The U.S. Food and Drug Administration (FDA) has accepted CSL Behring’s Biologics License Application (BLA) for priority review of etranacogene dezaparvovec, an investigational gene therapy for hemophilia B.

Etranacogene dezaparvovec, also known as CSL222, consists of adeno-associated virus serotype 5 (AAV5), which function as a delivery vector carrying a gene cassette with the Padua variant of factor IX (FIX). AAV5 is a variant of the type of the adeno-associated virus vectors (AAVs) investigated in multiple ongoing gene therapy clinical trials.

The next generation of researchers are continuously inspiring and fueling NHF’s renewed focus on research. Dr. Olubusola (Bosula) Oluwole is one of the first-ever recipients of the new Jeanne Marie Lusher (JML) Diversity Fellowship.

NHF’s newest research fellowship program is named in honor of Dr. Jeanne Marie Lusher, a distinguished clinician and researcher whose career focused on inhibitor development in factor VIII patients.

Meet NHF's Education Specialist, Sonia Roger! As one of NHF's most tenured employees, Sonia is a vital part of the NHF community and loves giving back to families and patients. Learn more about her role at NHF!

Tell us a little bit about yourself and your role at NHF.

The issue of poor bone health represents a significant public health concern in the U.S. population, including for individuals with hemophilia who are at increased risk for low bone mineral density (BMD) and bone fractures.