4D Aligns with FDA to Lift Clinical Hold on Phase 1/2 Trial for 4D-310 for Fabry Disease Cardiomyopathy

 

• Initiated single non-human primate (NHP) safety study evaluating intravenous 4D-310 combined with rituximab/sirolimus (R/S) immunosuppressive regimen
• Amended INGLAXA protocol to minimize risk of atypical hemolytic uremic syndrome (aHUS) associated with intravenous (IV) AAV dosing, including addition of R/S immunosuppressive regimen
• 4D-310 combines a novel, targeted next generation AAV vector (C102) and GLA transgene for a single low dose IV delivery to cardiomyocytes
• Interim clinical data from INGLAXA expected to be presented in Q1 2024

https://www.biospace.com/article/releases/4dmt-gains-alignment-with-fda-on-plan-to-lift-clinical-hold-on-phase-1-2-inglaxa-clinical-trial-for-4d-310-for-fabry-disease-cardiomyopathy-/