Beam Therapeutics Inc. announced the clearance of its clinical trial authorisation (CTA) application by the United Kingdom (UK) Medicines and Healthcare Products Regulatory Agency for BEAM-302, an in vivo base editor, as a potential treatment for patients with alpha-1 antitrypsin deficiency (AATD). Preclinical data demonstrated the ability of BEAM-302 to significantly increase levels of corrected and functional alpha-1 antitryps in (AAT) and reduce the mutant PiZ AAT protein in vivo rodent disease models at clinically relevant doses. These findings support the potential of BEAM-302 to efficiently correct the disease-causal PiZ mutation after a single dose and potentially address both the liver and lung disease associated with AATD.

The Phase 1/2 clinical trial is an open-label, dose-escalation study that will evaluate the safety, pharmacodynamics, pharmacokinetics and efficacy of BEAM-302 initially in patients with AATD-associated lung disease. The study design includes a dose exploration portion followed by a dose expansion portion to identify the optimal dose to take forward in a pivotal study. The company expects to initiate the Phase 1/2 trial of BEAM-302 in the UK in the first half of 2024.

BEAM-302 is a liver-targeting lipid-nanoparticle (LNP) formulation of base editing reagents designed to precisely correct the PiZ mutation, a single- letter genetic error found in the majority of severe homozygous patients (PiZZ) living with AATD. Beam is a values-driven organization committed to its people, cutting-edge science, and a vision of providing life-long cures to patients suffering from serious diseases.