PMS1-targeting program

Rgenta Therapeutics is developing first-in-class oral small molecule splice modulators targeting PMS1 (PMS1 protein homolog 1) for the treatment of repeat expansion diseases (REDs), including Huntington's Disease (HD) and Myotonic Dystrophy Type 1 (DM1). The program utilizes Rgenta's proprietary RNA-targeting small molecule discovery platform to modulate the splicing of PMS1 pre-mRNA, which reduces the production of the aberrant PMS1 protein. PMS1 is a critical component of the DNA mismatch repair (MMR) pathway, which has been implicated in the pathological somatic expansion of trinucleotide repeats. By curtailing PMS1 levels, these compounds aim to inhibit repeat expansions and potentially modify the trajectory of neurodegenerative and neuromuscular disorders. The program is currently in preclinical development and has demonstrated the ability to cross the blood-brain barrier and show efficacy in animal models.

Other names

PMS1 splice modulatorPMS-1 splice modulatorPMS 1 splice modulator