The first in vivo gene therapy product developed in China for hemophilia B granted ODD by FDA 

（Eastern Standard Time, December 14, 2021），VGB-R04 (an Adeno-Associated Viral Vector Delivering Human FIX Padua gene), the first drug candidate independently developed by Shanghai Vitalgen BioPharma Co., Ltd., has been granted Orphan Drug Designation (ODD) by the Office of Orphan Drug Product Development of FDA in the United States. 

 This is the first in vivo gene therapy product developed in China for hemophilia B to obtain orphan drug designation from FDA.