ADNP
Syndrome.

ADNP Syndrome and NAP: Pioneering Treatment for a Rare Neurodevelopmental Disorder

Understanding ADNP Syndrome

ADNP Syndrome is a rare neurodevelopmental disorder caused by mutations in the Activity-Dependent Neuroprotective Protein (ADNP) gene. This condition is part of a group of genetic disorders known as autism spectrum disorders (ASDs) and is characterized by a wide range of symptoms, including developmental delays, intellectual disabilities, speech and language impairments, and various physical abnormalities.

The ADNP gene plays a crucial role in brain development, synaptic function, and the regulation of various cellular processes. When this gene is mutated, it leads to the disruption of normal brain function, resulting in the array of symptoms seen in ADNP Syndrome.

The Role of NAP in ADNP Syndrome

NAP (NAPVSIPQ) is a neuroprotective peptide derived from the ADNP protein. Discovered by Professor Illana Gozes, NAP has been shown to stabilize microtubules, which are essential for maintaining cell structure, intracellular transport, and overall neuronal health. In the context of ADNP Syndrome, where the ADNP gene is mutated and its protective functions are compromised, NAP offers a potential therapeutic approach.

Research has demonstrated that NAP can mitigate some of the cellular dysfunctions associated with ADNP mutations. By stabilizing microtubules and enhancing neuronal resilience, NAP could potentially alleviate the neurological and developmental challenges faced by individuals with ADNP Syndrome.

Clinical Development: Moving Towards a Phase 3 Trial

Recognizing the promise of NAP as a treatment for ADNP Syndrome, we are excited to announce that we are preparing to commence a Phase 3 clinical trial in children during Q4 of this year. This trial represents a significant milestone in the development of a targeted therapy for ADNP Syndrome, aiming to confirm the safety and efficacy of NAP/Davunetide in a pediatric population.

Our goal is to provide a therapeutic option that can improve the quality of life for children affected by this debilitating disorder. By addressing the underlying molecular dysfunction, NAP/Davunetide has the potential to make a meaningful impact on the developmental trajectory of these patients.

The Vision: A Future of Hope for ADNP Syndrome

At ExoNavis Therapeutics, our vision is to bring hope to families affected by ADNP Syndrome through innovative, science-driven therapies. The upcoming Phase 3 trial is a crucial step towards making NAP/Davunetide a reality for those in need. We are committed to advancing this research, with the ultimate goal of securing regulatory approval and making NAP/Davunetide available as a treatment option for ADNP Syndrome.

By unlocking the therapeutic potential of NAP, we aspire to change the future for individuals with ADNP Syndrome, providing them with new possibilities for growth, development, and a better quality of life.