Beam Therapeutics’ clinical trial of BEAM-302 in AATD cleared in the UK

Beam Therapeutics announced the clearance of its clinical trial authorisation – CTA – application by the U.K. 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, or AATD. AATD is an inherited genetic condition that can cause serious lung and liver disease, which can lead to significant debility and reduced life expectancy for patients. Preclinical data demonstrated the ability of BEAM-302 to significantly increase levels of corrected and functional alpha-1 antitrypsin and reduce the mutant PiZ AAT protein in in vivo rodent disease models at clinically relevant doses. The Phase 1/2 clinical trial is a 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.