Biotech firm KSQ Therapeutics has received clearance from the U.S. Food and Drug Administration (FDA) to proceed with a phase I and II trial of KSQ-001EX, KSQ’s CRISPR-Cas9 engineered tumor-infiltrating lymphocyte (TIL) program.
The Lexington, MA-based company will enroll patients with melanoma, head and neck squamous cell carcinoma (HNSCC), and non-small cell lung cancer (NSCLC) at the University of Texas MD Anderson Cancer Center in Houston and the Cell Therapy Manufacturing Center, a joint venture of the MD Anderson Cancer Center and National Resilience, which collaborated with KSQ to develop KSQ-001EX.
The phase I/II clinical trial will be an open-label, safety lead-in study for patients with melanoma, HNSCC, and NSCLC. The primary objective of the phase I trial will be to evaluate safety and tolerability of KSQ-001EX, while the primary objective of the second phase study will be to evaluate antitumor activity in indication-specific cohorts.
In preclinical studies, KSQ-001EX demonstrated enhanced antitumor function in solid tumor models refractory to PD-1 inhibition, as well as enhanced persistence and memory formation. KSQ-001EX consists of TILs in which the SOCS1 gene has been inactivated by CRISPR-Cas9 gene editing.
KSQ’s CRISPRomics platform identified SOCS1 as a key gene regulating the antitumor potency and persistence of TILs. The treatment is derived from patient tumor samples, including core biopsies.