Current accelerated approval provisions are the ‘sweet spot’ for balancing access and efficacy, but more attention needs to be paid by sponsors and the agency to working out regulatory pain points earlier in the development process, CBER director tells gene/cell therapy conference.

In exploratory FDA analyses, PRO data appeared more sensitive at detecting an exposure-toxicity relationship for an oral small molecule cancer drug than clinician-reported data; Project Optimus representative dispels industry concerns that FDA wants firms to find the 'mythical' optimal dose.

Vir’s George Scangos, Westlake Village BioPartners’ Sean Harper, Kronos Bio’s Norbert Bischofberger, Scorpion’s Axel Hoos and Eikon’s Roy Baynes discuss the experiences they bring and the challenges they have faced in the transition from large drug makers to small biotechs. 

The third annual listing of In Vivo’s ‘Rising Leaders’ includes entrepreneurs and innovators from across the world who represent the next wave of creativity in health care.