Antisense oligonucleotides (ASOs) are short, engineered single-stranded DNA/RNA molecules that can selectively bind RNA to regulate its expression in the cell. ASO technology has been leading in the field of protein regulation and has since allowed us to develop treatments for ALS by changing the expression of genes connected to the disease.

QurAlis’ FlexASO™ Splice Modulator Platform was developed to generate splice-switching ASOs with improved potency, increased therapeutics index, and improved bio-distribution, and reduced off-target activity. This bespoke platform has the potential to tackle the spectrum of neurodegenerative and neurological diseases.

QurAlis is open to collaborating with companies on our FlexASO platform for splice modulator targets in applicable diseases.