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Rapid Non-Viral Platform for Generation of Genetically Modified T Cells for Therapy

Award Information
Agency: Department of Health and Human Services
Branch: National Institutes of Health
Contract: 1R44CA233143-01
Agency Tracking Number: R44CA233143
Amount: $218,588.00
Phase: Phase I
Program: SBIR
Solicitation Topic Code: 100
Solicitation Number: PA17-302
Solicitation Year: 2017
Award Year: 2018
Award Start Date (Proposal Award Date): 2018-08-06
Award End Date (Contract End Date): 2019-05-05
Small Business Information
Saint Paul, MN 55104-4179
United States
DUNS: 079960066
HUBZone Owned: No
Woman Owned: No
Socially and Economically Disadvantaged: No
Principal Investigator
 (612) 656-4576
Business Contact
Phone: (612) 309-7653
Research Institution

Cancer immunotherapyparticularly genetically engineered adoptive T cell transferhas shown great potential
for the treatment of cancer patientsThe use of T cells engineered to express a specific T cell receptorTCRsor chimeric antigen receptorCARsto treat cancer has generated durable cures for many cancer patients and
has resulted in the first FDA approved CAR T therapy to treat childhood acute lymphoblastic leukemia inMost gene therapies rely on viral methods to genetically modify human primary cellsHoweverviral delivery
method is expensivepoorly reproducible and associated with several safety concerns including insertion in or
near genes that may cause malignancy and generation of replication competent virusThusnon viral DNA
delivery methodssuch as Sleeping Beauty and piggyBachave been employed to generate CAR T cellsAlthough these non viral delivery methods have the advantage of lower costimmunogenicityand regulatory
considerationsthey have been limited by their low transposition efficiency in primary human hematopoietic cellsIn this applicationwe propose to rationally optimize a recently discovered transposonTcBuster to deliver CARs
to T cellsTo this endwe further enhance our already very active hyperactive mutants of the TcBuster
transposase and optimize the delivery of the transposon into cellsFollowing optimization of the TcBuster
transposon systemwe will combine these improvements with our proprietary methods to transfect T cells
efficiently and safelyand test the immunotherapeutic effectiveness of TcBuster delivered CAR into T cellsThe
successful completion of this project will result in the comprehensive methods to produce CAR T cells delivered
by TcBuster which we will license to pharmaceutical companies to produce highly efficient CAR T
immunotherapyMore broadlythese methods could be expanded beyond immunotherapeutic cancer
applications to various infectious diseases in which gene delivery by TcBuster in T cells could be advantageous

* Information listed above is at the time of submission. *

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