Pathway to Trials Webinar: KCNT1 Research Update from Actio Biosciences. Feb 22, 2025 3:00 ET

As part of our Pathway to Trials education series, we’re excited to share that Actio Biosciences has announced that their KCNT1 program is advancing into preclinical testing.

Join our upcoming webinar where foundation founder, Dr Justin West sits down with Actio co-founder, Dr David Goldstein who will share research findings and its significance for the KCNT1 community. It’s an exciting time to stay informed and explore what’s ahead.

Registration required: https://us02web.zoom.us/.../register/LXfrLtZlQOOHrHAT_csMTg

Actio is developing an oral, first-in-class small molecule inhibitor, ABS-1230, for the treatment of KCNT1-related epilepsy. KCNT1 is a potassium ion channel primarily expressed in the brain that, when functioning normally, regulates the flow of electricity to the brain. Mutations in the KCNT1 gene cause overactivation of the potassium channel and abnormal electrical brain activity, leading to a severe, early-onset epileptic encephalopathy and a high frequency of daily seizures. ABS-1230 is designed to inhibit the overactive ion channel and reduce seizure frequency.

ATL-201 from Atalanta Therapeutics is announced as a promising new treatment for KCNT1-related epilepsy. This report shows suppression of seizures in a genetic mouse model of KCNT1 epilepsy by reducing Kcnt1 transcript with divalent small interfering RNA (siRNA), an emerging variant of oligonucleotide technologydeveloped for the central nervous system. Atalanta is currently advancing its KCNT1 program toward an IND submission this year, with the goal of initiating a Phase 1 clinical trial if approved by regulatory authorities. Atalanta Therapeutics will provide periodic updates to the community on its progress during 2025. 

We are thrilled to announce that the KCNT1 Epilepsy Foundation (KEF) has been selected to join the Chan Zuckerberg Initiative’s (CZI) Rare As One Network and has been awarded a multi-year grant! This incredible opportunity will help us build capacity, transition from a volunteer-driven organization and expand to include more experienced professionals, and advance our mission to drive research collaborations and support families affected by the KCNT1 gene mutation.

Co-founder, Justin West, his family, and Executive Director, Sarah Drislane were interviewed by the LA Times/Daily Pilot about the importance of funding rare disease research.

Shifra Wagner’s six-year-old son Chaim Eliyahu has been medically fragile since birth. She tells a story of powerful love, powerful emunah, and powerful determination as she fights to keep the precious neshamah she’s been entrusted with not just alive, but thriving

We're excited to announce our Million Dollar Bike Ride grant is being awarded to the University College London team including Dr Rajvinda Karda and Dr Ellie Chilcott and Anna Keegan. Other collaborators include Dr Juan Antonio, Dr Amy McTague and Dr Gabriele Lignani. This marks a significant milestone in our journey to advance medical research for KCNT1-related disorders. They will be collaborating on a pioneering RNA editing treatment project.

You can find the Foundation slides here.

Arpin Strong’s board members were thrilled to host the organization’s largest fund-raising event on September 29 at the New England Country Club in Bellingham, MA.

Parents can keep up-to-date on some of the top news and studies.