BEAM's IND for BEAM-302 in Genetic Disorder Study Gets FDA Clearance

Core Insights - Beam Therapeutics Inc. has received FDA clearance for its investigational new drug application for BEAM-302, aimed at treating alpha-1 antitrypsin deficiency (AATD) [1] - AATD is a genetic disorder leading to early emphysema and liver disease, with no approved curative treatments currently available [2] - The company has reported positive initial safety and efficacy data from the phase I/II study of BEAM-302, indicating it was well tolerated and led to dose-dependent correction of the disease-causing mutation [4][5] Company Developments - The phase I/II study of BEAM-302 consists of two parts: Part A focuses on AATD patients with lung disease, while Part B will target patients with mild-to-moderate liver disease [6] - The first patient in Part B is expected to be dosed in the second half of 2025, with additional patients to be enrolled in a fourth-dose cohort [8] - Year-to-date, shares of Beam Therapeutics have declined by 10.6%, contrasting with a 3.3% increase in the industry [3] Market Context - Beam Therapeutics currently holds a Zacks Rank of 3 (Hold), while other biotech stocks like Jazz Pharmaceuticals, Dynavax Technologies, and Krystal Biotech have better rankings [9]