Unmask is a pre-clinical stage biotech company. It develops small-molecule therapeutics for mitochondrial disorders and age-related diseases, which are intended to repair the genetic damage underlying mitochondrial dysfunction.
Primary mitochondrial disorders are a group of devastating genetic conditions caused by defects in mitochondrial DNA (mtDNA). They frequently present in childhood or early adulthood and can affect the brain, skeletal muscle, heart, eyes, and other energy-intensive tissues. For most patients, treatment remains supportive or directed toward improving residual mitochondrial function. Few available therapies address the underlying genetic drivers, and there is no broadly applicable treatment that reduces the pathogenic mtDNA burden.
Unmask is developing small molecule drugs designed to selectively reduce pathogenic mtDNA. By lowering mutant mtDNA below the threshold at which cellular energy production is impaired, the approach has the potential to restore mitochondrial function rather than compensate for its loss. Because the strategy is intended to be mutation-agnostic, a single therapeutic approach should be applicable across multiple genetically defined indications. Small-molecule therapeutics also offer practical advantages in manufacturing, distribution, and administration relative to gene-therapy based approaches and chronically injected biologics.
In vitro studies with our lead candidate have demonstrated reduction of mutant mtDNA below the threshold associated with cellular dysfunction, together with restoration of oxidative phosphorylation. Initial rodent studies have shown favorable oral pharmacokinetics. The next stage of development is focused on dose optimization and evaluation of efficacy in relevant animal models.
Unmask Therapeutics, Inc
2897 N Druid Hills Rd NE
Suite 525
Atlanta, GA 30329
