Beam Therapeutics received FDA clearance for its Investigational New Drug application for BEAM-304. This approval allows the company to initiate a Phase 1/2 clinical trial to evaluate the safety and efficacy of the treatment.
BEAM-304 is an in-vivo base editing therapy designed to treat phenylketonuria (PKU). The therapy aims to directly correct the gene mutations that cause this rare inherited metabolic disorder.
PKU affects approximately 20,000 people in the United States. The condition results in a toxic buildup of the amino acid phenylalanine in the body.
Patients currently manage the disorder through a strict lifelong diet. Without intervention, the condition can lead to serious neurological and cognitive impairments.