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KSQ Therapeutics to Present Data at the American Association for Cancer Research (AACR) 2022 Annual Meeting

– Data supports ongoing Phase 1 study of KSQ-4279, a first-in-class USP1 inhibitor, in patients with advanced solid tumors

CAMBRIDGE, Mass., March 22, 2022- KSQ Therapeutics (KSQ), a clinical stage biotechnology company developing therapies to treat cancer and autoimmune diseases using its proprietary, integrated discovery CRISPRomics® platform, will give an oral presentation as part of the “New Drugs on the Horizon: Part 1” session at the upcoming American Association for Cancer Research (AACR) 2022 Annual Meeting in New Orleans. The company will provide an overview of preclinical data on its first-in-class USP1 inhibitor, KSQ-4279, which supports the ongoing Phase 1 clinical trial of KSQ-4279 being conducted in patients with advanced solid tumors.

Presentation Title: ND01 – KSQ-4279: A first-in-class USP1 inhibitor for the treatment of cancers with homologous recombination deficiencies

Session Title: DDT001 – New Drugs on the Horizon: Part 1

Session Date and Time: Sunday, April 10, 2022, 1:05-1:20 pm CT

Location: La Nouvelle Orleans A-B, Convention Center

Data Highlights:

  • First disclosure of KSQ-4279 structure, a potent and selective USP1 inhibitor
  • KSQ-4279 has a unique mode of allosteric binding to USP1 and a high degree of selectivity over other family members
  • Efficacy was observed both as a single agent and in combination with PARP inhibitors across multiple BRCA/HRD xenograft models
  • Functional genomic resistance screens indicate that the major genetic drivers of resistance to USP1 and PARP inhibitors are distinct, indicating that combination treatment may be able to delay or prevent the emergence of resistance
  • Favorable safety profile without overlapping toxicities suggests broad combination potential with PARP inhibitors

 “Based on the preclinical data we have observed from our studies of KSQ-4279, we believe it has great potential in a number of solid tumors, and we are looking forward to sharing the results from our ongoing Phase 1 clinical study in the future” said Frank Stegmeier, Ph.D., Chief Scientific Officer of KSQ. “USP1 is the first of multiple exciting targets that our CRISPRomics platform identified. In addition to KSQ-4279, we’re advancing multiple engineered TIL (eTILTM) adoptive cell therapy programs towards the clinic.”


KSQ-4279 is a first-in-class small molecule targeting USP1, a protein regulating DNA damage response (DDR). USP1 was identified by KSQ’s CRISPRomics® platform as a novel synthetic lethal target in cancers with certain types of genomic instability. KSQ-4279 is currently in a Phase 1 study in patients with advanced solid tumors.

About KSQ Therapeutics

KSQ Therapeutics is advancing a pipeline of tumor- and immune-focused drug candidates to treat cancer and autoimmune disease across multiple drug modalities, including targeted therapies, adoptive cell therapies, and immunotherapies. 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 and follow @ksq_tx on Twitter.

Media Contact:
Cory Tromblee

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