R&D Regulatory setback or scientific signal? The case for human-... Exploring a systemic challenge in translating biological promise into reliable human efficacy and safety.
News Dismay as FDA blocks Regenxbio's Hunter syndrome therapy The FDA said it needs more information before it can approve Regenxbio's gene therapy for Hunter syndrome, in a devastating decision for patients.
Oncology Astellas’ banner year and focused R&D strategy, with Adam Pe... At JPM2026, Astellas' chief strategy officer Adam Pearson walked pharmaphorum through the company’s four-pillared R&D strategy.
News Sarepta rises after FDA adds warning label to Elevidys Uncertainty ends Sarepta's Duchenne gene therapy Elevidys ends after an FDA label update, with the company's shares on the up despite restrictions.
News Trial sets up filing for enGene bladder cancer gene therapy enGene is hoping to file its gene for a form of bladder cancer before the end of 2026, based on phase 2 data revealing a 62% complete response rate.
News uniQure poleaxed as FDA blocks Huntington's therapy uniQure has revealed that the FDA has said it is not prepared to review its Huntington's gene therapy in what seems to be a change in policy.
Patients LSX2026: On promise and patience, with Hans Schambye At LSX World Congress 2026 in Lisbon, Portugal, web editor Nicole Raleigh spoke with Hans Schambye, CEO of BOOST Pharma.
Sales & Marketing Sponsored Using AI-driven synthetic personas to take your insights fur... As AI continues to reshape the healthcare landscape, pharma teams are beginning to leverage synthetic personas.
