Our science
At Inverna we are pursuing a novel approach for pseudo-exon activation oligonucleotide drugs to enable ultra-precise activation of single pseudo-exons with very low off-target activity.
Targeting of pseudo-exons to modulate RNA expression
Pseudo-exons represent a new class of therapeutic targets. These dormant exons are present in pre-mRNA but are normally removed during splicing and excluded from mature mRNA. Inverna has discovered how oligonucleotides can activate pseudo-exons and is harnessing this mechanism to create a novel therapeutic platform.
By directing a pseudo-exon–activating oligonucleotide to specific regulatory elements, the RNA-binding protein that normally represses the pseudo-exon is displaced. This “molecular switch” allows the pseudo-exon to be included during splicing, resulting in altered mRNA and protein production.
Inverna’s discovery process
Pseudo-exons have been thought of as a rare phenomenon, however, this is far from true. With PseuFi, our proprietary discovery software, we have generated a comprehensive map of pseudo-exons. This largest-to-date map provides the foundation to rationally design and evaluate oligonucleotides that can precisely activate pseudo-exons as therapeutic targets.
Inverna has licensed its core technology from University of Southern Denmark.
Our technology and the vast identified targeting space unlock new treatment possibilities across many diseases. Today, we are pursuing breakthroughs in neurological disorders such as Parkinson’s disease, somatic repeat expansion diseases, and Huntington’s disease, knowing that the approach can extend to other organ systems.
Setting new standards for pseudo-exon activation
Inverna’s oligonucleotides bind to the regulatory elements of individual pseudo-exons with ultra-high precision. This accuracy minimizes off-target effects and makes allele-specific targeting possible.
The result is safer, more effective therapies that can work at lower doses or target multiple genes simultaneously.
Oligonucleotide activator
no off-target activity
Small molecule activator
significant off-target activity
