ONEVAX, LLC — Department of Health and Human Services SBIR Phase I: NIAID

Description

Immune mediated diseases such as typediabetesT Dmultiple sclerosisrheumatoid arthritisand systemic lupus erythematosis are reaching epidemic proportions in the UST D results from abreakdown of self tolerance that is characterized by immune cell mediated destruction of theinsulin producingcells in the pancreasUltimatelyglucose metabolism is interrupted resultingin the development of life threatening complications such as heart disease and renal failureT Daffects an estimatedmillion Americanswith more thannew patients diagnosed annuallyresulting in roughly $B in health care costs in the US each yearClearlythe cost associatedwith treatment and care of T D in the US is significantboth to society and the individualand anovel therapy is desperately needed to thwart thisaberrant autoimmune activityThis is a needthat OnevaxLLC is committed to fulfillingThe biomedical research community has sought better ways to induce specific immune tolerance fornearlyyearsClinical intervention trials using immunomodulatory agentse ganti CDhavefailed to meet clinical endpointsdespite positive results in phase I II trialsMoreovertraditional vaccine strategies providing auto antigen or peptides alone failed to adequately blockongoing beta cell immunityThusa new treatment strategy that is both potent and durable isrequired to effectively halt the ongoing attack in T DTherapeutic vaccination approaches for T Dutilizing dendritic cellsDCshold promise to correct antigen specific autoimmune responsesbutcurrent strategies involving exogenous manipulation of DCs are unstableinefficient and expensiveOneVax has developed a novel tolerogenic vaccine approach using FDA approved and GMP compatiblecomponents to administer immuno modulatory factors in vivo to phagocytic DCsthe most efficientantigen presenting cell type of the body and key regulator of the immune systemOneVax s therapyis minimally invasiverequires no costly cell isolation and storageanddue to its stablecompositionhas an extended shelf lifesimplifying manufacturing and shippingPreliminary datastrongly suggests that this biomaterial basedmicroparticle vaccine system prototype holds greatpromise for correcting autoimmune responses in T DThe objective of this Phase I proposal is tooptimize the formulation of our vaccine system from a four microparticle formulation to a minimizedtwo particle system allowing for efficient manufacturing and safety validation of the vaccineformulation downstream for eventual use in human clinical trialsMore specificallythe sizingagglomeration statusencapsulation efficiencyand release kinetics of the new particle vaccinesystem will be assessedas will the capacity for the new system to induce DC tolerization in vitroand prevent diabetic onset in clinically relevant animal models in vivoMost importantlystrategic collaborations between OneVax and the Sid Martin Biotechnology Incubatorthe BiomedicalEngineering Departmentthe College of Medicineand the Diabetes Institute at the University ofFlorida boost the capability of OneVax to complete the desired goals TypediabetesT Dis an autoimmune disease that carries a personal health burden that extendsto a tremendous socioeconomic impact in the USTherapeutic vaccination approaches for T D holdpromise to correct these antigen specific autoimmune responsesWe seek to develop an easilyinjectablepolymeric biomaterial based microparticle vaccine capable of targeted delivery ofimmuno modulatory agents to immune cellsOur goal is to create a vaccine product that will reversethe autoimmune process that drives T Dwhile also limiting off target effects that result fromsystemic administration of immunosuppressive drugs