RGX has developed an innovative suite of therapeutic product candidates for neurodegenerative disease. Our lead candidate has demonstrated commercial potential in several animal models of Alzheimer's and is supported by significant data package supporting a disease altering mechanism, providing the Company with significant proof of concept efficacy, and coinciding with the mechanism a promising safety profile.

  RGX-100’s most recent in-vitro and in-vivo experiments demonstrate significant specificity (and thus also safety) through causing a drastic reduction in APP processing, and subsequently a reduction in the amounts of circulating A-beta (a key component of neuronal cell death), a reduced toxicity to circulating A-beta, as well as significant reductions in plaque formation in animal models of Alzheimer’s disease. RGX-Tgx, our Alzheimer’s drug discovery animal model platform, has recently demonstrated evidence that it may become the recommended animal model in preclinical trials for the next generation of disease altering drugs in development for Alzheimer’s and/or dementia. Significantly, the majority, if not all, of the existing animal models currently being used in this field are lacking significant biological relevance to the human disease. Our models were created specifically to be more biologically relevant, and thus facilitating more rapid translation of drug candidates to the clinic. Collectively, our research efforts to date provide significant evidence of safety and efficacy of our product portfolio, and continue to build value in RemeGenix.