Industry Insights: The adjuvant access problem: barriers to broader vaccine development

Abstract

A cell therapy safety signal defined the month: Novartis paused eight trials of its experimental CAR-T therapy rap-cel across autoimmune and neurological indications after three deaths from immune effector cell-associated hemophagocytic syndrome, prompting Bristol Myers Squibb to pause a rival program as a precaution. On the regulatory front, Ultragenyx secured the first US FDA approval of a gene therapy for glycogen storage disease type Ia, amid an otherwise busy month of designations, a clinical hold, and multi-billion-dollar dealmaking in CAR-T and gene therapy.