BEAM-302

Doctors at Children's Hospital of Philadelphia successfully treated a baby with severe CPS1 deficiency using a personalized CRISPR base-editing therapy, marking a first-of-its-kind approach for this rare metabolic disorder.

Beam Therapeutics' BEAM-302 demonstrated successful DNA correction in alpha-1 antitrypsin deficiency patients, marking the first clinical proof of concept for direct mutation correction using base editing technology.

Beam Therapeutics' BEAM-302 demonstrated the first-ever clinical genetic correction of the disease-causing PiZ mutation in Alpha-1 Antitrypsin Deficiency patients, with a single dose producing durable increases in functional AAT protein.

Beam Therapeutics will present updated data from the BEACON Phase 1/2 trial of BEAM-101 for sickle cell disease at the 2025 Tandem Meetings.

Korro Bio has received approval to begin a Phase I/IIa trial in Australia for KRRO-110, an RNA-editing therapy for alpha-1 antitrypsin deficiency (AATD).