Lead molecules are chemical compounds (here: oligonucleotides) that show a particular desired biological or pharmacological activity. Selected lead candidates are the basis for the development of a new clinically relevant compound.  

The lead identification process includes several steps that may be repeated iteratively before suitable lead candidates emerge. Those are further characterized and optimized.

Lead identification is a well-established and proven process at Axolabs. 

• An upstream bioinformatics assessment is performed for an in silico sequence pre-selection using our proprietary algorithm.
• Promising oligonucleotides are designed (structure and chemistry) and manufactured in a small scale.  
• These molecules undergo a high throughput in vitro screening process.
• Oligonucleotide safety is assessed by the analysis of immuno-stimulatory properties, in vitro specificity, off-target effects and by an early safety assessment in vivo.
• Assays are developed and applied for monitoring of mRNA, protein or phenotype.  
• An efficacy screening is performed in established cell lines or primary cells.  
• An educated chemical modification for the determination is performed to study structure-function relationships.
• The stability is analysed in relevant biological matrices.