ASOs are small synthetic nucleic acid molecules which can bind specific sequences within target RNA molecules. The ASO sequences are specific to the target mutation region in RNA and thus avoid potential off-target effects. ASOs were shown to penetrate the cells vis endocytosis with no delivery vehicles. ASOs are used for a variety of applications, i.e., splicing modulation, gene silencing by RNA cleavage and RNA editing.
SpliSense uses splice switching ASOs that align to the target sequence, such as consensus splicing motifs or splicing enhancer/silencer sequences. ASO is a precise targeting approach, which allows manipulation of specific exons without impacting other regions of the sequence.
SpliSense utilizes an IP-protected algorithm to support the design of ASOs that optimizes the efficiency of the ASO, reducing the potential for off target and immunity effects.
ASOs Technology is Clinically Validated
The high therapeutic potential of ASOs was demonstrated in approved drugs for human genetic diseases (SPINRZA ® – Nusinersen – developed by Biogen, and Ionis for SMA and Exondys 51 (Eteplirsen), an ASO-based drug for treating DMD).
The significant effect of these treatments offers a glimpse into the value of the splice switching ASO-based therapeutic approach for genetic disease treatment.
Inhaled ASOs – Effective Platform for Pulmonary Diseases
SpliSense treatment is delivered non-invasively, by inhalation, carrying large doses to the lungs with minimal systemic exposure. Our treatment’s efficacy is backed by research, showing that inhaled ASOs perform effective distribution of medication with cellular penetration and minimal systemic absorption and no toxicity in mice, non-human primates (NHPs) and humans. The treatment is administered weekly, reducing patients’ treatment burden.