FVIII-CAART

A New Supporting Role for CAAR Technology

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 FVIII 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.

Other

Therapeutic Area Indication Program Discovery Preclinical Phase 1 Phase 2/3
Hematology Hemophilia A with FVIII Alloantibodies FVIII-CAART
Discovery in progress
Preclinical not started
Phase 1 not started
Phase 2/3 not started

1 In our discovery stage, we perform epitope mapping and optimize CAAR construct and design.

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.

Posters & Publications

Learn more about our scientific research through posters at leading conferences and publications in peer-reviewed journals.

Examine Our Findings

Presentations

These posters and presentations hit the right notes.