The Cranbury, New Jerseybased biotech announced FDA acceptance of its Biologics License Application for RP-L201 (marnetegragene autotemcel), an investigational lentiviral vector (LV)-based gene therapy for LAD-I, a rare, genetic immune disorder that predisposes patients to frequent and often fatal infections
Similarly, cagrilintide is being developed by Novo Nordisk , primarily as part of the CagriSema combination, with regulatory submissions anticipated once Phase 3 data are fully analysed and published
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When used appropriately, medication may help reduce symptoms, improve daily functioning, and make it easier to benefit from therapy and other coping strategies
Fatty acid conjugation extends the half-life to ~7 days, enabling weekly dosing in research protocols