Is ONPATTRO a gene therapy? Unpacking RNA Interference vs. Genetic Modification
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4 min read
ONPATTRO (patisiran) was the first-ever small interfering RNA (siRNA) therapeutic approved by the U.S. Food and Drug Administration (FDA) in 2018 for treating a genetic disease. This groundbreaking treatment, which works by "silencing" a gene, has raised a crucial question in the field of genetic medicine: is ONPATTRO a gene therapy?
