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A Microscale Platform for Augmentation of Cell Transduction (MPACT) to Enhance Ex Vivo Gene Transfer

Award Number: R43HL146045
ORGANIZATION: NATIONAL HEART, LUNG, & BLOOD INSTITUTE
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 05/01/2019
PERIOD OF PERFORMANCE END DATE: 10/31/2021

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A Microscale Platform for Augmentation of Cell Transduction (MPACT) to Enhance Ex Vivo Gene Transfer - PROJECT SUMMARY/ABSTRACT Recent advances in clinical gene therapy highlight the need for improved lentiviral vector (LV) and adeno- associated viral (AAV) vector manufacture and utilization efficiencies for enhanced therapeutics outcomes. Expression Therapeutics in collaboration with Emory University are developing a microfluidic (µFluidic), technology-based solution that overcomes the mass-transport and diffusion limitations of current transduction platforms to enhance ex vivo vector gene transfer kinetics and efficiency. This novel ex vivo transduction platform is flexible in design, scalable, and compatible with standard cell culture transduction reagents and vector preparations as it mechanistically relies solely on physical principles. Using hematopoietic cell lines, primary human T-cells, and primary hematopoietic stem/progenitor cells, we have demonstrated that µFluidic transduction occurs up to 5-fold faster and requires ~1/20th of LV needed in conventional clinical transduction protocols. In vivo application of µFluidics using hematopoietic stem/progenitor cells of C57BL/6J hemophilia A mice transduced with factor VIII-encoding LV and transplanted into hemophilic donors demonstrated that LV usage and transduction time is significantly reduced with our platform, without loss of hematopoietic stem cell engraftment potential. The milestones proposed for the current phase I SBIR application are to 1) design and test microfluidic devices scaled-up to process > 108 target cell for genetically-modified cell product manufacture validation at clinically-relevant scales, 2) validate these devices using clinically-relevant primary human cell targets (CD34+ cells and CD3+ T cells), and 3) explore the opportunity for application of the microfluidic transduction devices to ex vivo AAV gene transfer, which is being pursued clinically for ex vivo gene-editing and transient gene expression applications.


Issue Date FY	Funding FY	Legal Entity Name	Legal Entity Address	Legal Entity City	Legal Entity State	Legal Entity Zip Code	Legal Entity COUNTY	Legal Entity COUNTRY	Assistance Listing	Award Code	Budget Year	Action Date	Action Type	Action Amount

Issue Date FY: 2022 ( Subtotal = $0 )
2022	2019	EXPRESSION THERAPEUTICS LLC	1860 MONTREAL RD	TUCKER	GA	30084	DEKALB	USA	Blood Diseases and Resources Research	000	1	4/26/2022	NEW	$0
														Subtotal = $0

Issue Date FY: 2021 ( Subtotal = $0 )
2021	2019	EXPRESSION THERAPEUTICS LLC	1860 MONTREAL RD	TUCKER	GA	30084	DEKALB	USA	Blood Diseases and Resources Research	000	1	12/1/2020	NEW	$0
														Subtotal = $0

Issue Date FY: 2019 ( Subtotal = $224,999 )
2019	2019	EXPRESSION THERAPEUTICS LLC	1860 MONTREAL RD	TUCKER	GA	30084	DEKALB	USA	Lung Diseases Research	000	1	4/18/2019	NEW	$224,999
														Subtotal = $224,999

Grand Total All Awards = $224,999

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A Microscale Platform for Augmentation of Cell Transduction (MPACT) to Enhance Ex Vivo Gene Transfer

Award Number: R43HL146045

ORGANIZATION: NATIONAL HEART, LUNG, & BLOOD INSTITUTE

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)

PERIOD OF PERFORMANCE START DATE: 05/01/2019

PERIOD OF PERFORMANCE END DATE: 10/31/2021

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