LEXINGTON, MA, USA I July 13, 2023 I KSQ Therapeutics, Inc. (“KSQ”), a clinical-stage biotechnology company developing cancer therapies using its proprietary CRISPRomics® discovery platform, announced today it has entered into a worldwide license and collaboration agreement with Roche for the development and commercialization of KSQ-4279. KSQ-4279 is a first-in-class, potent, and selective small molecule inhibitor of USP1, a protein that regulates DNA damage response (DDR) in a manner distinct from other approaches, including PARP inhibitors. The molecule is currently in a Phase 1 clinical trial for the treatment of solid tumors. Under the collaboration, Roche will assume development responsibilities for KSQ-4279, which has the potential to treat a variety of cancers.

“We believe KSQ-4279, which was discovered through our proprietary CRISPRomics® platform, has incredible potential to help patients with a variety of solid tumors based on the strong preclinical data we have seen to date. We are confident Roche is the right partner to further the development of KSQ-4279 and maximize its benefits for patients,” said Qasim Rizvi, Chief Executive Officer of KSQ. “For KSQ, this agreement enables us to fully focus our attention on advancing our own immunotherapy programs and to continue leveraging our successful platform to discover novel targets.”

“DDR is a promising and emerging area of research in oncology,” said James Sabry, M.D., Ph.D., Global Head of Pharma Partnering, Roche. “We are excited to collaborate with KSQ on their novel inhibitor of USP1 as a potential new treatment option for patients with significant unmet medical needs across a range of cancers. This partnership reflects our strategic focus of fitting treatments to patients and delivering personalized healthcare.”

Under the terms of the agreement, KSQ will receive an upfront payment and will be eligible to receive additional milestone and royalty payments. KSQ grants Roche a global license wherein Roche will be fully responsible for the further development of KSQ-4279 in 2024.


KSQ-4279 is a first-in-class small molecule inhibitor of USP1, a protein regulating DNA damage response (DDR). USP1 was identified by KSQ’s CRISPRomics® platform as an attractive cancer target with established roles in DNA damage repair processes that are distinct from PARP inhibitors and other approaches currently being tested in the clinic. KSQ-4279 is currently in a Phase 1 clinical trial (KSQ-4279-1101) in patients with advanced solid tumors. It is a dose-escalation and expansion trial of KSQ-4279 as a monotherapy and in combination.

About KSQ Therapeutics

KSQ Therapeutics is advancing a pipeline of tumor- and immune-focused drug candidates to treat cancer. KSQ’s proprietary CRISPRomics® discovery engine enables genome-scale, in vivo validated, unbiased drug discovery across broad therapeutic areas. For more information, please visit the company’s website at www.ksqtx.com and follow @ksq_tx on Twitter.

SOURCE: KSQ Therapeutics