KSQ Therapeutics has dosed the first patient in a Phase 1/2 clinical trial for KSQ-004EX, a CRISPR-engineered tumor-infiltrating lymphocyte (eTIL) therapy targeting advanced solid tumors. KSQ-004EX utilizes CRISPR/Cas9 gene editing to inactivate the SOCS1 and Regnase-1 genes, identified by KSQ’s CRISPRomics platform as inhibitors of TIL’s tumor-eradication abilities. The trial, conducted at The University of Texas MD Anderson Cancer Center, will assess the safety and efficacy of KSQ-004EX in patients with various solid tumors including melanoma, NSCLC, HNSCC, colorectal, pancreatic, and cervical cancers.
This development marks a significant advancement in the field of TIL therapy. Current TIL therapies face limitations in effectively treating solid tumors. The preclinical data suggesting enhanced anti-tumor functionality with the dual inactivation of SOCS1 and Regnase-1 offers a potential solution to these challenges, opening the door for a more potent and effective treatment option for patients with a range of difficult-to-treat cancers. This could lead to improved patient outcomes and potentially expand the application of TIL therapy to a wider patient population.
The Phase 1 portion of the trial focuses on safety and tolerability, while Phase 2 will evaluate anti-tumor activity in specific cancer types. KSQ-004EX, developed using KSQ’s proprietary CRISPRomics and CRISPR² screening approach, has demonstrated improved engraftment, expansion, tumor infiltration, and resistance to exhaustion in preclinical models. This approach of precisely targeting genes that limit TIL efficacy offers a new dimension in personalized cancer treatment.
The initiation of this clinical trial signifies a crucial step towards validating the potential of KSQ-004EX as a next-generation TIL therapy. Positive clinical results could pave the way for a new standard of care in solid tumor treatment, offering hope for improved outcomes for patients who currently have limited therapeutic options. Further, this trial could validate KSQ’s CRISPRomics platform, strengthening its potential for future drug development.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
