CP201 (NAP) shows promise in preclinical models as a potential targeted therapy for ADNP syndrome, a rare neurodevelopmental disorder.
CP201 shows preclinical promise in ADNP models but should not yet change practice; leaves open need for dedicated trials.
Activity-dependent neuroprotective protein ( ADNP ) syndrome, also known as Helsmoortel-Van Der Aa syndrome, is a rare condition, which is diagnosed in children exhibiting signs of autism. Specifically, the disease is suspected when a child is suffering from developmental delay and/or intellectual disability. The syndrome occurs when one of the two copies of the ADNP gene carries a pathogenic sequence variant, mostly a de novo mutation resulting in loss of normal functions. Original data showed that Adnp +/− mice suffer from learning and memory deficiencies, muscle weakness, and communication problems. Further studies showed that the ADNP microtubule-interacting fragment NAP (called here CP201) resolves, in part, Adnp deficiencies and protects against ADNP pathogenic sequence variant abnormalities. With a clean toxicology and positive human adult experience, CP201 is planned for future clinical trials in the ADNP syndrome.
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Illana Gozes (2020) studied this question.
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