Ascidian's lab in Boston is developing a new kind of RNA editing therapy (Credit: Kevin Trimmer)

First-ever RNA edit­ing tri­al in US gets clear­ance, pit­ting ‘ex­on edit­ing’ tech­nol­o­gy against vi­sion loss

The FDA told the Boston start­up As­cid­i­an Ther­a­peu­tics that it can be­gin the first clin­i­cal tests of a ther­a­py that “rewrites” RNA to cor­rect rough­ly two-thirds of the ge­net­ic ty­pos re­spon­si­ble for an in­her­it­ed form of vi­sion loss called Star­gardt dis­ease.

Tin­ker­ing with RNA, the short-lived cousin of DNA, could low­er some of the hy­po­thet­i­cal risks as­so­ci­at­ed with tech­nolo­gies like CRISPR that make per­ma­nent changes to the genome. That ar­gu­ment, made by As­cid­i­an and oth­er biotechs de­vel­op­ing RNA edit­ing ther­a­pies, now has back­ing from US reg­u­la­tors.

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