Federal Project Grant Award Summary

Emory University received a $391,211 Project Grant from the National Heart, Lung, and Blood Institute (NHLBI) under the Cardiovascular Diseases Research program (CFDA 93.837), effective June 1, 2026 through May 31, 2029. The award supports research investigating alloimmune responses to hemophilia therapeutics, specifically addressing the mechanisms underlying anti-drug antibody development against coagulation factor VIII (FVIII) and factor IX (FIX) protein replacement therapies. The research aims to identify earlier immune response markers that may predict inhibitor development and response to immune tolerance induction (ITI), with particular focus on understanding B cell activating factor (BAFF) regulation and its role in preventing antibodies to FVIII and FIX in hemophilia patients.

This research directly addresses a critical clinical gap in hemophilia management, as approximately 30% of severe hemophilia A patients and 3-5% of severe hemophilia B patients develop neutralizing anti-factor antibodies that complicate treatment efficacy and limit access to gene therapy. The project deliverables include scientific discoveries regarding the pathogenesis of inhibitor development, characterization of biomarkers for predicting immunogenic responses, and potential therapeutic targets through anti-BAFF intervention strategies that could improve clinical outcomes for hemophilia patients and inform immune tolerance approaches.