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Next generation gene writing platform to cure genetic and oncological diseases

Project description

Gene writing platform for genetic disease treatment

Gene therapy can treat over 10 000 genetic diseases and conditions such as cancer by repairing harmful genetic defects or engineering immune cells. However, many genetic disorders remain untreatable due to the limitations of current editing technologies, which often lack efficiency, precision, and safety. The EIC-funded FiCAT project seeks to overcome these barriers by developing an advanced gene-writing technology that combines RNA-guided nucleases with programmable transposases. This platform enables the safe and precise insertion of large DNA segments into the genome, achieving high integration efficiency and validated performance across multiple models. The FiCAT platform has the potential to transform treatments for genetic and neoplastic diseases, offering new hope for patients with unmet medical needs.

Objective

Gene therapy offers a promising opportunity for treating the more than 10,000 human genetic diseases, as well as other disorders, such as cancer, by editing disease-causing genetic defects or engineering immune cells to target cancer cells. However, most genetic disorders remain untreatable, as gene therapy still holds an untapped potential due to significant limitations of current editing technologies, that are not efficient, precise and safe enough and thus, present limitations in scope and applicability.
Integra Therapeutics, a spin-off of the Pompeu Fabra University (UPF) has developed an advanced genome writing platform that overcomes the limitations of current gene editing technologies. Our proprietary gene writing technology platform- FiCAT combines the precision of RNA-guided nucleases with the effectiveness of programmable transposases to insert large pieces of DNA in a safe, precise and stable way. FiCAT has the capacity to integrate virtually any gene into the genome with unprecedented efficiency, precision and safety. FICAT has already been validated in several ex vivo and in vivo models. With FiCAT, we will unlock the potential of gene writing to cure genetic diseases and bring life-changing therapies to patients suffering from high unmet need genetic and neoplastic diseases.

Fields of science (EuroSciVoc)

Keywords

Programme(s)

Topic(s)

Funding Scheme

HORIZON-EIC-ACC - HORIZON EIC Accelerator

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Call for proposal

(opens in new window) HORIZON-EIC-2024-ACCELERATOR-02

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Coordinator

INTEGRA THERAPEUTICS S.L.
Net EU contribution
€ 2 496 375,00
Address
CARRER DOCTOR AIGUADER 88
08003 Barcelona
Spain
SME
Yes
Region
Este Cataluña Barcelona
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Links
Total cost
No data