Swell of activity suggests the blood-brain barrier could become a drug gateway

Rocket resumes its Danon disease gene therapy trial, but the field still needs better ways to balance inflammatory and immunosuppressive risks

Plus: Novartis working with Flagship-backed Profound in CV area, and more

Danon patient dies after treatment with AAV9 gene therapy and novel immune suppression regimen 

Structured deal could double the payout to Regulus’ shareholders if microRNA therapy, headed for Phase III testing in ADPKD, wins U.S. approval

CEO Ingram sees “the one significant strategic transaction” to transform Sarepta’s future, while Arrowhead preps for first-ever launch

Plus: Readouts from Avidity, Apellis/Sobi, Levicept and Merck

Plus: New capital for CytoReason, Truvian and Renalys

April opinions also include earlier-line use of J&J’s Rybrevant and BMS’s Opdivo

March decisions also include label extensions for UCB’s Bimzelx and Astellas’ Xtandi. Takeda pulls an application for Adcetris