Purilogics, LLC — Department of Health and Human Services SBIR Phase I: 400

Description

Project Summary This SBIR Phase I project will address the need for a product that can dramatically increase the recovery and purification speed for viral vectors used in gene therapySeven thousand rare diseases that have few or no treatment options are affectingof the US populationAlthough gene therapies are promising for the treatment of many rare diseasesmost are at an early stage of development and face challenges for the largescale production of gene delivery vectorsTechnologies like the one proposed will contribute to improving human health by enabling the rapid and scalable purification of lentiviral vectorsLVswhich are among the fastest growing delivery vectors used in gene therapyConventional methods to purify LVs involve a large number of steps and achieve low overall yields ofThe goal of this SBIR Phase I project is to demonstrate the feasibility of developing affinity membrane chromatography columns that rapidly purify LVs from vector cell supernatant with high recovery and infectivity compared to conventional approachesProducts derived from this innovation will be first in marketdisposable membrane chromatography columns that can simplify the LV purification process and improve the overall yield toPurilogics expects these products will have a major impact on gene therapy development and commercialization by greatly improving the yield of this important type of delivery vectorThe aims of the Phase I study areito synthesize and characterize affinity membranes andto test prototype membrane columns for capture purification of viral vectorsIn Specific Aimwe will evaluate the roles played by synthesis conditions and membrane pore size on performance using in house screening experimentsWe will test the hypothesis that the unique combination of our proprietary ligand with high affinity for the envelope surface glycoprotein on LVsa high surface area support membraneand Purilogicsandaposproprietary membrane fabrication method will yield products with high LV binding capacityMild elution conditions will be developed that lead to high recovery of active LVsIn Specific AimPurilogics will collaborate with researchers at Clemson University to evaluate membrane performance for purification of LVs prepared using a commercial cell lineMultiple iterations of synthesis and performance characterization will improve membrane performanceIn Phase IIPurilogics plans toiestablish a scalable process to produce prototypes for comprehensive performance testingdetermining sterilization protocolsproduct shelf lifeand reusabilityandiiconduct field research with industrial partners who are potential customersMarket entry for the new column products will be sales to purification scientists and engineers in biopharmaceutical companiesThese individuals are end usersinfluencers and decision makersThe estimated total addressable market for prepackeddisposable capture step chromatography columns under development by Purilogics is expected to reach $M byPublic Health Relevance Statement Gene therapy is one of the most promising therapeutic modalities to treat and cure diseases with limited or no alternative treatment optionsLentiviral vectors are emerging as one of the favored gene delivery vehicles in clinical trialsThe goal of this SBIR Phase I project is to develop an affinity membrane chromatography product for the rapid purification and efficient recovery of lentiviral vectors from vector cell supernatantThese products will contribute to improving human health by enabling scalable production of the vectors that are needed for this emerging field of medicine