Knobias

Nobias Therapeutics is a clinical-stage biotechnology company focused on developing precision medicines for genetically defined rare diseases, with a primary emphasis on pediatric neuropsychiatric conditions. The company leverages a proprietary AI-driven platform that integrates large-scale genomic data with longitudinal electronic health records to identify novel drug targets and repurposing opportunities. Its lead candidate, NB-001 (fasoracetam), is a small-molecule metabotropic glutamate receptor (mGluR) activator currently in Phase 2 development for the treatment of 22q11.2 deletion syndrome (DiGeorge syndrome). Nobias aims to address the significant unmet medical needs of children with 22q11.2 deletion syndrome who experience comorbid symptoms of ADHD, anxiety, and autism. The company maintains a strategic partnership with the Center for Applied Genomics at the Children's Hospital of Philadelphia to access extensive patient datasets. Recently, Nobias reported positive Phase 2 data and is preparing for registrational studies.