Prime Medicine received FDA IND approval for PM-647, its investigational in vivo prime editor for α-1 antitrypsin deficiency (AATD). The company plans to initiate a first-in-human Phase I/II trial assessing a one-time intravenous infusion in adults with AATD, including patients with lung and liver manifestations. In vivo prime editing is designed to perform precise genetic changes inside the body using an engineered system, with the goal of reducing the lifelong treatment burden common in inherited metabolic diseases. The start of human dosing is a major regulatory milestone for the program. For the gene editing sector, IND clearance for an in vivo prime editing platform in a well-defined monogenic disorder strengthens the pipeline narrative around next-generation editing modalities progressing beyond preclinical proof-of-concept.