Our AO platform is the right platform for the CNS.
We use Splice Switching Oligonucleotides (SSOs) to modulate protein and isoform expression through a mechanism known as nonsense mediated decay.
SSOs are short, synthetic, antisense, modified nucleic acids that base-pair with a pre-mRNA and disrupt the normal splicing of the transcript by blocking the RNA–RNA base-pairing or protein–RNA binding interactions that occur between components of the splicing machinery and the pre-mRNA.
Splicing of pre-mRNA is required for the correct expression of the vast majority of protein-coding genes, and so by targeting this process it offers a means to manipulate protein production from a gene.
Three SSO drugs have been developed by our extended team and have been given accelerated FDA approvals for the treatment of Duchenne muscular dystrophy.
At BSP we are co-developing structural variant markers with our antisense therapeutics which has the potential to stratify patients for personalised therapies. This approach will lead to a greater chance of therapeutic success for our AOs.
Our AOs have an improved safety profile when compared to other AOs
Our AO approach is the right platform for the CNS