Science Science

Biomarker-Driven Development

The development path of each program is enabled by biomarkers to monitor early effects of the drug and select the right patients and the right dose in clinical development.

Biomarker-Driven Development
Patient Engagement Biomarker

Target Engagement

In the design of our Phase 1 trials, we integrate our target engagement biomarker data with pharmacokinetic analysis from both plasma and CSF to determine the relationship between dose, time, drug exposure and drug response. We develop integrated exposure response models that enable quantitative dose selection for future patient studies.

Pathway Engagement Biomarker

Pathway Engagement

In building deep expertise in the biologic understanding of degenogenes, we can identify and use pathway biomarkers to monitor effects on target biology that may be relevant to human disease outcomes. Demonstration of pathway engagement further de-risks our targets by establishing an effect on the relevant biology.

Target Engagement Biomarker

Patient Phenotyping

Patient phenotyping is the intersection of Pathway Biology and Disease Pathology. In building deep expertise in both disease biology and the pathway biology of degenogenes, we enable a patient selection strategy that can better target and select the best patient population for our clinical trials and product candidates.

Product Candidate Biomarkers

As part of our strategy, we are developing proprietary reagents and assays to enable biomarkers for each of our core programs. These biomarkers, which are relevant for both animal models and human trials, are critical for patient selection, predicting and measuring target engagement, supporting dose selection and enabling decisions on progression of product candidates to the next phase of development.

  • Preclinical
  • Clinical Data
Target Engagement BiomarkersPathway Engagement BiomarkersPatient Phenotyping Biomarkers
TargetTarget Engagement BiomarkersPathway Engagement BiomarkersPatient Phenotyping Biomarkers
LRRK2LRRK2pS935, Rab10pT73Rab10pT73, BMPLRRK2 mutation
RIPK1RIPK1pS166CytokinesNf-L, genetic data, Amyloid/Tau biomarkers of disease
ETV:IDSCNS GAGsBMP, lysosomal lipidsIDS mutation, lysosomal lipids
ATV:aSynBound/free Synuclein DAT imaging
ATV:TREM2Bound/free sTREM2 Amyloid/Tau biomarkers of disease
ATV:TauAbeta, pTau Amyloid/Tau biomarkers of disease
  • Preclinical
  • Clinical Data
Target Engagement Biomarkers
TargetTarget Engagement Biomarkers
LRRK2LRRK2pS935, Rab10pT73
ATV:aSynBound/free Synuclein
ATV:TREM2Bound/free sTREM2
ATV:TauAbeta, pTau
  • Preclinical
  • Clinical Data
Pathway Engagement Biomarkers
Targetpathway engagement biomarkers
LRRK2Rab10pT73, BMP
ETV:IDSBMP, lysosomal lipids
  • Preclinical
  • Clinical Data
Patient Phenotyping Biomarkers
TargetPatient Phenotyping biomarkers
LRRK2LRRK2 mutation
RIPK1Nf-L, genetic data, Amyloid/Tau biomarkers of disease
ETV:IDSIDS mutation, lysosomal lipids
ATV:aSynDAT imaging
ATV:TREM2Amyloid/Tau biomarkers of disease
ATV:TauAmyloid/Tau biomarkers of disease