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RGI-2001 (formerly ToleroVax)

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Company Regimmune Corp.
DescriptionLiposomal formulation of KRN7000, a synthetic derivative of alpha-galactosylceramide
Molecular Target Natural killer T cells
Mechanism of Action 
Therapeutic ModalitySmall molecule
Latest Stage of DevelopmentPhase I/II
Standard IndicationGraft-versus-host disease (GvHD)
Indication DetailsPrevent graft-versus-host disease (GvHD) following allogeneic hematopoietic stem cell transplantation (HSCT); Treat graft-versus-host disease (GvHD) associated with hematopoietic stem cell transplantation (HSCT)
Regulatory Designation

U.S. - Orphan Drug (Treat graft-versus-host disease (GvHD) associated with hematopoietic stem cell transplantation (HSCT))

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