Prime Medicine receives FDA clearance to test PM647 in humans

Prime Medicine ($PRME) received FDA clearance for its investigational new drug application for PM647, a prime-editing candidate for alpha-1 antitrypsin deficiency. The decision allows a first-in-human Phase 1/2 study of a single intravenous treatment to begin. Initial enrollment will focus on adults with lung manifestations, before considering patients with liver involvement after tolerability has been assessed.

The company expects initial clinical data in 2027. PM647 uses the same lipid-nanoparticle delivery platform as another Prime program, PM577, but that shared platform does not establish the new candidate’s clinical performance. The announcement advances PM647 from preclinical work into human testing; it is not marketing approval, evidence of a cure or a completed demonstration of safety and efficacy.

Sources:

• Prime Medicine — PM647 investigational application clearance, GlobeNewswire release