ATL-201

ATL-201 is an investigational divalent small interfering RNA (di-siRNA) therapy developed by Atalanta Therapeutics for the treatment of KCNT1-related epilepsy, a rare and severe early-onset seizure disorder caused by gain-of-function mutations in the KCNT1 gene. The drug is designed to reduce expression of the KCNT1 potassium channel in neurons, thereby normalizing neuronal excitability and suppressing seizures. Preclinical studies have demonstrated that a single dose of ATL-201 delivered via cerebrospinal fluid can achieve broad distribution throughout the brain, potently reduce KCNT1 transcript levels, block pathological potassium currents, and provide durable seizure suppression with favorable tolerability for up to six months in mouse models. An IND submission is planned for 2025[1][4][6][7][8].