BBT-002

BBT-002 is a preclinical-stage engineered B-cell gene therapy being developed by Bespoke Biotherapeutics for the treatment of severe hemophilia A associated with spontaneous bleeding. The therapy utilizes Bespoke's proprietary NINJA (non-viral, large transgene, homology-mediated end-joining) CRISPR/Cas9 engineering platform to create autologous B cells that are genetically modified to produce and secrete functional Factor VIII (FVIII). Unlike traditional gene therapies that use viral vectors, this approach employs non-viral genome editing to insert the FVIII gene into patient-derived B cells, which then act as 'living drug factories' to continuously produce the missing clotting factor. The therapy is designed to provide long-term FVIII replacement for patients with severe hemophilia A, potentially reducing or eliminating the need for frequent factor infusions.

Other names

B-cell gene therapyFVIII-producing engineered B-cell