This genome-wide depth and cell-type breadth unlocks an unparalleled pipeline opportunity with significant curative potential for a wide range of human diseases.
KSQ-4279 is a first-in-class small molecule inhibitor of USP1 and is currently in a Phase 1 clinical trial in patients with advanced solid tumors, both as a monotherapy and in combination. Because of its potential in patients with homologous repair deficiencies (HRD), across a range of cancers, we believe KSQ-4279 has the potential to become a pipeline of treatments in a single molecule. KSQ utilized its proprietary CRISPRomics® platform to identify the deubiquitinating enzyme USP1 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 utilized its proprietary CRISPRomics® platform to identify the deubiquitinating enzyme USP1 as an attractive cancer target with roles in DNA damage repair processes through mechanisms distinct from both PARP inhibitors and other targeted therapies currently being tested in the clinic. KSQ-4279 is a first-in-class small molecule inhibitor of USP1 currently in a Phase 1 clinical trial in patients with advanced solid tumors. Because of its potential in patients with homologous repair deficiencies (HRD) across a range of cancers, we believe KSQ-4279 has the potential to become a product pipeline in itself.
See the ENA 2020 Poster
For clinical trial information please visit clinicaltrials.gov.
Our eTIL™ autologous cell therapy candidates, KSQ-001 and KSQ-004, have demonstrated the potential for transformational efficacy in preclinical models. Their potential resides in the fact that they engage highly validated genomic targets (discovered from genome-scale screens) and are engineered for optimal potency, memory formation and durability. They also offer the possibility of lower dosing and reduced lymphodepletion when compared to other cell therapies. Our simplified ExPRESSTM baseline process is simpler and faster, enabling a more rapid tumor-to-vein turnaround time.
eTIL are gene-edited Tumor Infiltrating Lymphocyte (TIL) cell therapy products that have demonstrated transformational tumor-killing abilities in preclinical tumor models. We developed KSQ-001, our lead single-edit eTIL program, by applying our CRISPRomics platform to T cells to discover the top genes to inactivate with CRISPR/Cas9 with the goal of maximizing the ability of tumor-targeting T cells to destroy solid tumors. We combined our CRISPRomics platform with our CRISPR2 technology, which employs randomly paired CRISPR guide libraries, to discover KSQ-004, our lead dual-edit eTIL program. eTILs offer the possibility of lower dosing and reduced patient conditioning when compared to other cell therapies, potentially improving the patient experience. Our simplified ExPRESS™ eTIL manufacturing process promises simpler and more rapid tumor-to-vein turnaround times.
Targeted Oncology
Tumor Programs
Solid tumors
Immuno-Oncology
T cell Programs
Solid tumors
Cell Therapy
Dual-edit eTIL™ programs
PD-1 refractory solid tumors
Immuno-Regulation
Treg Programs
Autoimmune diseases
Targeted Oncology
DNA-Damage Response (DDR) Targets
Cell Therapy
Next-Generation eTIL™ programs & manufacturing processes
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