Imagine your DNA is like a giant instruction manual with a few typos causing disease. Instead of using a virus to sneak in a correction, this tool acts like a precise pair of scissors and a glue stick that can insert large, healthy pages of instructions. It uses tiny fat bubbles to deliver the fix safely and permanently without the risks associated with viral delivery.
Current gene therapies rely on viral vectors that are expensive to manufacture, potentially unsafe due to immunogenicity, and limited in the size of the genetic payload they can deliver.
A non-viral gene writing platform combining LNPs, RNA-guided nucleases, and programmable transposases for stable DNA integration.
If you are a gene therapy developer dealing with the high cost and safety risks of viral vectors — this project developed a non-viral LNP delivery system that allows for stable integration of DNA cargos up to 22 kb. This reduces immunogenicity and simplifies large-scale manufacturing.
If you are a cancer immunotherapy firm dealing with the difficulty of engineering immune cells to target tumors — this project developed a gene writing tool that can insert large therapeutic payloads precisely. This enables more complex and effective cell engineering for neoplastic diseases.
If you are an orphan drug developer dealing with monogenic diseases like PFIC2 that require large gene replacements — this project developed a platform capable of 11 kb in vivo integration. This provides a safer, non-immunogenic path to curative treatments.
The platform uses a non-viral delivery system based on lipid nanoparticles (LNPs), which the project aims to use to significantly lower production costs compared to traditional viral vectors.
Yes, the project specifically addresses the manufacturing constraints of viral vectors by utilizing a scalable non-viral LNP system to enhance global accessibility.
The project is based on a proprietary gene writing technology platform developed by Integra Therapeutics, a spin-off of Pompeu Fabra University.
The project period runs from 2024-12-01 to 2026-11-30, with an initial focus on a first-in-human clinical demonstration for a liver disorder.
It combines RNA-guided nucleases with programmable transposases to achieve precise and stable integration of DNA into the genome.
The project is led by a single SME, Integra Therapeutics S.L., based in Spain. With a 100% industry ratio and no university or research partners in the consortium, the project is structured for rapid commercial translation and direct ownership of the resulting intellectual property.
Contact Integra Therapeutics S.L. regarding their non-viral gene writing platform.
Contact us to explore licensing opportunities for the FiCAT platform.
