STRATEGIC RESEARCH ROADMAP

​PATH TO A CURE

1.  BUILD ADNP "MODELS"

• Mouse Models (ADNP Kids Research Foundation funded the creation of a mouse model line at Mount Sinai's Seaver Autism Center.  We are hoping to have a confirmed/validated mouse in 2021)
• Patient specific pluripotent stem cell models (5 ADNP patient iPSCs are held at Mount Sinai Seaver Autism Center as well as a line at University of Washington)
• Zebrafish Models

2.  STUDY THE MODELS

• Understand the impact of ADNP mutations on the brain and body:  Molecular, cellular and behavioral
• Identify robust biomarkers and endpoints (diagnostic biomarker identified, visual evoke change biomarker, and 2 types of methylation in ADNP) (endpoints are being validated through phase 2 FDA ketamine drug trial)
• Learn what time-points are effective for treatments 

3.  TEST THERAPIES ON MODELS

• Screen approved drugs
• Identify and screen various therapies including: Gene Therapies, ASOs. etc.
  

4.  SAFETY STUDIES

• Conduct toxicology and efficacy of new successful drugs and therapies on wild animal types 

5.  PATIENT CLINICAL TRIALS

• ​FDA and International Regulatory Agency review
• (*2021 - Conducting efficacy and safety phase 1/2 FDA low dose ketamine drug trial)

This is our Strategic Research Roadmap.  Please review our Treatment Research and Development Updates here.

---