Vanessa Pataia,  —

FDA Gives Fabry Gene Therapy, FLT190, Orphan Drug Status

The U.S. Food and Drug Administration (FDA) has designated FLT190, an investigational gene therapy for Fabry disease,  an orphan drug to support and speed its development and possible review, Freeline Therapeutics, its developer, announced.

Avrobio Pauses Patient Dosing in AVR-RD-01 Gene Therapy Trials Due to COVID-19

In response to the COVID-19 pandemic, Avrobio has temporarily paused enrollment and dosing of new patients in Phase 1 and 2 trials testing its investigational gene therapy AVR-RD-01 for the treatment of Fabry disease. However, identification of eligible patients will continue in Canada, Australia, and the U.S.