Turning disruptive
targets into

our unbiased approach

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.

And this was just the beginning.

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, including T cells, NK cells, and T regulatory cells, to determine which therapeutic targets could be of most benefit to patients. We then discovered and are advancing the most promising therapeutic candidates to fight disease.


Our screens have elucidated high-value targets, including:

  • SOCS1 stands for: Suppressor Of Cytokine Signaling 1. SOCS1 is a negative regulator of T cell signaling by cytokines, the small proteins critical in controlling the growth and activity of immune cells responding to pathogens and cancer.
  • We identified SOCS1 as a top target using our Immune CRISPRomics platform. Editing SOCS1 in TIL using CRISPR/Cas9 has demonstrated remarkable tumor-killing activity in in vivo mouse cancer models, including those refractory to inhibition by PD-1.
  • Our lead eTIL program, KSQ-001EX, is a CRISPR/Cas9 gene-edited eTIL with the SOCS1 gene inactivated. We are currently evaluating KSQ-001EX in the clinic in patients with a variety of treatment-refractory solid tumors.


  • Regnase-1 is an endonuclease that regulates immune responses by degrading the messenger RNA (mRNA) of pro-inflammatory proteins.
  • Using our in vivo CRISPR2TM dual gene-editing screening approach, we identified Regnase-1 and SOCS1 as the genome’s top dual-edit combination in T cells, synergizing to drive best-in-biology activity in mouse cancer models that are refractory to all other therapies.
  • We have translated these findings to our second eTIL program, KSQ-004EX, a Regnase-1 + SOCS1 dual-edited eTIL.


  • USP1 was 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).
  • USP1 is a member of the ubiquitin-specific processing (UBP) family of proteases with a role in DDR distinct from other mechanisms currently being evaluated in the clinic, including PARP inhibitors, which have shown significant clinical utility and opened up a new field of targeted oncology exploiting an approach known as “synthetic lethality.”
  • KSQ-4279 is a first-in-class, potent, and selective small molecule inhibitor of USP1 that has demonstrated strong activity in BRCA mutant preclinical cancer models, including strong combination activity with PARP inhibitors.
  • In July 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).


The clinical manufacture of TIL has historically required a complex and lengthy process. Traditionally, TIL have been obtained solely from surgically resected tumor samples and their manufacture has involved a multi-step process with the use of feeder cells.

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. In comparison to historical TIL manufacturing processes, ExPRESS generates a cryopreserved CRISPR/Cas9 edited eTIL product with a maximized potential to kill cancer cells. Furthermore, ExPRESS robustly manufactures eTIL therapies across a broad range of solid tumor types from surgical resections as well as Core Biopsies, potentially broadening the accessibility of eTIL therapeutics to patients unable to undergo surgical resection.


To date, we have successfully manufactured our investigational eTIL therapies using our ExPRESS process across 10+ solid tumor types using a 22-day manufacturing process. We believe our process as it exists today manufactures the best potential eTIL therapies to deliver Regnase-1 and SOCS1 target benefits to cancer patients. We are working with CTMC, our manufacturing partner, to deliver our eTIL therapeutic candidates to patients as quickly as possible.


As we advance our in-house oncology programs, we continue to explore the full potential of our disruptive targets through the continued discovery of novel candidates. We also continue to explore new biology using our CRISPRomics platform in additional cell types in therapeutic areas outside of oncology in support of new in-house as well as partnered opportunities.