RNA Therapeutics

Translational RNA-Editing Analytics

Making editing datasets decision-ready

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Computational workflows for RNA-editing drug discovery: editing efficiency, target engagement, isoform effects, off-target prediction and transcriptomic safety readouts.

Approach

  • RNA-seq experimental design and interpretation
  • ADAR-mediated editing efficiency quantification
  • In silico off-target prediction for oligonucleotide drugs
  • Reproducible pipelines in R, Python, Linux/HPC, Nextflow
  • Automated QC and reporting

Senior Scientist & Project Leader — ProQR Therapeutics (2025–present)


In my current role as Senior Scientist and Project Leader for the Bioinformatics Platform at ProQR Therapeutics, I bring a mechanistic and quantitative approach into therapeutic development. I lead transcriptomic and bioinformatics efforts supporting RNA-editing programs for genetic disease, including analyses of target engagement, editing efficiency, isoform effects and off-target risk.

What the work actually delivers

The impact is to make RNA-editing datasets interpretable and decision-ready for discovery teams. I connect molecular readouts with disease biology, mechanism of action, candidate selection, lead prioritization and translational planning.

I design reproducible pipelines, automated reporting workflows and data infrastructure that support decision-making across preclinical programs, while also exploring AI-assisted approaches for predicting editing outcomes and prioritizing lead candidates.

CNS discovery

I support CNS discovery programs in epilepsy, Rett syndrome and Alzheimer's disease by prioritizing oligonucleotide candidates and characterizing disease models — and contributed pharmacodynamic and biological data analyses supporting the investigational new drug application for AX-810 in cholestatic disease.

Why this matters

Turns high-dimensional editing data into candidate selection, lead prioritization and cross-functional project decisions — computational biology as a project-facing scientific function, not a downstream analysis service.

Highlights

  • ADAR
  • Editing efficiency
  • Target engagement
  • Off-target prediction
  • Nextflow
  • FAIR data
  • IND-supporting analysis

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