Atalanta Therapeutics has published preclinical data in Epilepsia supporting the efficacy of its investigational RNAi therapy, ATL-201, for KCNT1-related epilepsy. In a mouse model, a single CSF-administered dose led to broad brain distribution, durable seizure suppression for up to six months, and behavioral improvements, all with favorable tolerability. An IND filing is planned for 2025.

These results highlight the potential of our di-siRNA platform to broadly reach the CNS and deliver disease-modifying treatments, said Aimee Jackson, Ph.D., CSO of Atalanta. “We’re working to bring the first targeted therapy to families affected by KCNT1-related epilepsy.”

KCNT1-related epilepsy is a rare, severe pediatric seizure disorder driven by gain-of-function mutations in the KCNT1 gene. ATL-201 aims to lower KCNT1 expression and normalize neuronal activity. Study highlights include potent in vitro knockdown of KCNT1, deep CNS penetration, dose-dependent seizure suppression, and no observed toxicity in treated mice.

Source: https://www.atalantatx.com/atalanta-therapeutics-publishes-preclinical-data-supporting-investigational-rnai-therapy-for-kcnt1-related-epilepsy/