ADNP syndrome is caused by truncating mutations of the ADNP protein, which is essential for brain formation.
Children with ADNP syndrome suffer from intellectual disability, delayed language acquisition and speech impediments. A new Tel Aviv University study, led by Prof. Ilana Gozes finds that the drug candidate CP201, also known as NAP, may improve vocal communication abilities that are underdeveloped in ADNP patients. Ramot licensed NAP (CP201) to Coronis Neuroscience for development. The U.S. Food and Drug Administration (FDA) has since granted CP201 Orphan Drug status for the treatment of ADNP syndrome in the United States.