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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