Audion’s lead program, a small molecule gamma secretase inhibitor AUD1001, is in clinical development for acquired hearing loss.
Sensorineural hearing loss represents 90% of all hearing loss and is due to damage to the structures of the inner ear and/or the auditory nerve. It is most frequently characterized by dysfunction or death of the hair cells in the cochlea.
Gamma secretase inhibition in the cochlea has been shown to induce transdifferentiation of supporting cells into hair cells, restoring the lost function (Mizutari et al., Neuron 2013). Furthermore, recent insights have shown that inhibiting Gamma Secretase induces the formation of synapses enabling the transmission of sound signals to the auditory nerve.