Boston, MA– Atalanta Therapeutics has announced encouraging preclinical results for its experimental RNA interference (RNAi) therapy, ATL-201, aimed at treating KCNT1-related epilepsy — a rare and severe seizure disorder affecting infants and young children. The findings, recently published in the journal Epilepsia, highlight the potential of a single dose of ATL-201 to nearly eliminate seizures in a mouse model over a six-month period.
ATL-201 is designed to reduce expression of the KCNT1 gene, which produces a potassium channel in neurons. Gain-of-function mutations in this gene are known to drive KCNT1-related epilepsy, often leading to near-continuous seizures by six to nine months of age. Current treatments are largely ineffective, leaving affected children with persistent seizures, developmental delays, and intellectual disabilities.
In the study, a single dose of ATL-201 was delivered into the cerebrospinal fluid of mice engineered to carry mutations mimicking those in human patients. The treatment resulted in complete seizure suppression for up to four months, with lasting effects observed for as long as six months. The therapy also showed broad distribution across the brain, including deep regions often inaccessible to other types of oligonucleotide therapies.
“These preclinical results underscore the exciting therapeutic potential unlocked by our di-siRNA platform’s ability to achieve distribution broadly throughout the central nervous system,” said Aimee Jackson, Ph.D., Chief Scientific Officer of Atalanta Therapeutics and an author of the study. “We are working diligently to prepare ATL-201 to enter the clinic in the hopes of bringing the first disease-modifying therapy for KCNT1-related epilepsy to the families who urgently need it.”
Additional data from the study demonstrated that ATL-201 effectively lowered KCNT1 gene expression and blocked abnormal potassium currents in lab tests. The therapy also maintained a favorable safety profile in mice, with no significant changes in liver or kidney biomarkers.
Atalanta plans to submit an Investigational New Drug (IND) application to the U.S. Food and Drug Administration in 2025, marking the next step toward potential human trials.
If successful in clinical development, ATL-201 could represent a groundbreaking advance in treating KCNT1-related epilepsy — a condition for which no disease-modifying therapies currently exist.
