RAFT-OS

RAFT-OS is an investigational gene therapy candidate designed for the treatment of osteosarcoma, with a particular focus on managing metastatic disease in the lungs. Developed by Raft Pharmaceuticals, the therapy utilizes a recombinant adeno-associated virus (rAAV) vector to deliver the gene encoding Fas Ligand (FasL) directly to tumor cells. FasL is a transmembrane protein that, upon binding to its cognate receptor Fas (CD95) on the surface of malignant cells, triggers the extrinsic apoptotic pathway, leading to programmed cell death. This approach aims to exploit the Fas/FasL signaling axis, which is frequently dysregulated in osteosarcoma, to selectively eliminate cancer cells while minimizing systemic toxicity. The development of RAFT-OS followed a comparative medicine model, showing significant efficacy in canine osteosarcoma trials before transitioning toward human clinical evaluation.

Other names

AAV-FasLRecombinant Adeno-associated virus-mediated Fas Ligand Therapy