Establishing the POROS Intracellular Delivery System as a Platform for Cellular Therapeutics
建立 POROS 细胞内输送系统作为细胞治疗平台
基本信息
- 批准号:10495192
- 负责人:
- 金额:$ 118.02万
- 依托单位:
- 依托单位国家:美国
- 项目类别:
- 财政年份:2018
- 资助国家:美国
- 起止时间:2018-09-10 至 2024-08-31
- 项目状态:已结题
- 来源:
- 关键词:AcousticsAddressAllogenicAreaAutologousB lymphoid malignancyB-Lymphocyte SubsetsBiological SciencesBiophysicsBuffersCAR T cell therapyCD19 geneCD7 geneCRISPR/Cas technologyCell SurvivalCell TherapyCellsCellular MembraneCharacteristicsComplexConsumptionContractsDNADevelopmentDevice or Instrument DevelopmentDevicesElectrophoresisElectroporationEngineeringEventFDA approvedFibroblastsFrequenciesGenerationsGenesGenetic EngineeringGoalsHealthHumanHybridsImmuneJurkat CellsKnock-inLawsLegal patentMalignant NeoplasmsManufacturer NameMediatingMedical DeviceMethodsModificationOutcomePatientsPharmacologic SubstancePhasePhysiologic pulseProductionProteinsRNARecoveryRegenerative MedicineReporterReproducibilityResearchSalesSample SizeSmall Business Innovation Research GrantSterilitySystemT-Cell DevelopmentT-Cell ReceptorT-LymphocyteTechnologyTherapeuticTherapy Clinical TrialsTimeTransfectionValidationbasecancer therapycase-by-case basiscell injurycell typecellular engineeringchimeric antigen receptor T cellscostcost effectivedesignelectric fieldexperimental studyfightinggenome editinginstrumentinterestmacromoleculemanufacturing processneoplastic cellnew technologynext generationnovelnovel strategiesnucleasenucleic acid deliveryoperationparallelizationpersonalized medicineprogramsprototyperesearch and developmenttwo-dimensionaluptake
项目摘要
Abstract
CAR-T therapy is a rapidly emerging therapy for the treatment of cancer, with two companies receiving FDA
approval for their first CAR-T therapies in 2017. These therapies use immune cells called T cells, either from
the patient themselves, or a healthy donor, and genetically engineer them to kill the tumor cells. The genetic
engineering involves direct delivery of nucleic acids to these cells, which is challenging as current delivery
methods either lack efficiency, are very damaging to cells, or both. This engineering is performed on a case-
by-case basis and the methods are costly and time-consuming. Next generation CAR-T therapies will include
more complex genetic engineering, with two or more editing events required. This is currently being done in a
sequential fashion, which increases the time-to-patient of the final product. In order to make CAR-T therapies
available to more patients in a cost-effective, safe and timely manner, new approaches need to be developed.
Current research is addressing this need by developing new technologies for cell treatment that allow faster
recovery and expansion time of the treated cells, allow delivery of multiple molecules in one shot and that can
be integrated into a closed, manufacturing-ready system. Another focus of research is engineering healthy
donor T cells to be used in CAR-T therapy, which would allow for an off-the-shelf cellular therapeutic. For this
type of product to be realized, high capacity treatment methods need to be developed capable of treating >
1010 cells while also maintaining high cell viability and delivery efficiency.
OpenCell Technologies has developed a proprietary technology, POROS, to deliver macromolecules such as
DNA, RNA and protein to a wide variety of cell types. POROS uses an acoustic drive to push cells through an
array of nozzles one cell at a time, thus creating shear force that porates the cells. In this SBIR program,
OpenCell is expanding the capabilities of its POROS platform by developing higher capacity POROS devices.
Approach: In this Phase II project, OpenCell will develop a high capacity version of POROS, capable of
treating a larger number of cells to enable CAR-T development. We will develop a prototype POROS device,
capable of treating 1-5 × 108 cells in under 10 minutes, and demonstrate gene editing in T cells as a proof-of-
concept experiment. The POROS device development will pave the way for development of a large, all-in-one,
GMP-compliant POROS-giga device, for manufacturing of off-the-shelf cellular therapeutics. Additionally, the
POROS unit developed in this Phase II project will also be a salable unit of its own with anticipated applications
in research and development, personalized medicine and small batch production of cells for autologous cell
therapies and for clinical trials.
摘要
项目成果
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Michael M Binkley其他文献
Michael M Binkley的其他文献
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{{ truncateString('Michael M Binkley', 18)}}的其他基金
Establishing the POROS Intracellular Delivery System as a Platform for Cellular Therapeutics
建立 POROS 细胞内输送系统作为细胞治疗平台
- 批准号:
10257954 - 财政年份:2018
- 资助金额:
$ 118.02万 - 项目类别:
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