RNA interference (RNAi) Approaches for Allele Specific, Gain of Function Mutations in Rare, Monogenic Neurodevelopmental Disorders

Time: 5:30 pm
day: Day 2 afternoon


  • Advances in DNA sequencing has rapidly accelerated the identification and characterization mutations at the heart of rare neurodevelopmental and neurodegenerative disorders. More than 7,000 rare, monogenic disorders are known, of which 40% are neurological in nature.
  • Knowing the root causes of monogenic disorders has allowed mutationspecific therapies possible. To that end, use of RNAi as a gene silencing therapy is an attractive approach for disorders where gain-of function mutations are the underlying etiology such as the case with KIF1Aassociated Neurological Disorder (KAND). Technologies that can address
    allele-specific mutations will be critical for therapeutic development.
  • Ovid Therapeutics is focused in areas of significant unmet medical need and where opportunities to leverage academic, patient organizations and patient data are available to accelerate discovery and development of novel and innovative therapies.