De-Coding and Re-Wiring Human Biology at Genome Scale

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 entire genome to identify optimal gene targets for oncology and autoimmune disease. And we’re using our drug development capabilities, particularly our eTIL™ autologous cell therapy platform, to rewire human biology with the potential to cure diseases.

This depth and breadth unlocks an unparalleled pipeline opportunity, with significant curative potential for a wide range of cancers, in particular solid tumors, as well as autoimmune diseases.

 

CRISPRomics®

CRISPRomics® is our industrialized discovery engine that utilizes a suite of proprietary CRISPR/Cas9 tools to generate disease-specific insights for every human gene with great precision and at unprecedented scale. We have evolved this engine into multiple disease and cell-type specific platforms to identify and genetically validate optimal novel targets for drug discovery. We’re able to engineer and perform unbiased genome-scale screens in cancer cell lines and multiple immune cell types, including T cells, NK cells, Tregs, and are currently working to enable our platform into additional immune cell types. CRISPRomics has broad utility across multiple therapeutic areas, and the company is currently deploying this approach in the areas of oncology, immuno-oncology, and autoimmune disease.

Key Enabling Discoveries Timeline

Target & Drug Discovery Supercharged by CRISPR

We are applying our proprietary genome-scale CRISPRomics® engine to uncover new drug targets and rapidly rule out thousands of less relevant targets. Learn more about two of the ways we have deployed this approach to generate optimal drug targets.

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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

Our CRISPRomics® approach provides deeper validation up-front than traditional approaches and has rapidly generated a pipeline of discovery programs and product candidates to treat cancers and autoimmune diseases, using the company’s eTIL™ autologous cell therapy technology, immuno-oncology approaches, and novel small molecule target oncology programs.

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.

See the ENA 2020 Poster

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 ExPRESS baseline process is simpler and faster, enabling a more rapid tumor-to-vein turnaround time.

See the AACR 2020 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

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