RAPT - Beam Therapeutics Begins Dosing in Phase I/II AATD Study | Benzinga
Beam Therapeutics Inc. (NASDAQ: BEAM) announced that it has dosed the first patient in the phase I/II study, evaluating its investigational in vivo base editing therapy, BEAM-302, for the treatment of alpha-1 antitrypsin deficiency (AATD).
The open-label, dose-escalation study will investigate the safety, pharmacodynamics, pharmacokinetics and efficacy of BEAM-302 for the given indication. The study is designed to identify the optimal dosage of BEAM-302.
AATD is an inherited genetic disorder that can cause early onset of emphysema and liver disease. Currently, there are no curative treatments approved for AATD.
BEAM received clearance for its clinical trial authorization application from the United Kingdom Medicines and Healthcare Products Regulatory Agency for BEAM-302 in March 2024.
Shares of Beam Therapeutics have declined 8.7% year to date compared with the industry's decrease of 4.6%.
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BEAM is also developing another pipeline candidate in its genetic disease portfolio, BEAM-301. The company is looking to initiate ...