In Vivo Base Editing Gets Boost with Positive Initial Data Announced by Beam Therapeutics

Beam Therapeutics has announced promising initial results from its Phase I/II clinical trial of BEAM-302, a base-editing therapy designed to treat alpha-1 antitrypsin deficiency (AATD), a severe genetic disorder affecting the lungs and liver. The trial represents a significant milestone in in vivo base editing, offering the potential for a one-time curative treatment by correcting the disease-causing PiZ mutation at the DNA leve

GENE THERAPY RESEARCH