The US Food and Drug Administration has given approval for MVX-220, an investigational gene therapy for Angelman syndrome, to enter a first-in-human clinical trial.
Angelman syndrome is a rare genetic disorder that affects brain development. It is caused by the loss of function of the UBE3A gene in neurons. There are currently no approved treatments.
MVX-220 was developed at the University of Pennsylvania to deliver the human UBE3A gene – using an adeno-associated virus as a vector – to neurons of the brain with a single injection.
Please see Angelman Syndrome News for more information.