Refreshgene’s siRNA Therapy Enables Children with Genetic Deafness to Hear

A siRNA therapy developed by Shanghai Refreshgene Therapeutics enables children with genetic deafness to hear. In a China-US trial, six children aged one to seven were able to hear for the first time after a single dosing of an siRNA therapy that used an adeno-associated virus (AAV) to deliver a functioning copy of the human otoferlin gene to previously deaf children. The therapy was effective in five out of the six patients. In the five patients who responded, all were able to hear after a few months and also learned how to speak. The trial results were published in the Lancet on Wednesday. More details....

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