For the first time, four therapeutic genes have been inserted and one ‘unwanted’ gene removed from the DNA of preclinical models in a single intervention
These results come from a preclinical study conducted by the Cell Engineering team at Integra Therapeutics using the FiCAT gene-writing platform.
The aim is to enable next-generation CAR-T cell therapies with safe integration of large therapeutic designs, while simplifying the traditional cell therapy manufacturing process by reducing time and cost.
The data will be presented at the 29th Annual Meeting of the American Society for Gene and Cell Therapy, taking place this week in Boston.
The Cell Engineering team at Integra Therapeutics has conducted a preclinical trial demonstrating the FiCAT platform's ability to simultaneously insert up to four genes in a therapeutic payload into a defined, safe location in the genome, while deactivating an unwanted gene—all in a single step.
These results are significant because current CAR-T cell therapy manufacturing typically requires multiple steps to introduce each genetic modification, increasing complexity, time, and cost, and limiting the amount of genetic information incorporated into the final product. The level of precision and multiplexing achieved with FiCAT in a single intervention has not previously been demonstrated.
The new preclinical data have been selected for an oral presentation at the American Society for Gene and Cell Therapy (ASGCT) Annual Meeting, held May 11–15 in Boston, Massachusetts.
Currently, retroviral and lentiviral vectors (LVVs) are the standard for manufacturing CAR-T cell therapies and are used in all approved products. In a head-to-head comparison—the most rigorous method for evaluating new technologies—T cells modified with FiCAT matched or outperformed LVVs in key performance indicators, including cell viability, tumor-killing ability, and genotoxicity associated with integration. Unlike LVVs, which insert genetic material randomly into the genome—posing risks such as oncogene activation or variable expression—FiCAT inserts genetic payloads into one specific predetermined safe site.
“We are very encouraged by the results of this preclinical validation, which position our FiCAT platform at the forefront of next-generation CAR-T cell therapies—safer, more capable, and scalable—for the treatment of autoimmune and oncological diseases that currently lack therapeutic alternatives. However, clinical validation will be required,” said Avencia Sánchez-Mejías, PhD, CEO and Co-Founder of Integra Therapeutics.
According to Margot Pont, PhD, VP Translational Development at Integra Therapeutics, “The application of FiCAT in primary T cells opens new possibilities for designing next-generation CAR-T cells, as it removes the size limitations of the current gold standard, with increased safety. The data presented at ASGCT are highly relevant, but we are committed to continuing to generate additional data needed for regulators, investors, and the pharmaceutical industry to support the adoption of this new platform as an alternative to the current standard.”
The company will soon complete the submission of a manuscript for publication in a high-impact scientific journal.
Presentation Details
Title: FiCAT: A Precise Gene-Writing System for Next-Generation CAR-T Cell Therapy Production
Session: In Vivo Engineering of CAR T Cells for Autoimmune Disease
Date and Time: May 14, 10:15 a.m.–12:00 p.m. (local time)
Location: Westin Seaport, Grand Ballroom CDE (Concourse Level)
Abstract No.: 342
Title: FiCAT Gene-Writing Platform for Liver-Directed In Vivo Gene Therapy
Date and Time: May 14, 5:00 p.m.–6:30 p.m. (local time)
Location: Poster Reception
Abstract No.: 3093
…
About the FiCAT gene-writing platform
FiCAT (Find-Cut-and-Transfer) is a gene-writing platform developed by Integra Therapeutics to enable safer and more effective cell and gene therapies. This technology combines the precision of CRISPR-Cas, which targets specific DNA regions, with a proprietary PiggyBac transposase responsible for controlled gene insertion.
This approach enables the precise insertion of both small and large DNA fragments into the genome, improving stability, accuracy, and genetic payload capacity. It expands applications in developing treatments for genetic diseases, cancer, and autoimmune disorders, in both in vivo and ex vivo settings.
FiCAT addresses technical limitations and safety concerns of current technologies, offering a more flexible and universal gene-editing solution for clinically relevant cells. It also integrates high-throughput screening and AI-driven process optimization to improve efficiency and cell viability, surpassing traditional techniques.
…
About Integra Therapeutics
Integra Therapeutics is a biotechnology company developing next-generation gene-writing tools to improve the efficacy, precision, and safety of advanced therapies. Founded in 2020 by Marc Güell, PhD and Avencia Sánchez-Mejías, PhD as a spin-off from Pompeu Fabra University (UPF), the company is backed by international investors (AdBio Partners, Columbus Venture Partners, Invivo Capital, and Takeda Ventures), the European Commission, and the Spanish government. Headquartered in Barcelona, its laboratory is located at the Advanced Therapies Platform of Sant Joan de Déu Hospital in Esplugues de Llobregat. The company obtained My Green Lab certification in 2023. Learn more: https://integra-tx.com
These results come from a preclinical study conducted by the Cell Engineering team at Integra Therapeutics using the FiCAT gene-writing platform.
The aim is to enable next-generation CAR-T cell therapies with safe integration of large therapeutic designs, while simplifying the traditional cell therapy manufacturing process by reducing time and cost.
The data will be presented at the 29th Annual Meeting of the American Society for Gene and Cell Therapy, taking place this week in Boston.
The Cell Engineering team at Integra Therapeutics has conducted a preclinical trial demonstrating the FiCAT platform's ability to simultaneously insert up to four genes in a therapeutic payload into a defined, safe location in the genome, while deactivating an unwanted gene—all in a single step.
These results are significant because current CAR-T cell therapy manufacturing typically requires multiple steps to introduce each genetic modification, increasing complexity, time, and cost, and limiting the amount of genetic information incorporated into the final product. The level of precision and multiplexing achieved with FiCAT in a single intervention has not previously been demonstrated.
The new preclinical data have been selected for an oral presentation at the American Society for Gene and Cell Therapy (ASGCT) Annual Meeting, held May 11–15 in Boston, Massachusetts.
Currently, retroviral and lentiviral vectors (LVVs) are the standard for manufacturing CAR-T cell therapies and are used in all approved products. In a head-to-head comparison—the most rigorous method for evaluating new technologies—T cells modified with FiCAT matched or outperformed LVVs in key performance indicators, including cell viability, tumor-killing ability, and genotoxicity associated with integration. Unlike LVVs, which insert genetic material randomly into the genome—posing risks such as oncogene activation or variable expression—FiCAT inserts genetic payloads into one specific predetermined safe site.
“We are very encouraged by the results of this preclinical validation, which position our FiCAT platform at the forefront of next-generation CAR-T cell therapies—safer, more capable, and scalable—for the treatment of autoimmune and oncological diseases that currently lack therapeutic alternatives. However, clinical validation will be required,” said Avencia Sánchez-Mejías, PhD, CEO and Co-Founder of Integra Therapeutics.
According to Margot Pont, PhD, VP Translational Development at Integra Therapeutics, “The application of FiCAT in primary T cells opens new possibilities for designing next-generation CAR-T cells, as it removes the size limitations of the current gold standard, with increased safety. The data presented at ASGCT are highly relevant, but we are committed to continuing to generate additional data needed for regulators, investors, and the pharmaceutical industry to support the adoption of this new platform as an alternative to the current standard.”
The company will soon complete the submission of a manuscript for publication in a high-impact scientific journal.
Presentation Details
Title: FiCAT: A Precise Gene-Writing System for Next-Generation CAR-T Cell Therapy Production
Session: In Vivo Engineering of CAR T Cells for Autoimmune Disease
Date and Time: May 14, 10:15 a.m.–12:00 p.m. (local time)
Location: Westin Seaport, Grand Ballroom CDE (Concourse Level)
Abstract No.: 342
Title: FiCAT Gene-Writing Platform for Liver-Directed In Vivo Gene Therapy
Date and Time: May 14, 5:00 p.m.–6:30 p.m. (local time)
Location: Poster Reception
Abstract No.: 3093
…
About the FiCAT gene-writing platform
FiCAT (Find-Cut-and-Transfer) is a gene-writing platform developed by Integra Therapeutics to enable safer and more effective cell and gene therapies. This technology combines the precision of CRISPR-Cas, which targets specific DNA regions, with a proprietary PiggyBac transposase responsible for controlled gene insertion.
This approach enables the precise insertion of both small and large DNA fragments into the genome, improving stability, accuracy, and genetic payload capacity. It expands applications in developing treatments for genetic diseases, cancer, and autoimmune disorders, in both in vivo and ex vivo settings.
FiCAT addresses technical limitations and safety concerns of current technologies, offering a more flexible and universal gene-editing solution for clinically relevant cells. It also integrates high-throughput screening and AI-driven process optimization to improve efficiency and cell viability, surpassing traditional techniques.
…
About Integra Therapeutics
Integra Therapeutics is a biotechnology company developing next-generation gene-writing tools to improve the efficacy, precision, and safety of advanced therapies. Founded in 2020 by Marc Güell, PhD and Avencia Sánchez-Mejías, PhD as a spin-off from Pompeu Fabra University (UPF), the company is backed by international investors (AdBio Partners, Columbus Venture Partners, Invivo Capital, and Takeda Ventures), the European Commission, and the Spanish government. Headquartered in Barcelona, its laboratory is located at the Advanced Therapies Platform of Sant Joan de Déu Hospital in Esplugues de Llobregat. The company obtained My Green Lab certification in 2023. Learn more: https://integra-tx.com
