May 12, 2026 —
ModeX Therapeutics, an OPKO Health company, is set to present preclinical data at the American Society of Gene & Cell Therapy Annual Meeting in Boston showing in vivo generation of CAR-T cells and B-cell depletion using its investigational candidate MDX3001. The data include findings from humanized mouse and non-human primate models, supporting the potential of ModeX’s targeted gene delivery platform for immune cell engineering inside the body.
MDX3001 uses a CD3xCD28 antibody-conjugated lipid nanoparticle/mRNA platform designed to deliver genetic instructions directly to T cells in vivo. By combining CD3 targeting with CD28 costimulatory engagement, the platform aims to both transfect and activate T cells, enabling the generation of CAR-T cells without the need for traditional ex vivo cell collection, engineering, expansion, and reinfusion.
In the reported preclinical studies, MDX3001 demonstrated B-cell depletion in blood and lymphoid tissues, including the spleen, bone marrow, and lymph nodes. These findings suggest that the approach can generate functional immune activity across key tissue compartments. The mRNA-based design also offers potential advantages, including repeat dosing and avoidance of pre-conditioning chemotherapy, which is commonly required in conventional CAR-T treatment regimens.
ModeX described the platform as a simple and versatile approach to immune cell gene delivery. According to the company, its proprietary technology could support direct CAR-T generation in patients and may have applications across autoimmune diseases, oncology, and other immune-mediated conditions.
The presentation highlights a broader shift in cell therapy development toward in vivo immune engineering, where targeted delivery systems such as antibody-conjugated LNPs may help simplify manufacturing, reduce treatment complexity, and expand access beyond traditional autologous CAR-T models. While the data remain preclinical, MDX3001 represents an important program to watch as the field explores whether mRNA-LNP platforms can safely and effectively generate therapeutic immune cells directly inside the body.
