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A novel device and rescue solution for the improved post-thaw viability and function of human stem cells used in cell therapy.

Award Information
Agency: Department of Health and Human Services
Branch: National Institutes of Health
Contract: 1R43GM129851-01
Agency Tracking Number: R43GM129851
Amount: $307,412.00
Phase: Phase I
Program: SBIR
Solicitation Topic Code: 400
Solicitation Number: PA17-302
Solicitation Year: 2017
Award Year: 2018
Award Start Date (Proposal Award Date): 2018-08-03
Award End Date (Contract End Date): 2019-07-31
Small Business Information
Owego, NY 13827-1515
United States
DUNS: 150225337
HUBZone Owned: No
Woman Owned: No
Socially and Economically Disadvantaged: No
Principal Investigator
 (607) 427-3459
Business Contact
Phone: (607) 687-8701
Research Institution

There are overhuman stem cell trials currently underway worldwideMost of these trials
use adipose derived mesenchymal stem cellsadMSCswhile others use differentiated human
embryonic stem cellshESCsWhile human induced pluripotent stem cellsiPSCsare
currently in limited trials in select countriesthe future promise of these cells for treating disease
worldwide is indisputable and their increasing use in the drug discovery toxicity sciences is
assuredThus it is imperative to improve the bioprocessing and delivery of stem cells for patient
use through developing new stem cell dedicated instrumentsunique biomaterialsand
improved methods of cell cryopreservationNearly half of stem cells are cryopreserved and
thawed prior to infusion into the patientYet little attention has been paid to the post thaw
recoveryfunction and homing of these cells in patients that can be compromised as a
consequence of preservationCPSI Biotech personnel have worked in the stem cell
therapy cryopreservation arena for nearlyyears and have reported that delayed onset cell
death occurs up to several days post thaw in all cell types including stem cellsCurrentlymany
stem cell therapy companies thaw their cryopreserved cells immediately prior to infusion using a
cryopreservation solution that is a component of their FDA issued INDThe most common
method of thawing these cells is a standardnon programmablewater bath and little to no
emphasis is placed on delayed onset cell death that most assuredly occurs in the patient as a
consequence of the thawing process that is exacerbated by the physical stress to the cells due
to the infusion processCPSI Biotech will address this issue by developing two new devicesStemThaw and StemTransand one unique recovery reagentStemReviveIn Phase I CPSI s
prototype dry thaw systemSmartThawwill be tested as a dedicated stem cell thawing deviceStemThawthat uses stem cell specific thaw profiles that yield improved post thaw outcome
compared to standard methodsIn additiona post thaw dilution reagentStemRevivewill be
developed that will reduce the delayed onset cell death known to occurtohours postthawThe combination of these two complementary technologies should improve the viabilityfunction and engraftment of stem cells while at the same time limiting the chance of posttherapy rejectionPhasewill be dedicated to developing a unique deviceStemTransthat will
be designed for the improved shipping and storage of cryopreserved and hypothermically stored
stem cellsThe PhaseSpecific Aims are the followingDefine and develop unique cell type
matched thawing programs for StemThaw that improve the post thaw outcome of adMSCshESCs and iPSCsDefine the optimal formulation of StemRevive as an additive reagent to
thawed adMSCshESCs and iPSCs to prevent delayed onset cell death resulting in optimal
viabilityCombine StemThaw and StemRevive together to determine efficacyPhasespecific aims will includeDevelop and test unique thaw programs for StemThaw defined by
PhaseDevelop StemTrans for the improved distribution of cryopreserved stem cellsDetermine if the optimal StemThaw and StemRevive combination defined in Phaseresults in
improved homing and engraftment in a mouse SCID modelTest the combination of
StemTransStemThaw and StemRevive as a candidate platform for the improved shippingstoringthawing and recovery system that will yield improved viabilityfunction and engraftment
of stem cells for stem cell therapy thus limiting graft rejection and improving stem cell therapy
efficacy Of thestem trails currently ongoing worldwidenearly half use cells that are cryopreserved in
liquid nitrogen so that they can be shipped and stored prior to infusion into cell therapy patientsThis
Phaseproject proposes to develop a unique instrumentStemThawand a companion solutionStemRevivethat when used either separately or together should yield improved viability and function
of cryopreserved stem cells used for stem cell therapyThis will lead to improved health benefits and
outcome to stem cell patients

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

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