A shot that edits the message, not the genes: regulators clear a trial path for a lung-and-liver fix

Alpha-1 antitrypsin deficiency is a cruel inheritance: a single-letter typo in one gene leaves lungs unprotected and lets a toxic protein clog the liver. Now Wave Life Sciences has the FDA's agreement on a registrational path for WVE-006, an injection that fixes the typo in the RNA message — the working copy the cell reads — without touching the DNA itself. One under-the-skin shot is designed to raise the healthy protective protein and clear the toxic buildup, treating lung and liver together; today's IV infusions leave lungs exposed during flare-ups and do nothing for the liver. The plan: a single two-year trial, with a one-year interim look that could support accelerated approval. About 200,000 people in the US and Europe carry the severe form.