MT1621

MT1621 is an investigational oral substrate enhancement therapy composed of a combination of deoxynucleosides—specifically deoxycytidine and deoxythymidine. It is designed to treat thymidine kinase 2 deficiency (TK2d), a rare mitochondrial DNA depletion syndrome that leads to progressive muscle weakness and respiratory insufficiency. The drug works by replacing deficient pyrimidine nucleosides in patients with TK2d, thereby restoring mitochondrial DNA (mtDNA) production and improving cellular energy metabolism. Clinical studies have shown that MT1621 can improve or stabilize motor function and survival in both pediatric and adult patients with TK2 deficiency. The therapy has received Breakthrough Therapy designation from the FDA as well as PRIME designation from the EMA due to its potential to address a high unmet medical need in this ultra-rare disease[5][6][7][8].

Brand names

doxecitine + doxribtimine

Other names

Deoxycytidine/deoxythymidineDoxecitine and doxribtimineThymidine/deoxycytidine