271: Racing for gene therapy, a pioneering approval, & startups in the lurch

Approval of landmark gene therapy for Duchenne muscular dystrophy initiates a race against time for children to receive treatment before their sixth birthday. Biotech startups face financial challenges. FDA approves first medicine for rare bone disease despite not meeting primary endpoint. Approval and pricing of medicine for rare disease costing $624,000 per year. Gene therapy approval for Dushan Muscular Dystrophy sparks race for treatment. Age restrictions impact access to approved drug for Duchenne muscular dystrophy. Shifts at Silicon Valley Bank and HSBC impacting startup banking.