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

Description

TypeDiabetesT Dresults from a breakdown of self tolerance that is characterized by immune cell mediated destruction of the insulin producingcells in the pancreasUltimatelyglucose metabolism is interrupted resulting in the development of life threatening complications such as heart disease and renal failureT D affects an estimatedmillion Americanswith more thannew patients diagnosed annuallyresulting in roughly $B in health care costs in the US each yearIt is thought that arrest of the autoimmune processes underlying this disease could avert the long term complications associated with the disease and perhaps even reverse the disease processgiven sufficient insulin producing cells remainClinical intervention trials using immunomodulatory agentse ganti CDhave failed to meet clinical endpointsdespite positive results in phase I II trialsand traditional vaccine strategies providing auto antigen or peptides alone failed to adequately block ongoing beta cell immunityThusa new treatment strategy that is both potent and durable is required to effectively halt the ongoing attack in T DThe immune system utilizes two important strategies to prevent the aberrant targeting and destruction of self tissuesthe production of regulatory T cellsTregsand the limitation of inflammatory responses to prevent collateral damageRecent studies have revealed that the intracellular protein suppressor of cytokine signalingSOCSplays a crucial role in regulating immune responses and in the survival and phenotypic stability of regulatory T cellsand that defects in its production and availability plays a critical role in the manifestation of a variety of auto immune diseasesDespite its obvious appeal as a potential therapyenthusiasm has been dampened due to the extremely limited stability of therapeutically administered SOCSin its pure formWe have managed to synthetically manufacture small peptide SOCSmimetics that possess the functional capacity of SOCSallowing for costand time efficient production of this potential therapeuticFurthermorewe have patented means of manufacturing injectable polymeric microparticlesMPsencapsulating a variety of immunomodulatory agents allowing for sustainedtunable delivery of those agents to treat T DThe objective of this Phase I proposal is to establish the feasibility of encapsulating our small peptide SOCSmimetics in MPsand to determine the efficacy of this MP system in the prevention of T D onset in an animal model of the diseaseThe results from these studies will allow us to proceed to Phase II of the SBIRin which studies will be conducted to satisfy the FDA requirements for the production of a clinicalgrade drug suitable for Human Phase I II clinical trials as a novel therapy for T DOur long term goal is to develop an easily injectable therapy capable of prevention and reversal of a wide variety of auto immune diseasesgreatly enhancing the potential for widespread useOur preliminary data strongly suggests that this therapy holds promise for correcting autoimmune responses in T DAdditionallyour strategic collaborations with the Diabetes Center of Excellence at the University of Florida and the internationally recognized Sid Martin Biotechnology Institute bolster our ability to complete the desired goals TypediabetesT Dis an autoimmune disease that carries a personal health burden that extends to a tremendous socioeconomic impact in the USand therapeutic approaches for T D focusing on restoring immune tolerance mechanisms hold huge promise to correct the autoimmune responseWe seek to combat the underlying autoimmune processes that drive T D by utilizing a biomaterial based delivery system to administer suppressor of cytokine signaling peptide mimetics designed to inhibit inflammatory cytokine responses and enhance regulatory T cell functionthereby restoring immune tolerance in a stable and robust manner