The SCN2A Foundation today announced a research collaboration with Unravel Biosciences, Inc., an AI-enabled therapeutics company established to advance drugs for complex diseases. The collaboration intends to advance preclinical research for SCN2A-related disorders caused by loss-of-function mutations, a subset of SCN2A conditions driven by insufficient functional protein.​

SCN2A is a gene critical for normal brain signaling and one of the largest genetic causes of autism and epilepsy. In many individuals, certain genetic changes, including splice-site, frameshift, nonsense, and select missense mutations, result in the body producing too little working SCN2A protein, causing serious neurological symptoms such as epilepsy and neurodevelopmental impairment. This project is specifically designed to address that shared biological problem.

“Our focus is on SCN2A mutations where the fundamental issue is a lack of functional protein,” said Jason Curry, Co-Founder of the SCN2A Foundation. “By partnering with Unravel Biosciences, we are taking a disciplined, mechanism-driven approach to identify strategies that may increase functional SCN2A protein in the brain.”​

“We are excited to collaborate on a program that is clearly defined by the complex patient biology rather than diagnosis alone,” said Richard Novak, PhD, CEO and Co-Founder of Unravel Biosciences. “Precision matters in rare diseases where each patient with a shared diagnosis may have quite different therapeutic responses; this effort reflects a thoughtful approach to matching therapies to the right mutation mechanisms for each patient using our Living Molecular Twin approach.”

Under the collaboration, the teams will evaluate therapeutic approaches in mutation-relevant laboratory models, with an emphasis on understanding how loss-of-function SCN2A protein responds at the RNA and protein level, influenced by other genetic and environmental factors specific to each patient. The work is intended to inform future therapeutic development.

About Unravel Biosciences

Unravel Biosciences is a therapeutics company founded on the principle that biological networks inform clinical outcomes. Unravel leverages its proprietary BioNAV™ platform and primary transcriptomics data to create Living Molecular Twins of real patients that can be used to predict therapeutic response. This enables target and drug discovery, preclinical screening, patient stratification, and clinical validation to find treatments for complex diseases using a systematic, data-driven approach called Predictable Medicine™.

Unravel's platform discovered RVL002, a first-in-class small molecule that targets mitochondrial metabolism and has multiple clinical applications, including neurodegenerative and metabolic disorders, and identified RVL069, a molecule that targets a novel mechanism to treat dystonias. The rareSHIFT™ program provides platform and proprietary datamine access to foundation and biotech partners to accelerate and clinically derisk therapeutics. www.unravel.bio and www.rareshift.org

About The SCN2A Foundation

Founded by the parents of an SCN2A affected son, our mission is to accelerate targeted research and treatment for SCN2A through strategic and cutting edge technologies, processes, and partnerships. For more information, visit https://www.scn2afoundation.org.

To further our epilepsy and autism treatment research, please also consider donating here.

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Under the collaboration, the teams will evaluate therapeutic approaches in mutation-relevant laboratory models, with an emphasis on understanding how loss-of-function SCN2A protein responds at the RNA and protein level.