Prime Medicine said Tuesday it will ask the Food and Drug Administration to approve a gene-editing treatment that has been given to only two patients.
The application will test an agency that has promised to speed new gene-editing treatments to patients but has recently come under scrutiny from regulators, executives, and advocates for spurning a string of gene therapies for neurological diseases.
Prime’s drug is the first to employ prime editing, a CRISPR-based tool debuted in 2019 by Broad Institute biochemist David Liu for making virtually any small change to DNA. The treatment is designed to insert two missing DNA letters into the blood cells of people with one form of chronic granulomatous disease (CGD), which leaves patients susceptible to life-threatening infections and debilitating inflammatory disorders.
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