Induced pluripotent stem cells (iPSCs) have become a commonly used tool to generate multiple cell types froma given individual patient. Reprogramming non-invasively harvested cells, such as dermal fibroblasts, intoiPSCs, allows investigators to generate any cell type from a patient with known genetic and clinical backgrounds.This method has been particularly powerful for cases in which a disease condition manifests in a tissue whereacquiring patient-derived primary cells is challenging, or there are genetic mutations. However, the workflowsassociated with reprogramming, CRISPR gene editing, and differentiating iPSCs are low-throughput, costly,time-consuming, and not supported by commercially available automated instrumentation. Customizedautomation systems have been published and generally require considerable capital investment, multiple typesof equipment, custom programming, and commitment to a single reprogramming method. There is an unmetneed in academia and industry for technology that can improve the efficiency and success rates of a)reprogramming, b) iPSC viability, and c) the ability to generate clonally derived CRISPR edited cell lines in anautomated fashion. The development of streamlined workflows on a single platform that can deliver higherefficiencies in iPSC reprogramming and cell line development is likely to dramatically enhance the utility andthroughput of the technology, thereby accelerating the use of iPSCs in personalized medicine and drugdiscovery. Cell Microsystems proposes the use of the CellRaft Technology, comprising the automated AIRSystem and CytoSort Array cell culture consumable as a cost-effective, automated, and gentle solution that cansolve many of the pain points that plague these critical workflows. In this Phase I program, we will developprotocols for the culture, reprogramming, and editing of iPSCs using our proprietary consumable andinstrumentation and demonstrate that our technology provides faster, more cost effective, and more efficientworkflows for iPSC cloning. Commercially, there is a tremendous potential market in laboratories across allindustries that perform these workflows, and our company has a proven successful track record in developingtools for the drug discovery market.
Public Health Relevance Statement: Project Narrative
Despite the advent of multiple sophisticated techniques for generating a clonal population of reprogrammed or
edited induced pluripotent stem cells (iPSCs), there remains a clear need for a more automated, rapid, and cost-
efficient means to identify, phenotypically characterize, isolate, and edit iPSC clones. The CellRaft AIR System
and CytoSort array consumables are uniquely suited for alleviating pain points in the CRISPR and iPSC
workflows such as low efficiency, low cell viability, and manual culture and isolation, to enable more efficient
and robust cell line development for drug and therapeutic discovery pipelines.
Project Terms: Sickle Cell Anemia ; Hb SS disease ; HbSS disease ; Hemoglobin S Disease ; Hemoglobin sickle cell disease ; Hemoglobin sickle cell disorder ; sickle cell disease ; sickle cell disorder ; sickle disease ; sicklemia ; Automation ; beta Thalassemia ; B-thalassemia ; p-Thalassemia ; β-thalassemia ; Capital ; Cell Culture Techniques ; cell culture ; Cell Line ; CellLine ; Strains Cell Lines ; cultured cell line ; Cell Separation ; Cell Isolation ; Cell Segregation ; Cell Separation Technology ; cell sorting ; Cell Survival ; Cell Viability ; Cells ; Cell Body ; Clinical Trials ; Clone Cells ; Disease ; Disorder ; Equipment ; Fibroblasts ; gene therapy ; DNA Therapy ; Gene Transfer Clinical ; Genetic Intervention ; gene-based therapy ; genetic therapy ; genomic therapy ; Genes ; Human ; Modern Man ; Industry ; instrumentation ; Investments ; Laboratories ; Maintenance ; Manuals ; Methods ; Methodology ; Pain ; Painful ; Patients ; Phenotype ; Plague ; Yersinia pestis disease ; Polystyrenes ; Polystyrol ; Publishing ; Research Personnel ; Investigators ; Researchers ; Stains ; Staining method ; stem cells ; Progenitor Cells ; Technology ; Time ; Tissues ; Body Tissues ; Measures ; Case Study ; case report ; Dissociation ; Custom ; base ; culture plates ; Microscope ; improved ; Clinical ; Microscopic ; Phase ; Electroporation ; electroporative delivery ; gene electrotransfer ; Ensure ; Evaluation ; Dermal ; Therapeutic ; Genetic ; Morphology ; tool ; Nature ; programs ; Protocol ; Protocols documentation ; cell type ; Techniques ; System ; Viral ; success ; cell immortalization ; sorting ; Sorting - Cell Movement ; drug development ; drug discovery ; Academia ; Monolayer culture ; Adherent Culture ; DNA Alteration ; DNA mutation ; Genetic mutation ; Sequence Alteration ; genomic alteration ; DNA Sequence Alteration ; Harvest ; Seminal ; Monitor ; Cellular Morphology ; cell morphology ; Process ; Development ; developmental ; Image ; imaging ; cost ; feeding ; microsystems ; cost effective ; cost efficient ; Population ; Consumption ; pluripotency ; induced pluripotent stem cell ; iPS ; iPSC ; iPSCs ; next generation sequencing ; NGS Method ; NGS system ; next gen sequencing ; nextgen sequencing ; Clustered Regularly Interspaced Short Palindromic Repeats ; CRISPR ; CRISPR/Cas system ; personalized medicine ; personalization of treatment ; personalized therapy ; personalized treatment ; individual patient ; Visualization ; precision drugs ; personalized drugs ; stem cell growth ; progenitor Cell growth ;
