Engineering Cells for LifeTM

Changing the Probabilities

Our Vision

  • The Ambition to Cure

    Tackling the toughest challenges in medicine with the aspiration to cure human diseases

  • Disruptive Targets

    Leveraging our CRISPRomics® platform to discover the genome’s top therapeutic targets with curative potential

  • Transformational Medicines

    Advancing our pipeline of CRISPR/Cas9-engineered Tumor Infiltrating Lymphocytes (eTIL®) programs and our partnered first-in-class candidates for the treatment of cancer

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Our CRISPRomics® Platform

With curative ambition and a mission to use gene-editing to explore the entirety of the genome, we have industrialized genome-wide CRISPR/Cas9 functional genomic screens across a wide array of cancer models and immune cell types, seeking to identify the genome’s top therapeutic targets. We call this approach CRISPRomics.

To date, our CRISPRomics platform has enabled us to do what no other company had done before – perform genome-scale knockouts across a wide range of immune and cancer cells in disease-relevant models to discover the right targets and develop the right therapies for patients with solid tumors.

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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

eTIL® Therapies

By their very nature, Tumor-Infiltrating Lymphocytes (TILs) are an ideal therapy for the treatment of solid tumors, as they are a patient’s personalized immune response against cancer cells. We seek to maximize the tumor-killing activity of TIL therapies by knocking out targets identified using our CRISPRomics platform. To date, our investigational engineered Tumor Infiltrating Lymphocyte (eTIL) therapies have demonstrated the remarkable ability in preclinical models to boost T cell infiltration and to kill tumors even in settings where no other therapies work. We are currently advancing multiple eTIL candidates into the clinic.

In addition, we have developed ExPRESS™ (Ex Vivo, Potent, Economical, Streamlined, Simple), a proprietary, next-generation process to manufacture single and multiplex CRISPR/Cas9-edited eTIL therapies that we believe has distinct advantages over current manufacturing processes.

Advancing Transformational Medicines

Applying an unbiased approach, we were able to leverage our disease models to query >20,000 genes – the entirety of the genome – across more than 600 cancer lines and immune cell types to determine which therapeutic targets could be of most benefit to patients.

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Our eTIL® Programs

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.

Our eTIL® Programs

KSQ-001EX: Using our platform, we identified SOCS1 (Suppressor Of Cytokine Signaling 1) as a top target constraining TIL anti-tumor function, engraftment, and persistence. KSQ-001EX consists of an investigational eTIL therapy in which the SOCS1 gene is inactivated by CRISPR/Cas9 gene editing. We are currently conducting a Phase 1/2 clinical study (NCT06237881) of ExPRESS-manufactured KSQ-001EX in people with melanoma, head and neck squamous cell carcinoma (HNSCC), and non-small cell lung cancer (NSCLC).

KSQ-004EX: We also identified Regnase-1 as a top target of interest through our screening platform. Regnase-1 is an endonuclease that regulates immune responses by degrading the messenger RNA of pro-inflammatory proteins. KSQ-004EX, an investigational dual-edit eTIL therapy in which both the Regnase-1 and SOCS1 genes are inactivated by CRISPR/Cas9 gene editing, is also manufactured using our ExPRESS process. We are currently conducting IND-enabling studies to advance KSQ-004EX into clinical development.

Our Partnered Programs

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.

KSQ-4279 is a first-in-class, potent, and selective small molecule inhibitor of USP1, a target identified using our Targeted Oncology CRISPRomics platform due to its profile as selectively essential in certain biomarker-enabled cancer cell models and its role in DNA damage repair processes (DDR). KSQ-4279 has demonstrated strong activity in BRCA mutant preclinical cancer models, including strong combination activity with PARP inhibitors. In June 2023, we entered into a worldwide license and collaboration agreement with Roche for the development and commercialization of KSQ-4279; under the collaboration, Roche has assumed all clinical development responsibilities for KSQ-4279 (now RO7623066) and is currently conducting a Phase 1 clinical study (NCT05240898) in patients with a variety of advanced solid tumors.

We are also engaged in partnerships with Takeda, Ono Pharmaceutical, and others to explore the full potential of our platform and continue discovery of novel targets and candidates.

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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