ViaCyte, Inc. — Department of Health and Human Services SBIR Phase II: 200

Description

Current diabetes therapies cannot prevent life threatening hypoglycemic eventsand chronic complications for hundreds of thousands of typediabetesT Dand typediabetesT DpatientsThese critical need patients represent a substantial investment opportunityby their urgent need for a new solution beyond lifesustaining exogenous insulin replacementnamely one that could provide long term and precise glucose controlSuch a robust and long acting cure is presently availableislet and pancreas transplantation demonstrates the proof of conceptPOCto replace beta cellinsulin producing cellfunction and eliminate dependency on insulin injectionsHowevertheir wide spread use is severely curtailed by limited organ supply and the requirement for chronic immunosuppressionIn contrastViaCyte s GMP human embryonic stem cellhESCline provides an unlimited supply of stem cells and their differentiated functional beta cells as a starting materialViaCyte s immune protection macro encapsulation device can potentially eliminate the need for chronic immunosuppression in critical need patientsIndeedas a first stepPEC ENCAP or hESC derived pancreatic progenitor cellStagecellin a macro encapsulation deviceproves that such combined technology is feasible in the clinicit is safe and well tolerated by patientsprogenitor cells can survive and differentiate to bona fide beta cells in a device in T D patientsYethESC derived functional beta cells in a macro devicewe hypothesizewill be a more efficacious cell therapy treatment option requiring a smaller therapeutic footprint per patientwill be less prone to off target differentiationand will provide a more rapid diabetes reliefOur goal is to advance the hESC derived functional beta cell in a macro device conceptdefined asVCtowards commercializationWe are uniquely positioned for high probability of future commercial successby our ability to test multiple GMP compliant hESC derived stages of advanced beta cell differentiationendocrine precursorStageimmature beta cellStagefunctional beta cellStagein two macro devicesmacro encapsulationdirect deliveryWe aim to provide POC information required to initiate IND enabling studiesby determining which specific combination of Stageorcell and macro device type is most appropriate for the eventual commercialization of the VCproduct for critical need patientsThe goal of Aimis to provide the scale up and cryopreservation capabilities to Stagesof differentiationensuring a stable cell supply for eventual INDenabling studiesclinical trialsThe goal of Aimis to lay the groundwork for in vivo efficacy data required by IND enabling studiesThe quarterly feedback cycle between characteristics obtained in parallel from Aimin vitro manufacturingand Aimgraft survivalin vivo functional analysiswill provide an unbiased platform for deciding which advanced beta cell stagemacro device combination is most appropriate for the IND enabling studies of VCViaCyte has proven competencies in the establishment of a safetytarget profile of a combined cell and device technology required for eventual IND success