Leveraging our Oligonucleotide Platform Technology
targeting CNS disorders
RNA Modulating Therapeutics
VICO’s platform technology
Our platform technology is built on decades of in-house knowledge and experience on the design, manufacturing and (pre)clinical development of antisense oligonucleotides as RNA modulating therapeutics.
Focusing on RNA as target, our antisense therapies allow correction of genetic mutations at the protein level without inducing permanent changes to the original DNA code. The reversible and transient nature of our RNA modulating therapies therefore offer clear safety advantages.
Our antisense oligonucleotides are simple synthetic but biodegradable stretches of nucleotides that allow high sequence-specificity, straightforward optimization of structure activity relationship, and finetuned implementation of state-of-the-art chemical modifications.
Our technology can be applied to develop drugs with different corrective mechanisms of action:
Inhibiting mRNA translation
The binding of oligonucleotides to mature mRNA to sterically hinder ribosomal translation initiation and/or elongation, thereby conferring lower levels of (toxic) proteins
Modifying pre-mRNA splicing
The binding of oligonucleotides to splice-regulatory sequences in pre-mRNA to sterically hinder the splicing machinery and induce exon skipping or exon inclusion in order to correct the translational open reading frame and restore protein function and/or levels
RNA-editing of point mutations
The binding of oligonucleotides to certain disease-causing point mutations in (pre)mRNA to guide and recruit endogenous enzymes that are naturally capable of changing/correcting a specific nucleotide (like adenosine or cytosine deaminases) and thus restore protein function and/or levels
RNA-editing in premature termination codons (PTCs)
The binding of oligonucleotides to a sequence-stretch containing a disease-causing nonsense mutation in (pre)mRNA to guide and recruit endogenous adenosine deaminase-like enzymes that are capable of changing one or more adenosines in the resulting premature stop codons, in order to convert these into functional codons allowing restored protein function and/or levels
Reducing mutant mRNA levels
The binding of oligonucleotides to a specific sequence-stretch in target (pre)mRNAs to recruit RNaseH and thus reduce levels of transcripts encoding (toxic) proteins