KRN7000, a synthetic derivative of alpha-galactosylceramide, is a small molecule that potently activates natural killer T (NKT) cells. RGI-2001 is a proprietary liposomal formulation of KRN7000. RGI-2001 has shown to induce antigen-specific immune suppression by inducing regulatory T cells (Tregs). Tregs are believed to play a central role in inducing and maintaining immune tolerance.
RGI-2001 is in late preclinical development and REGiMMUNE plans to file an Investigational New Drug (IND) for the prevention of acute GvHD associated with bone marrow transplantation later this year.