Model FVIII-CAART - Discovery-Stage Candidate for Hemophilia
In addition to our work on autoimmune disease treatments, we are investigating the viability of CAART as an adjunct in cases where the immune system produces antibodies against current therapies (rather than against the patient’s cells). We believe our approach has the potential to destroy those antibodies by ablating B cells, similar to the method we have used to combat autoimmune disease. FVIII-CAART is a discovery-stage candidate for Hemophilia A patients who have an anti-Factor VIII immune response. FVIII-CAART is designed to destroy the antibodies and B cells that neutralize the FVIII clotting factor – potentially restoring the effectiveness of FVIII infusions.
FVIII-CAART Harmonizes with Essential Therapies
FVIII-CAART is designed to improve the efficacy of FVIII replacement infusions in patients with Hemophilia A.
About Hemophilia
Hemophilia A is an X-linked bleeding disorder caused by a deficiency of functional FVIII, a critical factor in blood coagulation. It affects about 1 in 5,000 male births. Severe Hemophilia A, which accounts for about 60% of all cases, is marked by FVIII levels below 1% of normal, rendering these patients vulnerable to frequent spontaneous bleeds. Currently, Hemophilia A is treated with intravenous FVIII replacement. However, nearly a third of patients who receive this therapy develop neutralizing alloantibodies against the FVIII protein. To reverse alloantibody formation, patients are given repeated, high-dose infusions of FVIII, often at a high cost and with limited clinical success. We believe FVIII-CAART could effectively eliminate FVIII-neutralizing alloantibodies and the B cells that produce them, offering a more tolerable and durable treatment option for patients with Hemophilia A and FVIII alloantibodies.
FVIII CAAR Discovery
Discovery
Our FVIII-CAART program is currently in the discovery phase, as we identify additional pathogenic epitopes and construct a FVIII-CAART that includes additional FVIII domains. Given the large size of the FVIII protein, we are evaluating potential technologies to reduce the size of the final CAAR construct.