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A Treatment for Henry

 

While there historically has not been a treatment for AHC, Henry’s parents are developing an ASO treatment for Henry which would be the first treatment for AHC.


We are thrilled to announce that in January 2024, we officially partnered with Boston Children’s Hospital and Dr. Tim Yu’s team to lead the remaining development of Henry’s ASO treatment. Dr. Yu and his team will work in collaborative with Dr. Alfred George’s team at Northwestern University whose ongoing efforts established proof of concept in vitro. We are also working with The Rare Disease Translational Center at the Jackson Laboratories for toxicology testing in mice & a proof of concept in vitro study.

This ASO treatment would serve as a blueprint to treat others with AHC and has implications for hundreds of other neurological diseases.
 
Antisense oligonucleotides, or ASOs, are short strings of DNA or RNA letters that can bind to the mRNA to modify gene expression in the nervous system. ASOs can knockdown (reduce) or upregulate (increase) protein expression, depending on the functional consequence of the mutation and how to correct it. 


We believe a knockdown ASO will be most useful to treat Henry as this type of treatment may be particularly useful for mutations that are dominant negative or which make a toxic protein which adversely affect the wild type (or healthy gene) product in the same cell. These ASOs can “knockdown” such mutations and eliminate these or “toxic” adverse effects.  


Henry is on track to be treated in 14 months which will forever change the course of his life.


We need to raise $1.5M more in order for this to happen.
 

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Updated January 2024
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