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Nature Biotechnology 26, 1057–1059 (1 October 2008) | doi:10.1038/nbt1008-1057

Ark floats gene therapy's boat, for now

Randy Osborne

In August, gene therapy's turbulent ride through the clinical rapids took a new twist as Ark Therapeutics released positive top-line results from a phase 3 trial of its adenoviral gene therapy Cerepro (sitimagene ceradenovec) for malignant brain tumors. Although the news boosted the London-based firm's shares, the course to market authorization and registration remains strewn with uncertainty—as Introgen, of Austin, Texas, found, to its cost, when the US Food and Drug Administration (FDA) recently refused its Biologics License Application (BLA) for Advexin (contusugene ladenovec), an orphan-designated adenoviral gene therapy for treating head and neck cancer and Li-Fraumeni syndrome.