Our Approach
Highly Selective GCN2 Inhibitors
At Alesta Therapeutics, our highly selective GCN2 inhibitors demonstrate exceptional primary pharmacology, characterized by low nanomolar potency and oral bioavailability. Target engagement has been clearly demonstrated, underscoring the efficacy of our compounds.
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Disease
GCN2 Activation in Charcot-Marie-Tooth Disease
Alesta Therapeutics is focused on addressing Charcot-Marie-Tooth (CMT) disease, a debilitating inherited neurological disorder that impacts motor and sensory functions in the hands and feet. This condition affects approximately 126,000 people in the United States and 2.6 million individuals globally.
Research has identified specific genes involved in the loading of tRNA with amino acids, known as tRNA synthetases, as an underlying cause of CMT. Among these, tRNA synthetase variants, such as GARS, are particularly significant. The prevalence of tRNA synthetase-related CMT is estimated to be between 2,000 and 3,000 cases in the US.
At Alesta Therapeutics, we are dedicated to developing innovative therapies that target the activation of GCN2 in CMT, aiming to provide new hope for patients affected by this challenging condition.
NORMAL TRNA LOADING
PHYSIOLOGICAL
SITUATION
MUTANT TRNA LOADING
TRNA SEQUESTRATION
> Ribosome stalling
> GCN2 activation
> CMT