Our Approach

A bold vision

Thousands of genetically defined diseases have a known root cause. Our product engine enables us to systematically approach drug discovery in genetically defined diseases. We aim to discover and develop disease modifying therapies that result in meaningful outcomes for patients and families.

Our vision is to treat genetically defined diseases by addressing their root cause.

An integrated, patient-driven approach

Disease modeling in relevant human cells & tissue
  • Fully differentiated patient-derived or other relevant human cells from genetically defined disease populations
Scalable cell biology with custom robotics
  • Long-term culturing of human cells to enable comprehensive target identification
  • Robust assays using relevant models
Identification of cellular drug targets to modulate gene expression
  • Multiple chemogenomic and genomic screening approaches
    • Proprietary annotated, pharmacologically diverse small molecule compound library
    • Customized CRISPR-Cas9 guide libraries
Approach Graphic Computational Analyses
  • Unique biology pathway insights to inform target identification and biomarker strategy
  • Proprietary relational database (FulcrumSeek)
Approach Graphic Advanced Physiology
  • Demonstrate functional benefit and maintenance of cell health
  • Genome-wide profiling to understand full impact of drug/target interaction
Profiling of development candidates across multiple patient-derived tissue-relevant cells
  • In vitro analysis of a product candidate to ensure target is representative of disease across populations
  • Provides increased confidence in breadth of therapeutic applicability
  • Generates insights around patient stratification
Biomarker-driven drug discovery and development
  • Biomarker strategy enabled and guided by data from patient-derived cell models