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- Beam Therapeutics reports year-long reduction in sickle cell crises in phase 1/2 risto-cel studyBeam Therapeutics disclosed data from its phase 1/2 trial of risto-cel, a base-edited autologous cell therapy targeting the BCL11A gene in sickle cell disease. The trial enrolled 31 adults and ado…
- Ionis shares tumble over $1 billion as Tryngolza sales forecast cut in halfIonis Pharmaceuticals saw its market value drop by more than $1 billion after it lowered its sales outlook for Tryngolza, a triglyceride‑lowering therapy. The company now expects $50 million in sa…