announced the identification and validation of a novel target, CT-1, for the development of engineered tumor infiltrating lymphocyte (eTIL™) therapies for refractory solid tumors. Data from two large-scale CRISPR-Cas9 functional screens using the company’s novel CRISPRomics® platform and in vivo validation data will be presented today at the Engineering the Genome conference, which takes place in Banff, Alberta, Canada.

“Adoptive cell therapies have long been hypothesized to be potentially curative treatments for refractory solid tumors, but their efficacy has been limited by the immunosuppressive tumor microenvironment,” said Frank Stegmeier, Chief Scientific Officer of KSQ Therapeutics. “Our CRISPRomics® platform enables us to identify gene targets that improve the ability of the T cell to function in this hostile tumor microenvironment. These insights allowed us to develop a pipeline of CRISPR/Cas9 eTIL™ programs that have the potential to unlock adoptive cell therapy in PD-1 refractory solid tumors.”

KSQ’s proprietary CRISPRomics® platform was used to identify the top targets across the T- cell genome that increase the efficacy of adoptive T cell transfer therapy (ACT) in PD-1 refractory mouse solid tumor models. CT-1, an undisclosed target identified by KSQ, emerged as a top target from these screens.  CT-1-edited T cells produced a 10-fold increase in anti-tumor activity in vivo, and CT-1 edited human TILs exhibit an enhanced cytokine production profile. The data presented support the development of eTIL™ products with the potential to increase the efficacy of TIL adoptive cell therapy.

Click to download PDF file of poster.

About KSQ Therapeutics

KSQ Therapeutics is advancing a pipeline of tumor- and immune-focused drug candidates for the treatment of cancer, across multiple drug modalities including targeted therapies, adoptive cell therapies and immuno-therapies. KSQ’s proprietary CRISPRomics® discovery engine enables genome-scale, in vivo validated, unbiased drug discovery across broad therapeutic areas. KSQ was founded by thought leaders in the field of functional genomics and pioneers of CRISPR screening technologies, and the company is located in Cambridge, Massachusetts. For more information, please visit the company’s website at

KSQ Contact:
Michael Lampe
Tel: 484-575-5040

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