Ionis’ ION582 Receives the US FDA’s Breakthrough Therapy Designation to Treat Angelman Syndrome

Shots:The US FDA has granted BTD to ION582 (UBE3A protein expression stimulant) for the treatment of Angelman syndromeDesignation was based on P-I/II (HALOS) trial assessing ION582 in pts with Angelman syndrome, which showed improvements across all functional domains incl. communication, cognition & motor function; safety was favorableAdditionally, ION582 is being investigated in…