Hi! I’m Brooke Babineau, the new Scientific Director for the KCNQ2 Cure Alliance (KCA). I’m honored to join this devoted community and support a mission that empowers families through education, meaningful connections, and advances in innovative research focused on the development of new treatments—and ultimately, cures —for people affected by KCNQ2-related disorders.

A bit about me and how I got here:

Growing up, I had a very special person in my life. Her name was Becky. She was about two years younger than I and lived just down the street. Our parents were best friends, and her family was like my own. Becky’s older sister Abby was two years older than I, and since I had no siblings of my own, I had fun imagining myself as their middle child. I don’t remember when I first noticed that Becky was different from other kids, but I know that at a fairly young age, my parents explained she had autism. Becky didn’t speak using words other people would understand; she had a language uniquely her own. I remember learning that ‘tete’ meant band-aid, which she loved and wore in multiples on her arms. Over time, Becky developed more language skills and eventually started calling me ‘Brooka’. I always enjoyed my time with Becky, whether we were re-watching her favorite movies or visiting animals at her favorite farms. As I grew older, my curiosity about autism deepened, and I wanted to really understand why everyday life was so challenging for Becky.

Inspired by my relationship with Becky, I wrote my high school senior thesis on autism. That project launched what has become a 20+ year scientific journey to understand the brain, unravel the underlying biology of neurodevelopmental disorders, and advance new therapeutic solutions.

At the University of California, Davis, I studied a Fragile-X-related condition as an undergraduate and then evaluated the social-emotional impacts of brain injury during my graduate studies. At the National Institute of Mental Health, I learned how to characterize behavior in mouse models with specific genetic changes related to neurodevelopmental disorders, a skill I applied to research projects at the University of California, San Francisco, and Stanford University.

My passion for translating discoveries into new treatments pushed me to pursue drug development. As a research scientist at Encoded Therapeutics, I contributed to advancing projects that evaluated gene therapy candidates for pediatric epilepsy and related disorders. Today, in addition to my role at KCA, I serve as an Associate Director of In Vivo Pharmacology at SonoThera, a genetic medicine company that utilizes the world’s first ultrasound-mediated non-viral platform for delivering genes to the body.

Thanks to an extraordinary investment from the Chan-Zuckerberg Institute, KCA now has the resources to lay the scientific groundwork that will accelerate real progress in advancing new interventions for KCNQ2-related disorders. This support enables us to expand our research team and pursue a clear and ambitious agenda: setting research priorities, forging collaborations with industry and academia, and keeping our community informed every step of the way. We have also brought on a Science Communication Intern, Michelle Keihold, who will support multiple efforts, including the creation of materials aimed at informing industry and clinicians about KCNQ2-related disorders, as well as the research and therapeutic programs we are advancing.

I am energized by the opportunity to contribute to something I care deeply about and the chance to drive progress in a community that still has no approved therapies for KCNQ2-related disorders. With the availability of incredible new tools, including AI, to probe biology more deeply than ever, we’re poised to enable the discovery of new insights that we hope will translate into meaningful medical interventions for families and everyone living with KCNQ2.

This blog will play a key role in that effort, serving as a channel for our community to stay informed about KCA’s ongoing progress. Stay tuned for our next post, where we will discuss our goals and current research priorities in more detail.

Want to get more involved? We are always looking for people who are motivated to help us advance our progress! Email us at: info@kcnq2cure.org.