Spinal Bulbar Muscular Atrophy (SBMA), or Kennedy’s disease, is a rare, inherited neuromuscular disorder that leads to progressive muscle degeneration, weakness, and twitching. While it affects approximately 1 in 100,000 individuals worldwide, currently, there are no approved treatments for SBMA, leaving a substantial unmet medical need.
Our program focuses on developing a novel therapeutic approach targeting PRMT6, a co-activator of the Androgen Receptor (AR) implicated in SBMA progression. The aim is to design a highly selective, brain-penetrant PRMT6 inhibitor targeting an allosteric site on the enzyme. Preliminary studies have shown that inhibiting PRMT6 reduces mutant AR activity and improves motor function in disease models.
M4ND is wholly owned by Agora Open Science Trust, a Canadian charity that promotes open science in drug discovery.
Each month, our scientific team, collaborators, and partners gather online to share updates and discuss the latest advancements in our PRMT6 project for SBMA. These meetings are open to anyone interested. You can find past recordings here.