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KSQ Therapeutics Gets FDA Clearance to Begin Phase I/II Trial of CRISPR-Cas9-Edited TIL Therapy

NEW YORK – KSQ Therapeutics on Wednesday said it was cleared by the US Food and Drug Administration to begin a Phase I/II trial of its autologous tumor-infiltrating lymphocyte (TIL) candidate, KSQ-001EX, in solid tumors.

The company will enroll patients with melanoma, head and neck cancer, and non-small cell lung cancer at the University of Texas MD Anderson Cancer Center and the Cell Therapy Manufacturing Center, which codeveloped the treatment with KSQ.

KSQ-001EX consists of TILs, in which the SOCS1 gene is inactivated by CRISPR-Cas9 gene editing. The firm's CRISPRomics platform identified SOCS1 as a key gene that regulates the anti-tumor potency and persistence of TILs. The treatment is engineered from patient tumor samples, including core biopsies.

KSQ, based in Lexington, Massachusetts, has one other engineered TIL candidate in preclinical development, along with two undisclosed TIL candidates. The firm is also studying a USP1 inhibitor, KSQ-4279, in Phase I/II trials. Last year, the firm began exploring combination treatment strategies with KSQ-4279 in patients with advanced solid tumors.