FDA hold for gene therapy program in Fabry disease

Genetic medicines company 4D Molecular Therapeutics (Nasdaq: FDMT) has announced that its Fabry disease gene therapy program has been placed on clinical hold by the US Food and Drug Administration (FDA).

There are currently two clinical trials, one in the USA and one in Taiwan and Australia, evaluating a single intravenous administration of 4D-310 in classic or late-onset Fabry disease.

Last month, the Californian company reported three cases of atypical hemolytic uremic syndrome (aHUS) in the first six patients enrolled in the program.