Integra Therapeutics Presents CAR-T Cell Data at ASGCT 2025
The company’s technology was used to create CAR-T cells that demonstrated the expression of complex CARs in a single DNA donor.
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Integra Therapeutics, a creator of gene-writing tools, announced it is presenting new pre-clinical data at the 28th annual meeting of the American Society of Gene and Cell Therapy (ASGCT), which is taking place in New Orleans May 13–17. According to the company, its technology has been used to create chimeric antigen receptor (CAR) T cells that allow for the expression of complex CARs, such as bispecific and with a kill switch, in a single DNA donor.
Integra stated in a press release that the functionality of its CAR-T cells simplifies the production process and is comparable to the methodology of leaders in the market. “An improved ability to eliminate B-ALL tumoral cells and B cells from autoimmune diseases patients (i.e., lupus and rheumatoid arthritis) was demonstrated,” the company stated in the release (1).
“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 Avencia Sánchez-Mejías, CEO and co-founder of Integra Therapeutics, in the release.
Development of effective gene-editing technologies for ex vivo therapies, such as hematopoietic stem cells and CAR-T cells, is challenging, according to Integra, and current platforms have technical limitations and safety concerns. However, Integra’s Find and Cut-and-Transfer (FiCAT) gene delivery tool provides a flexible and universal solution for editing small regions and large genes in clinically relevant primary cells, according to the company (1). FiCAT has a CRISPR-Cas nuclease and a proprietary engineered transposase that provides efficiency through rational protein engineering and an unsupervised variability generation approach. “Over 400,000 variants are tested using a proprietary high- throughput screening system grounded in synthetic biology principles. Our system compared favorably against existing systems like homology directed repair or homology-independent targeted insertion using CRISPR-Cas9 alone. To enhance both efficiency and viability, electroporation is coupled with inhibitors targeting toxicity associated with double-stranded exogenous DNA,” the company stated in the release.
Integra will also present results from a partnership with Caszyme, through which CAR-T cells were generated with clustered regularly interspaced short palindromic repeats (CRISPR)-Cas12I nucleases in combination with the FiCAT tool, showing better efficiency than CRISPR-Cas9.
In November 2024, the two companies signed a licensing agreement that allowed Integra to use Caszyme’s novel Cas121 nucleases to develop gene and cell therapies (3). Integra will incorporate Cas121 into the FiCAT 2.0 gene-writing platform. Milestone payments of up to Euro 40 million and royalties will be provided to Casyme as part of the agreement.
“Caszyme’s goal is to enable therapeutic companies to develop novel, efficient and affordable gene editing modalities. This collaboration with Integra Therapeutics is a perfect alignment between two highly innovative European companies, and when successful, will help bring advanced gene editing based therapies to the market,” Monika Paulė, CEO and co-founder of Caszyme, said at the time in a press release (2).
The company will be presenting both “Precise Gene Writing System for CAR-T Cell Therapy Generation” and “Development of Viral-free FiCAT Gene Writing Platform for Liver-directed In Vivo Application” at ASGCT on May 14 at 5:30PM CDT.
References
1. Integra. Integra Therapeutics Announces New Pre-Clinical Data at 28th Annual Meeting of American Society of Gene and Cell Therapy (ASGCT). Press Release. May 13, 2025.
2. Integra. Gene Writing Platform, The Next Step in Gene Writing. Integra-tx.com (accessed May 14, 2025). https://integra-tx.com/gene-writing-platform/
3. Caszyme. Caszyme and Integra Therapeutics Sign Licensing Agreement for Novel CRISPR Cas12l Nucleases. Press Release. Nov. 4, 2024. https://caszyme.com/news/caszyme-and-integra-therapeutics-sign-licensing-agreement-for-novel-crispr-cas12l-nucleases/
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