A novel biomaterial based therapy utilizing auto antigen and adjuvant for the amelioration of Type Diabetes

Immune mediated diseases such as type diabetes T D multiple sclerosis rheumatoid arthritis and systemic lupus erythematosis are reaching epidemic proportions in the US T D affects an estimated million Americans with more than new patients diagnosed annually resulting in roughly $ B in health care costs in the US each year T D is an autoimmune disease characterized by effector T cell mediated destruction of insulin producing cells which renders the patient unable to produce insulin and forces the patient to constantly monitor their glucose levels and manually administer insulin for the remainder of their life Ultimately glucose metabolism is interrupted resulting in the development of life threatening complications such as heart disease and renal failure Clearly the cost associated with treatment and care of T D in the US is significant both to society and the individual and a novel therapy is desperately needed to thwart this aberrant autoimmune activity This is a need that Onevax LLC is committed to fulfilling The biomedical research community has sought better ways to induce specific immune tolerance for nearly years Clinical intervention trials using immunomodulatory agents e g anti CD have failed to meet clinical endpoints despite positive results in phase I II trials Moreover traditional vaccine strategies providing auto antigen or peptides alone failed to adequately block ongoing beta cell immunity Thus a new treatment strategy that is both potent and durable is required to effectively halt the ongoing attack in T D Therapeutic vaccination approaches for T D utilizing dendritic cells DCs hold promise to correct antigen specific autoimmune responses but current strategies involving exogenous manipulation of DCs are unstable inefficient and expensive OneVax has developed a novel in vivo tolerogenic vaccine approach using FDA approved and GMP compatible components to administer immuno modulatory factors to phagocytic DCs the most efficient antigen presenting cell type of the body and key regulator of the immune system OneVax s therapy is minimally invasive requires no costly cell isolation and storage and due to its stable composition has an extended shelf life simplifying manufacturing and shipping Preliminary data strongly suggests that this biomaterial based microparticle vaccine system holds great promise for correcting autoimmune responses in T D The objective of this Phase I proposal is to complete the necessary preclinical studies to demonstrate the feasibility of this vaccine formulation for use in humans More specifically the biodistribution bioavailability toxicity and potentially harmful off target effects of the particle vaccine will be assessed Most importantly strategic collaborations between OneVax and the Biomedical Engineering Department the College of Medicine and the Diabetes Institute at the University of Florida boost the capability of OneVax to complete the desired goals Type diabetes T D is an autoimmune disease that carries a personal health burden that extends to a tremendous socioeconomic impact in the US Therapeutic vaccination approaches for T D hold promise to correct these antigen specific autoimmune responses We seek to develop an easily injectable polymeric biomaterial based microparticle vaccine capable of targeted delivery of immuno modulatory agents to immune cells Our goal is to create a vaccine product that will reverse the autoimmune process that drives T D while also limiting off target effects that result from systemic administration of immunosuppressive drugs