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- 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…
- FDA's new rare-disease framework faces first test with Capricor's Duchenne therapy decisionThe FDA announced a new "fit-for-purpose" rare disease framework that aims to evaluate therapies based on patient need and realistically attainable evidence. The first real test of the approach wi…BioSpace · 4h agoRegulatoryNeutral