PACT Pharma Highlights Capabilities and Versatility of Novel Non-Viral Gene Editing Technology in Presentation at American Society of Gene & Cell Therapy (ASGCT) 25th Annual Meeting

Highly Versatile PACT^NV™ Non-Viral Gene Editing Platform Demonstrates Ability to Knock-Out, Knock-Down, Knock-In and Precisely Regulate Target Genes in Single Step


Technology Has Potential to Expand the Applicability of T Cell-Based Therapeutics and Other Cellular Therapies


South San Francisco, CA – May 16, 2022 – PACT Pharma, Inc., a clinical-stage company developing transformational personalized neoantigen-specific T cell receptor (neoTCR) T cell therapies for the eradication of solid tumors, today announced that new data on PACT^NV™, the company’s non-viral precision gene editing technology, were highlighted in a poster presentation at the American Society of Gene & Cell Therapy (ASGCT) 25th Annual Meeting. Presented findings demonstrated the potential of the PACT^NV technology to enhance the functionality and applicability of T cell-based therapeutics, as well as other cellular therapies, through targeted gene editing. Specifically, researchers showed the ability of the versatile platform to knock-out, knock-down, knock-in and precisely regulate target genes in a single step to achieve desired activity for its neoTCR T cells. The ASGCT conference is being held May 16-19, 2022, in Washington, D.C.


The poster presentation reported research findings by PACT scientists demonstrating the ability to utilize the company’s PACT^NV technology to generate TCR T cells in a single step. In creating these novel patient-specific neoTCR T cells, PACT highlighted the following capabilities of its gene editing platform:


  • Insertion and expression of neoTCRs within T cells.


  • Simultaneous elimination of the expression of the endogenous TCRs within same T cells.


  • Knock-out of the transforming growth factor beta (TGF-β) receptor type 2 (TGFBR2) gene, generating a T cell that can both recognize a patient-specific tumor and is resistant to the immunosuppressive signaling by TGF-β.


  • shRNA driven knock-down of multiple additional genes without any associated double-stranded break, generating enhanced T cells that can both recognize a patient-specific tumor and, depending on the targeted transcript, possess resistance to immunosuppression, increased persistence, and/or improved functional avidity.


  • Development of T cell activation-induced promoters for conditional expression of payloads. 


“Taken together, the powerful gene editing capabilities possessed by our PACT^NV platform offer the potential to expand the applicability of T cell therapeutics, while extending the potential use of the technology to other cellular therapies,” said Stefanie Mandl, Ph.D., senior vice president, head of research at PACT Pharma. “We are particularly excited about the versatility to add, eliminate and/or precisely modulate multiple genes within our neoTCR T cells to create novel, personalized therapeutic candidates with the potential to address a range of solid tumors. We look forward to continuing our research and development efforts in support of our PACT^NV technology, while continuing our ongoing Phase 1 clinical trial of non-viral PACT^NV™ gene-edited autologous neoTCR T cells in advanced and metastatic solid tumors.”


PACT is currently conducting a Phase 1 clinical trial evaluating the safety, tolerability and feasibility of adoptive cell therapy with its non-viral PACT^NV gene-edited autologous neoTCR T cells in advanced and metastatic solid tumors. This trial, in combination with extensive preclinical studies, has provided the company with unique data sets and insights derived from the core technologies that comprise its personalized adoptive T cell therapy platform for the treatment of solid tumors.


A copy of the poster presented at the ASGCT conference is available on the “Events” page of the PACT Pharma website at:



About PACT Pharma, Inc.

PACT is a clinical-stage biopharmaceutical company pioneering individualized, non-viral precision genome engineered, tumor-specific T cell therapies for the treatment of patients with solid cancers. PACT is now enrolling patients in its first-in-human Phase 1 clinical trial.


Each person's cancer emerges with a private signature of mutations. PACT identifies these cancer mutation targets (neoantigens) for each person, biochemically verifies and captures T cells from their blood and uses its proprietary, non-viral precision genome engineering technologies to manufacture a personalized immune cell therapy product for each person with cancer.


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