Biotechnology companies Integra Therapeutics and Factor Bioscience unveiled significant advancements in gene-editing technologies for CAR-T cell therapies at the 28th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in New Orleans, taking place May 13-17, 2025.
Integra Therapeutics Demonstrates Enhanced CAR-T Cell Engineering
Barcelona-based Integra Therapeutics presented new pre-clinical data showcasing their FiCAT gene-writing platform's capabilities in creating more sophisticated CAR-T cells. For the first time, the company demonstrated the expression of complex CARs—including bispecific CARs and those with kill switches—in a single DNA donor, potentially simplifying production processes for next-generation cell therapies.
"The presentation at ASGCT marks an important milestone in our work to develop safer and more capable cell engineering with our gene-writing platform with double digit editing efficiencies and multi gene integration for the next generation of cell therapies for oncological and autoimmune diseases," explained Dr. Avencia Sánchez-Mejías, CEO and Co-Founder of Integra Therapeutics.
The pre-clinical data revealed that CAR-T cells created using Integra's technology demonstrated improved ability to eliminate B-ALL tumoral cells compared to methodologies used by market leaders. Additionally, these engineered cells showed enhanced efficacy against B cells from patients with autoimmune diseases such as lupus and rheumatoid arthritis.
Integra also presented results from CAR-T cells generated using novel CRISPR-Cas12l nucleases in combination with their FiCAT platform. This approach, developed through a strategic agreement with Caszyme, demonstrated better efficiency than conventional CRISPR-Cas9 systems.
The company's presentations included:
- "Precise Gene Writing System for CAR-T cell therapy generation" (Abstract #1140)
- "Development of viral-free FiCAT gene writing platform for liver-directed in vivo application" (Abstract #1141)
Factor Bioscience Advances Non-Viral Gene Editing Technologies
Cambridge, Massachusetts-based Factor Bioscience presented four studies highlighting their non-viral gene editing and cell engineering innovations, with a particular focus on allogeneic CAR-T cell therapy programs.
"We are excited to present the latest data from our allogeneic CAR-T cell therapy programs, as well as recent advances in our nucleic acid delivery and cell differentiation platforms at ASGCT 2025," said Dr. Matt Angel, Co-Founder, Chairman and CEO of Factor Bioscience. "The data that we will be showcasing include non-viral transgene insertion for next-generation allogeneic CAR-T and other cell and gene therapy applications."
Dr. Kyle Garland, Director of Translational Science at Factor, added, "This past year, we have made substantial progress advancing our pipeline programs by strategically applying and optimizing the technologies that Factor has developed over the past fourteen years."
Factor Bioscience's presentations covered several technological innovations:
- UltraSlice Gene-Editing mRNA for efficient transgene insertion into the TRAC locus in primary human T cells
- Incorporation of 5-methoxyuridine for improved targeted transgene insertion in iPSCs and T cells
- A novel polyvalent ionizable lipid library for nebulized delivery of mRNA to lung cells
- Neural induction of gene-edited mRNA-reprogrammed iPSCs
Implications for Cell and Gene Therapy Development
The advancements presented by both companies represent significant progress in addressing key challenges in cell and gene therapy development. Non-viral gene editing approaches may offer advantages over viral vector-based methods, including reduced immunogenicity, lower manufacturing costs, and improved safety profiles.
For CAR-T cell therapies specifically, these technologies could enable the development of more sophisticated therapeutic approaches with multiple targeting mechanisms and built-in safety features. The ability to create complex CARs in a single DNA donor, as demonstrated by Integra Therapeutics, could streamline manufacturing processes and potentially reduce production costs.
Additionally, the improved efficiency in targeting B cells from autoimmune disease patients suggests these technologies could expand the application of CAR-T therapies beyond oncology into autoimmune conditions like lupus and rheumatoid arthritis, representing a significant potential expansion of the therapeutic landscape.
Future Directions
As these technologies continue to mature, they may enable the development of "off-the-shelf" allogeneic CAR-T therapies that overcome many of the limitations of current autologous approaches. The non-viral delivery methods being developed by both companies could also address manufacturing challenges and safety concerns associated with viral vectors.
The data presented at ASGCT 2025 by Integra Therapeutics and Factor Bioscience highlight the rapid pace of innovation in gene editing and cell engineering technologies, with potential implications for improving treatment options for patients with cancer and autoimmune diseases in the coming years.
