Lead Identification 

Well established and proven process including:

Bioinformatics assessment for in silico sequence pre-selection

  • Considering species cross-reactivity
  • Proprietary algorithm for selection of specific oligonucleotide (avoiding off-targets)

Oligonucleotide design (structure and chemistry) and synthesis

Lead identification by high throughput in vitro screening

  • Assay development for monitoring of mRNA, protein or phenotype by technologies including Quantigene®, (q-) PCR, ELISA and cell based assays
  • Efficacy screening in established cell lines or primary cells by transfection and/or direct incubation

Iterative process for lead characterization and optimization

  • Determination of IC50
  • Analysis of immuno-stimulatory properties (PBMC assay)
  • Oligonucleotide safety
    • In vitro specificity and off-target analysis
    • In vitro monitoring of toxicity (e.g. xCELLigence system)
  • Stability analysis in biological matrices
  • Educated chemical modification

In vivo efficacy and early safety assessment of oligonucleotide-based therapeutics