Fate Therapeutics Presents Late-Breaking Preclinical Data Highlighting Unique Advantages of Clonal Master Engineered iPSC Lines for Off-the-Shelf CAR T-cell Therapy at 2019 AACR Annual Meeting
High-Throughput Selection of a Single Gene-Edited iPSC Enables Mass Production of Uniformly Engineered CAR T Cells
FT819 Off-the-Shelf CAR19 T-cell Product Candidate Demonstrates Complete Elimination of TCR Expression and Integration of Novel 1XX CAR into TRAC Locus
The Company’s proprietary approach to CAR T-cell therapy utilizes a one-time genetic modification event followed by high-throughput selection of a single gene-edited induced pluripotent stem cell (iPSC). In contrast to repeatedly engineering large populations of patient- or donor-derived T cells which results in batch-to-batch and cell-to-cell variability, the Company has shown that a single gene-edited iPSC can be maintained as a clonal master engineered iPSC line which can be repeatedly used for production of uniformly engineered CAR T cells.
“The efficiency, accuracy, and uniformity of genetic modifications have the potential to impact the safety and efficacy of CAR T-cell therapy. We know that, when engineering a population of T cells, current gene-editing technologies create genomic heterogeneity and result in undesired byproducts, such as DNA mutagenesis and translocations. The selection of a single gene-edited cell for the derivation of a clonal master engineered iPSC line ensures that the effects of gene editing can be fully characterized and that only engineered T cells meeting rigorous quality standards are administered to patients,” said
New preclinical data presented today at AACR for FT819, the Company’s universal, off-the-shelf CAR19 T-cell product candidate being developed under a collaboration with
Scientists from the Company and MSK are using clonal master engineered iPSC lines under the collaboration to overcome the complexity, heterogeneity, and substantial costs associated with engineering T cells from a patient or a donor. As proof-of-principle for the therapeutic advantages arising from selecting a single gene-edited cell for the production of CAR T-cell therapy, the scientists engineered a population of 545 cells using CRISPR-directed gene editing and found that only about 5% of cells contained both bi-allelic disruption of the TCR and insertion of the CAR into the
Previously published work by Dr. Sadelain in the journal Nature has shown that directing a CD19-specific CAR to the
About Fate Therapeutics’ iPSC Product Platform
The Company’s proprietary induced pluripotent stem cell (iPSC) product platform enables mass production of off-the-shelf, engineered, homogeneous cell products that can be administered in repeat doses to mediate more effective pharmacologic activity, including in combination with cycles of other cancer treatments. Human iPSCs possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Company’s first-of-kind approach involves engineering human iPSCs in a one-time genetic modification event and selecting a single iPSC for maintenance as a clonal master iPSC line. Analogous to master cell lines used to manufacture biopharmaceutical drug products such as monoclonal antibodies, clonal master iPSC lines are a renewable source for manufacturing cell therapy products which are well-defined and uniform in composition, can be mass produced at significant scale in a cost-effective manner, and can be delivered off-the-shelf to treat many patients. As a result, the Company’s platform is uniquely capable of addressing the limitations associated with the production of cell therapies using patient- or donor-sourced cells, which is logistically complex and expensive and is fraught with batch-to-batch and cell-to-cell variability that can affect safety and efficacy. Fate Therapeutics’ iPSC product platform is supported by an intellectual property portfolio of over 100 issued patents and 100 pending patent applications.
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Forward-Looking Statements
This release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 including statements regarding the Company’s advancement of and plans related to its research and development of iPSC-derived cell products for the treatment of cancer, the safety and therapeutic potential of its iPSC-derived cell product candidates, including FT819, and the scope of the Company’s intellectual property rights. These and any other forward-looking statements in this release are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that the Company may cease or delay preclinical or clinical development of any of its product candidates for a variety of reasons (including requirements that may be imposed by regulatory authorities on the initiation or conduct of clinical trials or to support regulatory approval, difficulties in manufacturing or supplying the Company’s product candidates for clinical testing, and any adverse events or other negative results that may be observed during preclinical or clinical development), the risk that results observed in preclinical studies of its product candidates, including FT819, may not be replicated in ongoing or future clinical trials or studies, the risk that its product candidates may not produce therapeutic benefits or may cause other unanticipated adverse effects, and the risk that any of the patents in the Company’s intellectual property portfolio may be challenged and that such a challenge may be successful, resulting in loss of any such patent or loss or reduction in the scope of one or more of the claims of a such patent. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company’s actual results to differ from those contained in the forward-looking statements, see the risks and uncertainties detailed in the Company’s periodic filings with the
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Source: Fate Therapeutics, Inc.