Beam reports early mutation-correction signals

Beam Therapeutics reported preliminary clinical evidence that its base-editing treatment BEAM-302 may correct the PiZ mutation that causes alpha-1 antitrypsin deficiency (AATD). In a Phase 1/2 study, a single dose was associated with increased total and functional AAT and reduced mutant AAT; effects rose with dose. The early results are proof-of-concept, not confirmation of long-term benefit or safety. Beam said it plans to use a proposed $500 million financing to advance the program.

Separately, Beam announced initial data for BEAM-301, a different investigational treatment designed to correct the R83C mutation in glycogen storage disease type Ia (GSDIa). Early results described restoration of the body’s own glucose production after one treatment in the small Phase 1/2 trial.

Pfizer and Enodia Therapeutics announced a research and licensing collaboration to discover small-molecule inhibitors of Sec61, a protein complex involved in newly made proteins. Enodia will conduct early discovery work; Pfizer would take over clinical development, manufacturing, regulatory and commercial activities afterward. The agreement could exceed $500 million in upfront, milestone and royalty value, though its detailed financial terms were not disclosed.