FERROLOGIX INC — Department of Health and Human Services SBIR Phase I: 100

Description

Project Summary Gene modified autologous hematopoietic stem cell transplantation is a transformative approach for treating variety of blood cancers and immunodeficiency disordersHoweverthe current methods for therapy manufacture need substantial quantities of costly GMP grade viral vectors to deliver the nucleic acids for deriving therapeutic cellsThe high cost and limited supply of GMP grade vectors contributes to large expenses incurred by patients as well as long waiting lists which delay patient treatmentAdditionallyvector availability to clinical researchers developing new therapies is also reduced which slows clinical translationWhile other non vectorbased methods for nucleic acid delivery are availablethey have disadvantages in working with delicate stem cells such as low cell viability and highly delivery efficiency which limits their clinical applicabilityFerrologix proposes to develop a modular product that can achieve afold reduction in the quantity of vector necessary to produce a therapeutic cell batch compared to the current co culture methodsUtilizing arrays of ferromagnetic micropillarsmagnetically tagged target cells and viral vector can be concentrated to discrete points for highly controlled exposureBy confining the volume of cells and vectorprecision vector mediated nucleic acid delivery can be achievedIn this phase I submission we propose to develop a magnetic transduction platform that can significantly reduce per batch vector consumptionwhich can lead to more rapid clinical translation of new therapies and scaled patient treatment capabilities Project Narrative The goal of this project is to develop a magnetic based nucleic acid delivery platform which can reduce consumption viral vector for autologous stem cell therapiesThe limited supply and high cost of vector translates to significant cost and treatment delays for patients as well as impeding research for new treatmentsCurrent techniques and technologies either require large quantities of vector to manufacture the therapyare too harsh for use with stem cellsor have limited precisionOur proposed technology can reduce the required vector usage in a gently waytranslating to accelerated research and improved patient outcomes