We are building a strategic roadmap to find treatments for POGZ disorders. We are on a road to cure.

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This is our strategic roadmap to find treatments for POGZ-Related Syndrome/White Sutton Syndrome

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Therapeutic Program 1 :

Gene Replacement Therapy

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The goal of our gene replacement therapy program is to insert a healthy copy of the POGZ gene into cells in the body of individuals living with POGZ syndrome, to replace the loss of POGZ protein caused by the mutations. This is done through injection of an AAV9 viral vector that encodes the healthy POGZ gene.

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Basic Steps to build a Gene Replacement Therapy:

 

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Therapeutic Program 2 :

POGZ Drug Repurposing Therapy

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Drug Repurposing involves taking a drug that has been successful for another target and using it to treat symptoms of POGZ disorders. The drugs are small molecules which are chemical compounds with a lower molecular weight (like Tylenol). The process involves scanning vast libraries of small molecules to identify "hits" with therapeutic potential. Small molecules can be very important in treating important symptoms for POGZ disorders like movement disorders, memory, communication etc. 

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CPDF urgently need to run a 'High Throughput Screening' to find the right compound that will impair neural functions in POGZ mutant neurons. 

 

Basic Steps to build an HTS Program:

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