Demonstrating Feasibility of Producing a Highly Purified and Concentrated Stock Solution of Retrovirus Like Particles for Biopharmaceutical Process Development Viral Clearance Studies

PROJECT SUMMARY ABSTRACT MockV SolutionsIncMockVis an early stage company creating viral surrogate tools that solve the unmet needs of biopharmaceutical process development scientists as they develop manufacturing techniques to purify their therapeutics while removing or inactivating virusa contaminant of great concern during the production of biopharmaceuticalsIn factmammalian cell lines typically used for biopharmaceutical production endogenously produce retrovirus like particlesRVLP sAlthough non infectiousthe potential for reversion has caused global regulators to approve mammalian derived biopharmaceutics for clinical or commercial use only after their manufacturing processes demonstrates sufficient retroviral clearance capabilitiesThis is accomplished through small scalespiking studieswhereby model virusesexXenotropic Murine Leukemia VirusXMuLVare artificially introduced into biopharmaceutical material and removed or inactivated by purification techniquesThese studies require specialized Biological Safety Level laboratoriesBSLand trained personnel resulting in costs that can soar well above $Due to these hurdlesmost companies delay assessmentsthereby increasing the risk of a validation failureValidation failures lead to cost over runs and delayed regulatory approval which can cost a company valuable revenue patent life and cost patients timely access to therapiesMockV is developing a BSLcompatiblelow cost retrovirus clearance prediction kit based on a purified and concentrated stock solution of noninfectious RVLP s Utilized during manufacturing process developmenttheRVLP Kitwould provide scientists with a unique tool to generatereal timedata on retroviral clearanceWith aQuality by Designapproachthese scientists could confidentially optimize their manufacturing process to clear retrovirus and determine the efficacy of process steps before investing significant resources in costly validation studiesThe time and resources saved by the RVLP Kit would translate into cheaper $ gram manufacturing costs and increase the likelihood of passing regulatory hurdlesViral clearance is an international regulatory requirement for themammalian cell derived BP in worldwide developmentWith the projected cost of a RVLP Kit to be $we estimate an annual market value of$M yearTo set us on the path toward commercializationwe propose to first test the feasibility of economically producing a highly purified and concentrated stock solution of RVLPAfter successful completion of this Phase I feasibility study we will conduct extensive clearance testingcomparing RVLP clearance to live XMuLV clearance through variety of purification techniqueschromatography and nanofiltrationIn this Phase I SBIR studyMockV Solutions plans to test the feasibility of producing a highly purified and concentrated stock solution of non infectious Retrovirus Like ParticlesRVLPThis stock solution would serve as the centerpiece of a commercial Kitenabling scientists to develop more efficient biopharmaceutical manufacturing processes and predict viral clearance validation failures prior to expensive regulatory testingThese benefits will reduce the $ gram costs of biopharmaceuticals and ensure the timely regulatory approvals of life altering therapies