Electroporation-Mediated Gene Therapy with IFN-b for MS

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DESCRIPTION (provided by applicant): Ichor proposes to assess the basic feasibility of using its proprietary TriGrid electroporation (EP) system for in vivo intramuscular delivery of a plasmid encoding for interteron-beta (IFNb) into normal mice. This approach aims to improve the current route of administration of IFNb, an FDA approved recombinant protein drug for relapse-remitting multiple sclerosis. This improved administration method should reduce the number of treatments necessary for efficacy, reducing the cost for this lifetime therapy, and increasing patient compliance. A comprehensive-dose analysis of the magnitude and duration of lFNb expression from EP-mediated intramuscular delivery will be performed to confirm that this approach results in sufficient levels of protein production. Considering that current recombinant lFNb therapy is associated with minor toxicities, these studies also aim to identify and characterize toxicities associated with both the administration of EP and IFNb production from muscle tissue. The management of these toxicities with controlled dosing using TriGrid electroporation or a gene regulation system will be assessed, since current management of recombinant IFNb-induced toxicities entails control of protein dosing regimens. These findings will provide guidance for future Phase II studies in large animal disease models, and perhaps humans.