KSQ Therapeutics Announces FDA Clearance of IND Application for KSQ-001EX, a CRISPR/Cas9 Engineered Tumor Infiltrating Lymphocyte (eTIL®) Therapy

Phase 1/2 clinical study will evaluate the safety, pharmacokinetics, and preliminary efficacy of KSQ-001EX in melanoma, head and neck squamous cell carcinoma, and non-small cell lung cancer 

Lexington, Mass., November 29, 2023 — KSQ Therapeutics, Inc. (KSQ), a clinical-stage biotechnology company developing novel treatments for solid tumors, in collaboration with The University of Texas MD Anderson Cancer Center and the Cell Therapy Manufacturing Center (CTMC), today announced the Food and Drug Administration (FDA) has cleared an Investigational New Drug (IND) application for a Phase 1/2 clinical study of KSQ-001EX, KSQ’s lead engineered tumor-infiltrating lymphocyte (eTIL®) program. KSQ-001EX consists of TILs in which the SOCS1 gene is inactivated by CRISPR/Cas9 gene editing. KSQ’s CRISPRomics® platform identified SOCS1 as a key gene regulating the anti-tumor potency and persistence of TIL and provides KSQ-001EX with best-in-class potential in the treatment of solid tumors.

“Our team has made great progress advancing our KSQ-001EX and KSQ-004EX eTIL® programs. Through our platform insights, we’ve identified how to enhance the effectiveness of T cells in the treatment of solid tumors. We believe our eTIL® cell therapies have tremendous potential to benefit patients with cancer who have limited treatment options. We look forward to working closely with the clinical experts at MD Anderson, where the study will be initiated,” said Qasim Rizvi, Chief Executive Officer of KSQ. “Our partnership with CTMC has yielded a robust manufacturing process for our eTIL® cell therapy that is derived from patient tumor samples, including core biopsies. The collaborative effort allowed the IND to be submitted in an accelerated timeframe.”

“New treatment options are urgently needed in melanoma and other solid tumors, and the potential to improve outcomes and patient experience is very exciting. KSQ-001EX exhibited considerable efficacy in preclinical studies,” said Rodabe Amaria, M.D., professor of Melanoma Medical Oncology at MD Anderson and principal investigator of the KSQ-001EX study. “We look forward to working closely with KSQ as we advance this program.”

Phase 1/2 Trial Design

The Phase 1/2 clinical trial is an open-label, safety lead-in study for patients with melanoma, head and neck squamous cell carcinoma (HNSCC), and non-small cell lung cancer (NSCLC). The primary objective of the Phase 1 portion of the trial is to evaluate safety and tolerability of KSQ-001EX. In the safety lead-in portion, a cohort of patients will be initially dosed without IL-2. The primary objective of Phase 2 is to evaluate antitumor activity in indication-specific cohorts.

About KSQ-001EX

KSQ’s lead eTIL® cell therapy program, KSQ-001EX, in which TILs are edited to inactivate the SOCS1 gene, has the potential to revolutionize the treatment of solid tumors. In preclinical studies, KSQ-001EX demonstrated enhanced anti-tumor function in solid tumor models refractory to PD-1 inhibition, as well as enhanced persistence, and memory formation.

About KSQ Therapeutics

KSQ Therapeutics is advancing a pipeline of novel 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 insights driving novel therapeutic discovery. For more information, please visit the company’s website at

KSQ Media Contact:
Cory Tromblee