Prime Medicine obtained IND approval from the FDA for PM-647, its in vivo Prime Editor targeting α-1 antitrypsin deficiency (AATD). The company plans to evaluate a one-time intravenous infusion in a first-in-human Phase I/II trial in adults with AATD, including patients with lung and liver manifestations. PM-647 is intended as an in vivo approach to directly edit disease-relevant genetic information using prime editing chemistry, aiming to reduce or correct the underlying molecular defect rather than treat downstream symptoms. With IND clearance, Prime Medicine moves from preclinical and IND-prep work into clinical initiation. For the AATD space, the development is another attempt to translate gene-editing modalities into durable, systemic therapies, where long-term safety, delivery performance and efficacy durability remain key determinants for patient impact.