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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 abandons CAR-T programs, shifts focus to in vivo gene editingCellectis announced it will discontinue its donor-derived CAR‑T programs and redirect its efforts toward in vivo gene‑editing medicines. The company said the commercial and clinical landscape for…