FDA's Double-Edged Sword: New Hope for Rare Diseases, New Scrutiny for Chinese Trials

The FDA greenlit a first-of-its-kind gene therapy for a metabolic disorder that traps patients in a relentless cycle of raw cornstarch consumption. Pariglasgene brecaparvovec (Genglycos) cuts daily cornstarch needs by 41% for people with glycogen storage disease type Ia, a condition affecting roughly 1,500 to 2,500 Americans. The one-time adeno-associated virus vector delivers a functional gene to the liver, targeting the root cause rather than just managing...