ASOs are small synthetic nucleic acid molecules which can bind specific sequences within target RNA molecules. The ASO sequences are specific to target regions in RNA and thus avoid potential off-target effects. ASOs are used for a variety of applications, i.e., splicing modulation, gene silencing or lowering by RNA cleavage and RNA editing.
SpliSense utilizes IP-protected algorithms to support the design and optimization of ASOs for efficiency, and safety including reducing the potential of off target and immunogenic effects.
ASO Technology is Clinically Validated
The high therapeutic potential of ASOs was demonstrated in approved drugs for human genetic diseases (SPINRAZA® – Nusinersen – developed by Biogen, and Ionis for SMA and Exondys 51 (Eteplirsen), an ASO-based drug for treating DMD and others).
The significant effect of these treatments offers a glimpse into the value of the splice switching ASO-based therapeutic approach for disease treatment.
SpliSense Inhaled ASOs – Effective Platform for Pulmonary Diseases
SpliSense treatment is delivered non-invasively, by inhalation, to the lungs with minimal systemic exposure. Our treatment’s efficacy is backed by the company data showing that inhaled ASOs distribute effectively with proper cellular, and nucleus penetration to target lung cells penetrating even through thick and concentrated mucus covering the airways in obstructive lung diseases, with minimal systemic absorption and no toxicity in mice, non-human primates (NHPs) and humans. The treatment is given weekly / every other week by inhalation reducing patients’ treatment burden.