11:27 AM
 | 
Apr 05, 2018
 |  BC Innovations  |  Emerging Company Profile

Stoking protein production

How Stoke is attacking autosomal-dominant disorders with ASOs

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.

Stoke’s idea is to turn the pool of non-productive pre-mRNAs into normal mRNAs that compensate for the protein deficiency caused by the mutant allele. “If you changed some of the splicing in the nucleus at the pre-mRNA level, you could dramatically increase the amount of protein” in...

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