Hemophilia A (HA) is a rare genetic bleeding disorder that is caused by a lack of clotting factor VIII (FVIII). FVIII replacement is a standard of care treatment of HA, but, the formation of anti-FVIII antibody (a.k.a., “FVIII inhibitor”) is often problematic in the FVIII-replaced HA patients. FVIII Immune Tolerance Induction (ITI) is currently the only treatment option, which costs an average of $1M annually per patient. Thirty percent (30%) of the patients with ITI still fail to control the FVIII Anti-Drug Antibody (ADA), rendering the treatment ineffective.
Treg therapy has been highlighted as an ideal approach in autoimmune diseases and autoimmune disorders such as an anti-drug antibody formation. However, the technical hurdles to manufacture the FVIII-specific immune cell therapies have delayed the development of immune cell therapies for ADA in hemophilia A.
TeraImmune has dedicated to the development of Treg therapy specific to HA (Pipeline TI-168). By combining the patented Treg culture method (TREGableTM) and FVIII-specific TCR, our team has successfully demonstrated the therapeutic concept of FVIII TCR-Treg therapy in controlling of FVIII ADA in a hemophilic animal model. To translate this success to the clinic, TeraImmune has been continuing development towards the first-in-human (FIH) clinical trial in HA patients with anti-FVIII ADA.