Freeing Regulatory Proteins to Facilitate Normal Splicing Function
Expansion’s approach to treating DM1 is focused on designing novel small molecule drugs that can selectively bind the disease-causing toxic CUG repeats in DMPK mRNA and liberate key sequestered proteins so that they can function properly.
The Difference Between "A" and "U" Makes all the Difference...
DM1 patients carry toxic RNA repeat sequences lurking in an intron of the DMPK pre-mRNA.
Myotonic Dystrophy Type 1
r(CUG)exp DMPK mRNA
- to Cure
Myotonic Dystrophy is a Multi-Organ Disease Mediated by Abnormalities in the RNA of a Key Gene
The Expansion Therapeutics Difference
Expansion’s approach to treating DM1 is focused on designing novel small molecule medicines that can selectively bind the disease-causing toxic r(CUG) structures in DMPK RNA. By doing so, these molecules can displace MB from the RNA and free it to fulfill its function and regulate pre-mRNA splicing. In this way, the splicing of hundreds of genes will be corrected across multiple tissues, including brain and heart, as well as muscle, thus restoring function across the whole body.