Pharma Focus America

KSQ Therapeutics Launches Clinical Trial for KSQ-001EX, a CRISPR/Cas9 Modified Tumor Infiltrating Lymphocyte Therapy

Thursday, June 13, 2024

KSQ Therapeutics, Inc., a clinical-stage biotechnology firm dedicated to developing treatments for solid tumors, has commenced the first patient in its Phase 1/2 clinical trial for KSQ-001EX, an innovative edited TIL therapy. Utilizing CRISPR/Cas9, KSQ-001EX targets the SOCS1 gene for inactivation in TIL, offering the potential to become a leading treatment for various solid tumor conditions. The CRISPRomics® platform at KSQ pinpointed SOCS1 as a vital gene that hampers T cell growth, survival, and differentiation by negatively affecting cytokine signaling within the tumor microenvironment.

"TIL therapy is proving to be a powerful treatment option, and we are optimistic about our eTIL® programs, including KSQ-001EX and KSQ-004EX, to potentially transform the field by enhancing TIL therapy's effectiveness in treating a wide array of solid tumors. In preclinical trials, KSQ-001EX demonstrated superior anti-tumor activity, polyclonality, persistence, and memory formation in various solid tumor models compared to unmodified TIL. It also exhibited significant anti-tumor responses in models that are resistant to PD-1 inhibition," said Qasim Rizvi, Chief Executive Officer of KSQ. "We look forward to evaluating KSQ-001EX in patients with particularly difficult-to-treat solid tumors, such as melanoma, head and neck squamous cell carcinoma, and non-small cell lung cancer."

Rodabe Amaria, M.D., professor of Melanoma Medical Oncology and the study's principal investigator at The University of Texas MD Anderson Cancer Center, enrolled the first patient in the trial.

The Phase 1/2 clinical trial is an open-label, safety lead-in study aimed at patients with melanoma, head and neck squamous cell carcinoma (HNSCC), and non-small cell lung cancer (NSCLC). The primary aim of the Phase 1 segment is to evaluate the safety and tolerability of KSQ-001EX. During the initial safety lead-in phase, a cohort of patients will be treated without IL-2. The main objective of Phase 2 is to assess the anti-tumor effectiveness within specific patient groups.

KSQ's primary eTIL® cell therapy program, KSQ-001EX, aims to revolutionize solid tumor treatment through the inactivation of the SOCS1 gene in TIL. Preclinical studies have indicated that KSQ-001EX significantly enhances anti-tumor activity in models of solid tumors that are resistant to PD-1 inhibition and improves persistence and memory formation in these treatments.



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