Application

Chemotherapy remains a cornerstone of cancer treatment, aiming to eliminate rapidly dividing tumor cells through cytotoxic agents.

However, response to treatment varies widely among patients due to genetic differences in tumors. Paclitaxel and carboplatin are frontline chemotherapy drugs for multiple cancers, yet resistance to these treatments remains a major clinical obstacle. Up to 30–50% of patients exhibit innate or acquired resistance, resulting in ineffective therapy and reduced survival. Current methods lack predictive power for drug responsiveness at the individual level, leading to trial-and-error treatments.

Our Innovation

This novel platform utilizes genome-wide CRISPR/Cas9 loss-of-function screens in haploid human embryonic stem cells (hESCs) to identify key genes that confer resistance to paclitaxel and carboplatin. By linking specific gene disruptions to drug resistance phenotypes, the platform enables development of a predictive algorithm. This algorithm, when applied to a patient’s tumor genetic profile, can stratify patients as likely responders or non-responders, improving clinical decision-making and treatment planning.

Advantages

  • High-Resolution Screening: Haploid hESCs allow clear identification of gene-function relationships without the masking effects of gene redundancy.
  • Clinically Validated Algorithm: Predictive model applied to TCGA patient data showed high correlation with treatment outcomes and survival.
  • Key Biomarkers Identified: Genes like TP53 and PTEN are central to drug resistance and were validated in both cell lines and patient tumors.
  • Actionable Insights: Enables early treatment adjustment, reducing exposure to ineffective therapies and improving outcomes.

Commercial Opportunity

With a global oncology market exceeding $200 billion and precision oncology rapidly expanding, this platform offers:

  • Companion diagnostics for chemotherapeutic selection.
  • Licensable IP around drug-resistance gene panels.
  • Partnering potential with pharma for patient stratification in clinical trials.
  • Regulatory pathway for CDx development supported by retrospective validation on real-world patient data.

This innovation positions itself at the intersection of functional genomics and precision oncology, offering an immediately translatable tool to improve cancer care.