Autifony Therapeutics is focused on pioneering the development of novel treatments for rare central nervous system (CNS) disorders through its expertise in ion channel science, particularly targeting the Kv3 family of potassium channels. Their lead clinical candidates, AUT00206 and AUT00201, are designed to modulate Kv3 channels to address conditions such as Fragile X syndrome and rare epilepsy syndromes. AUT00206 has received FDA Orphan Drug Designation for Fragile X syndrome and is poised to enter Phase 2 clinical trials, demonstrating promising preclinical results in animal models. The company is also exploring the potential of its preclinical-stage assets to treat a wider range of CNS disorders, including schizophrenia and age-related hearing loss.

The scientific foundation of Autifony's approach lies in the understanding of ion channels as critical regulators of neuronal excitability and function. Their proprietary drug discovery platform focuses on developing small molecule modulators that selectively target specific ion channel subtypes, particularly Kv3 channels, which are known for their unique fast kinetic properties that enable high-frequency neuronal firing. By enhancing the function of these channels, Autifony aims to restore normal neural activity and address the underlying pathophysiology of various CNS disorders. Their research highlights the therapeutic potential of Kv3 modulators in improving cognitive and auditory functions, particularly in conditions like Fragile X syndrome and Alzheimer's disease.

United Kingdom

Autifony Therapeutics Limited (“Autifony”) is a clinical stage biotechnology company dedicated to developing new medicines to treat rare genetic CNS disorders by deploying its pioneering ion channel drug discovery platform.

Autifony Therapeutics

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