miREX

miRNA sponge for X-Gene Regulation

Alcyone’s miREX platform addresses X-linked genetic disorders by partially re-activating the silent X-chromosome in affected females through a novel epigenetic mechanism. Due to the natural process of random X-chromosome inactivation, about 50% of cells in females suffering from X-linked disorders express a healthy, functional copy of a gene while the remainder of cells will express a damaged version. This variation depends on whether a healthy X chromosome, or one carrying an X-linked mutation, is active. Traditional gene therapy would deliver a replacement gene coding sequence to both healthy and affected cells, relying on artificial elements to regulate expression levels. This approach can result in overexpression of gene products in healthy cells which might cause unwanted side effects.

Alcyone’s approach does not need artificial regulation, instead, it reactivates the cell’s own healthy gene-copy from the natural silent locus utilizing gene-control elements already present to avoid the risks associated with overexpression.