Investigational Drug for Huntington's Disease Granted Orphan Drug Status

There are currently no available treatments to slow down the progression of Huntington's Disease
There are currently no available treatments to slow down the progression of Huntington's Disease

Wave Life Sciences announced that the Food and Drug Administration (FDA) has granted Orphan Drug designation to the investigational drug WVE-120101 for the treatment of Huntington's disease (HD).

Huntington's disease is a hereditary neurodegenerative disease that causes the progressive breakdown of nerve cells in the brain. It is a fatal disorder with currently no available treatments to slow down the progression or reverse the course of the disease. 

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WVE-120101 is an allele-specific antisense therapy which targets rs362307, a Single Nucleotide Polymorphism (SNP) that is associated with the disease-causing mutation in the huntingtin (HTT) gene.

For more information visit Wavelifesciences.com.


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