A new experimental RNA therapy from Wave Life Sciences has shown potential in helping patients with a rare genetic disorder called alpha-1 antitrypsin deficiency (AATD). This condition prevents the body from making a crucial protein that protects the liver and lungs. In a recent clinical trial, the therapy helped patients produce this missing protein, though the results were not as strong as investors had hoped. The treatment, called WVE-006, works by editing RNA—the molecules that help turn DNA instructions into proteins. This approach is seen as a safer and more flexible alternative to DNA editing. The trial involved patients receiving either a single higher dose or multiple lower doses of the therapy. While protein levels increased in all patients, the higher dose did not produce significantly more protein than the lower doses. Despite this, the therapy was well-tolerated with no serious side effects. Wave Life Sciences plans to continue testing the treatment with monthly dosing and expects more results in early 2026. This therapy could offer a new way to manage AATD, but further research is needed to confirm its long-term benefits.