Stoking protein production

Stoke Therapeutics Inc. is developing ASOs to boost normal protein expression as disease-modifying therapies for autosomal-dominant diseases that are not amenable to recombinant protein or gene therapies.

Co-founders Adrian Krainer and Isabel Aznarez previously discovered cell nuclei from patients with some autosomal-dominant disorders harbored reservoirs of precursor mRNA (pre-mRNA), transcribed from the wild-type allele of the disease-related gene, that did not undergo normal splicing. mRNAs generated from these non-productive pre-mRNAs degraded after nuclear export, or did not leave the nucleus at all. ...