Cell Programming Science

Better Cells for Better Therapies

We currently apply our cell programming approach to immune cells, such as T cells, NK cells and CD34+ cells, for application in oncology and immune disorders. We believe ex vivo cell programming can have profound therapeutic effects upon the adoptive transfer of programmed immune cells to patients. In addition to the use of small molecules to program immune cell function, we have pioneered a proprietary, small molecule‑enhanced induced pluripotent stem cell (iPSC) platform that allows us to program cell fate. The genetic engineering and indefinite expansion of iPSCs, followed by their subsequent differentiation to immune cells, holds great promise as a potentially disruptive approach to the development of “off‑the‑shelf” cell-based cancer immunotherapies.

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Programming Cell Function

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Transforming the Biological Properties and Therapeutic Functions of Cells

Tremendous opportunity exists to pharmacologically program the biological properties and therapeutic function of cells. While advancements in the use, expansion, processing and engineering of hematopoietic cells have opened new avenues of therapeutic application, we believe the biological properties and therapeutic function of hematopoietic cells remains to be optimized. Since our founding, we have been dedicated to programming hematopoietic cells ex vivo. Using advanced molecular characterization tools and technologies, we can systematically and precisely modulate the biological properties of hematopoietic cells ex vivo. We have identified small molecules and biologic modulators that promote rapid and supra physiologic activation or inhibition of therapeutically relevant genes and cell surface proteins, such as those involved in the homing, proliferation and survival of hematopoietic stem cells or those involved in the persistence, proliferation and reactivity of T cells. We believe that this highly-differentiated therapeutic paradigm – systematically and precisely programming the biological properties and therapeutic function of cells ex vivo – is an elegant, cost effective and scalable approach to maximize the safety and efficacy of cellular immunotherapies.

Programming Cell Fate

A Pluripotent Cell Platform for Enabling an Off-the-Shelf Engineered Immunotherapy Revolution

Induced pluripotent stem cell (iPSC) technology has the potential to enable the next frontier in the development of cellular therapies. The seminal discovery that the fate of fully differentiated human cells can be programmed ex vivo through the expression of certain genes is one of the most remarkable scientific breakthroughs of the past decade and was recognized with the award of the 2012 Nobel Prize in Science and Medicine. The advent of iPSCs, with their capacity to be genetically engineered and indefinitely expanded in culture, has served to create a potentially unlimited cell source for differentiation into specialized cell types and has served to enable a profoundly disruptive strategy for developing “off-the-shelf” cellular therapies.

We have pioneered the highly efficient derivation and expansion of human iPSCs for therapeutic use. Since our founding, we have developed a proprietary, small molecule enhanced iPSC platform. Our patent protected iPSC platform enables the isolation, genetic engineering, selection and characterization of pluripotent cells, at the single cell level, for clonal expansion. We believe our iPSC platform can enable the development of entirely new classes of autologous, allogeneic, and genome edited cellular therapeutics with disease transforming potential.

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