FDA Clears IND for Innovative TIL Therapy

KSQ Therapeutics, a leading US-based biotechnology company leveraging CRISPR for cancer immunotherapy, announced that the US FDA has cleared its Investigational New Drug (IND) application for KSQ-004EX—a novel, CRISPR-Cas9 engineered tumor-infiltrating lymphocyte (TIL) therapy. The approval enables KSQ, in partnership with The University of Texas MD Anderson Cancer Center, to initiate a Phase 1/2 clinical trial targeting advanced solid tumors, including melanoma, non-small-cell lung cancer, colorectal cancer, and cervical cancer.

Next-Generation CRISPR-Engineered TILs

KSQ-004EX represents a new wave of personalized cell therapy. The product uses CRISPR to knock out two key suppressor genes, SOCS1 and Regnase-1, within patient-derived TILs. Preclinical data suggest that inactivating these genes boosts anti-tumor potency and helps TILs persist longer within the tumor microenvironment—a critical advantage in cancers that have resisted standard immunotherapies such as PD-1 inhibitors.

“Advancing KSQ-004EX into human trials is a landmark for our CRISPR platform. We believe this approach could deliver best-in-class efficacy for hard-to-treat solid tumors,” said Frank Stone, Chief Medical Officer at KSQ Therapeutics.

Clinical Trial Details and Goals

The multi-center, open-label study is sponsored by MD Anderson Cancer Center. It will evaluate the safety, efficacy, and persistence of CRISPR-edited TIL therapy in patients with several advanced solid cancers for whom alternative treatments have failed. The primary endpoints include safety and objective tumor response; secondary measures will track persistence of modified T cells and overall survival outcomes.

KSQ-004EX is KSQ’s second gene-edited immune cell therapy to enter human studies. Its first candidate, KSQ-001EX (SOCS1 knockout only), is currently in early-stage trials for melanoma and other advanced cancers.

Broader Impact: CRISPR Gene Editing in Oncology

The FDA’s green light marks continued momentum for CRISPR-based therapies in oncology. As cancer researchers harness genome engineering to make TILs more resilient and aggressive against tumors, investors and scientists alike are watching the field for signals of durable, curative responses. This news closely follows other major milestones for gene editing, including recent approvals for blood disorders and rapid growth in early-stage pipeline candidates across the sector.

What’s Next?

KSQ and its academic collaborators plan to begin enrolling patients in late 2024, with initial results expected in 2025. “Precision immunotherapy powered by genome editing could redefine the treatment paradigm for solid tumors,” said principal investigator Dr. Katherine Chen at MD Anderson.