Rocket’s Danon restart puts spotlight on gene therapy field’s AAV redesign challenge

It’s been a year of setbacks and serious safety concerns for AAV gene therapies, but Rocket Pharmaceuticals Inc. (NASDAQ:RCKT) is resuming its Danon disease program with a proposed solution to one type of adverse event that had raised fresh safety fears.

The company is addressing capillary leakage syndrome with reduced dosage and a modified immunosuppression regimen. However, tackling the broader challenge of balancing the inflammatory and immunosuppressive risks of AAV gene therapies — both of which can be fatal — may require more fundamental vector redesign...