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On Monday, Beam Therapeutics Inc. (NASDAQ:BEAM) announced initial safety and efficacy data from its Phase 1/2 trial of BEAM-302, establishing clinical proof-of-concept as a potential treatment for alpha-1 antitrypsin deficiency (AATD) and for in vivo base editing.
The initial safety and efficacy data reported are from all nine patients as of February 26, the data cut-off date.
Preliminary results from the first three single-ascending dose cohorts demonstrated that BEAM-302 was well tolerated, with single doses of BEAM-302 leading to durable dose-dependent correction of the disease-causing mutation.
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