KSQ Therapeutics Announces First Patient Dosed in Phase 1/2 Study for KSQ-004EX, a CRISPR/Cas9-Engineered Tumor Infiltrating Lymphocyte (eTIL®) Therapy

Phase 1/2 clinical study will evaluate the safety and preliminary efficacy of KSQ-004EX in advanced solid tumors

/EIN News/ -- LEXINGTON, Mass., April 09, 2025 (GLOBE NEWSWIRE) -- KSQ Therapeutics, Inc. (KSQ), a clinical-stage biotechnology company developing treatments for solid tumors, today announced the first patient dosed in the Phase 1/2 clinical study of KSQ-004EX, a novel CRISPR-engineered Tumor Infiltrating Lymphocyte (eTIL^®) therapy. KSQ-004EX consists of eTIL in which the SOCS1 and Regnase-1 genes are inactivated by CRISPR/Cas9 gene editing, giving it the potential to be a best-in-class treatment for a variety of solid tumor indications. KSQ’s CRISPRomics^® platform identified SOCS1 and Regnase-1 as key genes inhibiting the ability of TIL to eradicate solid tumors in preclinical models.

“The promise of TIL therapy remains high, but current approaches fall short of addressing the needs of most patients with solid tumors. Preclinically, the combined inactivation of SOCS1 and Regnase-1 in KSQ-004EX strongly increased anti-tumor functionality. We believe these enhancements give KSQ-004EX the potential to significantly advance TIL therapy for the treatment of solid tumors,” said Qasim Rizvi, Chief Executive Officer of KSQ and CEO-Partner at Flagship Pioneering.

The first patient treated with KSQ-004EX was enrolled by Rodabe Amaria, M.D., professor of Melanoma Medical Oncology and principal investigator of the study at The University of Texas MD Anderson Cancer Center.

Phase 1/2 Trial Design
The Phase 1/2 clinical trial is an open-label, dose escalation study for patients with advanced solid tumors (including melanoma, non-small cell lung cancer (NSCLC), head and neck squamous cell carcinoma (HNSCC), colorectal cancer (CRC), pancreatic cancer, and cervical cancer. The primary objective of the Phase 1 portion of the trial is to evaluate the safety and tolerability of KSQ-004EX. The primary objective of Phase 2 is to evaluate antitumor activity in indication-specific cohorts.

About KSQ-004EX
KSQ-004EX is an autologous cell therapy generated from TIL engineered with CRISPR/Cas9 to inactivate SOCS1 and Regnase-1 to maximize anti-tumor activity. SOCS1 and Regnase-1 were discovered in preclinical studies using KSQ’s proprietary CRISPRomics platform and CRISPR^2® screening approach, which identified the top dual-edit combinations enhancing the activity of adoptive T cell therapies against solid tumors. Upon transfer in preclinical solid tumor models, KSQ-004EX demonstrated improved engraftment, expansion, tumor infiltration and killing while being refractory to exhaustion. Long-term persistence and memory formation were additionally established.   KSQ-004EX has a broad potential to address the unmet need of patients with late-stage, metastatic solid tumors.

About KSQ Therapeutics
KSQ Therapeutics is advancing a pipeline of tumor- and immune-focused drug candidates to treat cancer across multiple drug modalities, including targeted therapies, adoptive cell therapies, and immunotherapies. KSQ’s proprietary CRISPRomics^® discovery engine enables genome-scale, in vivo-validated, unbiased target discovery across broad therapeutic areas. For more information, please visit www.ksqtx.com and follow us on LinkedIn and X.

Media Contact:
Cory Tromblee
cory@scientpr.com