MR-004

MR-004 is a first-in-class small molecule inhibitor of Transmembrane Protein 106B (TMEM106B), currently under development by Muna Therapeutics. TMEM106B is a lysosomal membrane protein that acts as a key genetic modifier in frontotemporal dementia (FTD) and other neurodegenerative disorders. Elevated levels or specific risk variants of TMEM106B are associated with lysosomal dysfunction and accelerated disease progression, particularly in patients with progranulin (GRN) mutations. By inhibiting TMEM106B, MR-004 aims to restore lysosomal health, normalize progranulin levels, and prevent the formation of pathological protein aggregates, such as TDP-43. The drug is primarily being investigated for the treatment of frontotemporal dementia and potentially other tauopathies or synucleinopathies where lysosomal clearance mechanisms are compromised.