ATL201 / Telix - LARVOL DELTA

Durable suppression of seizures in a preclinical model of KCNT1 genetic epilepsy with divalent small interfering RNA. (PubMed, Epilepsia) - "Patients with KCNT1-driven epilepsy experience up to hundreds of seizures per day and have severe impairment in cognitive, motor, and language development and high mortality. The dose-dependent efficacy and long durability of ATL-201 in mice show promise for ATL-201 as a disease-modifying treatment of KCNT1 epilepsy."

Journal • Preclinical • CNS Disorders • Cognitive Disorders • Epilepsy

Durable Suppression of Spontaneous Seizures in a Mouse Model of KCNT1 Genetic Epilepsy with Divalent Sirna, a Novel Small Interfering RNA Technology (AES 2024) - "Patients with KCNT1-driven epilepsy experience up to hundreds of seizures a day and have severe impairment in cognitive, motor, and language development. ATL-201 reduced KCNT1 channel function, suppressed seizures, and restored an aspect of mouse behavior. ATL-201 targets a sequence that does not encompass known pathogenic variants in KCNT1, suggesting it should be effective against any genotype of KCNT1 epilepsy."

Preclinical • CNS Disorders • Cognitive Disorders • Epilepsy