Fatality in Rocket Pharma trial renews scrutiny of AAV-immune interactions

A patient death in the Phase II study of RP-A501 from Rocket Pharma is the latest reminder that AAV gene therapies can trigger complex and sometimes catastrophic inflammatory consequences. 

The company reported that a patient with Danon disease — a rare, X-linked disorder caused by mutations in the lysosomal LAMP2 gene — died from a systemic infection after experiencing capillary leakage syndrome. FDA has placed a clinical hold on the study. ...