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Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) said September 15 that the FDA agreed to count three patients treated under the modified RP-A501 protocol toward the pivotal Phase 2 efficacy cohort of 12 male patients. Nine additional patients remain to be enrolled and treated, with dosing expected to finish by mid-2027. Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) is developing RP-A501 […]
Ultragenyx’s second gene therapy approval since August gives it another sellable voucher. Elsewhere, Roche declared success in a blood cancer trial and a cell therapy maker is cutting costs.
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