Following a single subcutaneous 240 mg dose, WVE-007 (INHBE GalNAc-siRNA) improved body composition at three months compared ...

The publication, titled “Prime Editing for p47-phox Chronic Granulomatous Disease,” reports initial data for two patients treated in the Phase 1/2 trial of PM359, which was designed to assess safety, ...

The company’s chief medical officer discusses its trajectory with the blockbuster Brukinsa and its strong hematology pipeline.

BGE-102 was well-tolerated in SAD and initial MAD cohorts, with a pharmacokinetic profile supporting once-daily oral dosing Strong target engagement: BGE-102 achieved 90-98% suppression of IL-1β, a ...