Technology

Leveraging our Oligonucleotide Platform Technology
targeting CNS disorders

RNAMODULATING THERAPEUTICS SCA1, HD Inhibiting mRNAtranslation RETT RNA-editingpoint mutations RETT RNA-editingPTCs NOT DISCLOSED Reducing mutantmRNA levels SCA3 Modifying pre-mRNA splicing

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