The U.S. FDA has granted Orphan Drug Designation to DTX101 for the treatment of hemophilia B.   Hemophilia B is a rare X-linked genetic disorder caused by mutations of the factor IX gene, resulting in missing or defective factor IX and consequently leading to increased propensity for bleeding.  DTX101 is an adeno-associated virus vector encoding the human factor IX gene and currently under development by Dimension Therapeutics, Inc., a biotechnology company based in Cambridge, MA.  It is designed to deliver Factor IX gene expression for patients with hemophilia B and according to the company’s website, DTX101 is expected to enter a multi-center phase I/II study at the end of this year.

 

Original Article:

http://dimensiontx.com/pressrelease/dimension-therapeutics-announces-fda-acceptance-of-investigational-new-drug-application-and-orphan-drug-designation-for-lead-candidate-dtx101-in-patients-with-hemophilia-b/