Platform

Bringing Personalized Medicine to ALS Patients

Antisense Oligonucleotides

Antisense oligonucleotides (ASOs) are short, engineered single-stranded DNA/RNA molecules that can selectively bind RNA to regulate its expression in the cell. QurAlis’ scientists are using ASOs to develop treatments for ALS that change the expression of genes connected with the disease.

By delivering these ASOs to motor neurons in ALS patients, we intend to target the mRNA of specific disease related proteins, regulating the protein’s amount and countering the diseased pathway.

Since ALS is not one disease but in fact a spectrum of diseases with numerous distinct subgroups, ASOs are highly suitable drug candidates as they can be tailored for each specific patient sub-population.

QurAlis’ scientists are using ASOs to develop treatments for ALS that change the expression of genes connected with the disease.