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- Novartis' Zolgensma linked to tumors as FDA probes gene therapy and orders GLP-1 eye risk warningsNovartis confirmed that nine patients who received its Zolgensma gene therapy for spinal muscular atrophy have developed tumors, prompting the FDA to open an investigation into the approved treatme…
- Beam Therapeutics' base-edited risto-cel shows 12-month pain-crisis freedom in sickle cell trial, paving way for FDA filingBeam Therapeutics reported that its base-edited cell therapy, risto-cel, achieved at least 12 consecutive months without severe vaso-occlusive crises in patients with sickle cell disease during a m…BioSpace · 1d agoPositive readoutPositive