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- FDA approves Scholar Rock's muscle-targeted SMA therapy IsembyldThe Food and Drug Administration has approved a new medication for spinal muscular atrophy (SMA). The drug, apitegromab marketed as Isembyld, is the first muscle-targeted therapy for the disease.…
- Cellectis quits CAR-T and pivots to ‘in vivo’ gene editingLong a frontrunner in donor-derived cell therapy research, the French biotech cited a shifting landscape in changing course and choosing to offload its lead programs.BioPharma Dive · 46m ago↗ source