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.

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.

Experts in drug development, clinical care and digital therapeutics discuss gaps in the treatment landscape for Alzheimer’s disease, as well as opportunities for prevention and innovation in care delivery.   

The company, which has treatments in the clinic for CLN2 Batten disease and APOE4-associated Alzheimer’s disease, is developing AAV-mediated gene therapies designed for safer outcomes.