First Clinical Trial Begins for ASO Therapy Targeting KCNT1 Mutations

We are deeply moved to see the start of the first clinical trial for an antisense oligonucleotide (ASO) therapy targeting KCNT1 mutations — a moment that once felt far out of reach. This journey began with our daughter, born in 2018, for whom the first ASO — Valeriasen — was developed. At that time, everything was new. The science was pioneering, but also early. Despite all efforts, unforeseen side effects occurred, and in 2021, we lost Valeria. Valeria fought with incredible strength. Her story has become part of a much larger mission: to help all children born with this devastating KCNT1 mutation. We never stopped believing. Together with our dedicated team in Boston and Yale, alongside close collaborators in France and especially one family in France, we continued to push forward. We always believed that Valeriasen was not the end, but the beginning — a first step toward making ASO therapies safer, more precise, and more effective. Today, we are incredibly grateful and proud to see this next step become reality. We warmly congratulate our friend Hélène Tran and her team at Servier on initiating this clinical trial. It represents hope — not only for families affected by KCNT1 mutations, but for the future of precision medicine as a whole. We wish everyone involved great success, strength, and courage for the journey ahead. Valeria’s legacy lives on — in science, in hope, and in every child this work aims to help. Find trial sites here: https://clinicaltrials.gov/study/NCT07227857