The CRISPRomics® Company

Engineering Cells for LifeTM

We systematically discover and pursue the best therapeutic targets for disease intervention, aspiring to the day when cures become the standard of care for patients.

Changing the Probabilities

Genome-scale understanding of biology is enabling breakthrough medicines. We are a clinical-stage company using our CRISPRomics® discovery platform to systematically decode the genome to identify optimal gene targets for oncology and autoimmune disease. And we’re using our drug development capabilities, including our first-in-class USP1 inhibitor KSQ-4279 and our eTIL™ autologous cell therapy platform, to rewire human biology with the potential to cure diseases.

This genome-wide depth and cell-type breadth unlocks an unparalleled pipeline opportunity with significant curative potential for a wide range of human diseases.

Target & Drug Discovery Supercharged by CRISPR

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CRISPRomics for Targeted Cancer Therapies

Identify and validate the most selective and potent targets for new cancer therapies
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CRISPRomics for Immuno-Oncology Therapies

Identify and validate in vivo the best tumor-killing targets in T cells for new cancer monotherapies

Product Development Pipeline

 

Expanded Access Policy
Read our Expanded Access Policy

KSQ-4279 – A pipeline in a single molecule

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-4279 – A pipeline in a molecule

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

See the ENA 2020 Poster

For clinical trial information please visit clinicaltrials.gov.

eTILs™ - Revolutionizing the treatment of solid tumors

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 have developed KSQ-001 and KSQ-004, our lead eTIL programs, by applying our CRISPRomics approach 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. eTILs also offer the possibility of lower dosing and reduced patient conditioning when compared to other cell therapies, potentially improving the patient experience. Our simplified ExPRESSTM eTIL manufacturing process promises simpler and more rapid tumor-to-vein turnaround times.

See the AACR 2020 Poster

2021 SITC Poster | 2022 SITC Poster

Discovery Programs

Cancer

Targeted Oncology
Tumor Programs
Solid tumors

Immuno-Oncology
T cell Programs
Solid tumors

Cell Therapy
Dual-edit eTIL™ programs
PD-1 refractory solid tumors

Autoimmunity

Immuno-Regulation
Treg Programs
Autoimmune diseases

Cancer

Targeted Oncology
DNA-Damage Response (DDR) Targets

Cell Therapy
Next-Generation eTIL™ programs & manufacturing processes

Autoimmunity

Immuno-Regulation
Treg Targets
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