The Uni-large project, which is developing new technology capable of modifying the genome to address various diseases more safely and efficiently than other solutions, has been selected by the Caixaimpulse Consolidate programme to speed up its development. This initiative of “la Caixa”and Caixa Capital Risc will provide until 300,000 euros of funding and advice to four innovative projects in the field of health that have emerged in universities, hospitals and research centres in Spain and Portugal. The purpose of the grant is to help technologies reach maturity to drive them to the investment phase.

Uni-large is led by Marc Güell, principal investigator of the Translational Synthetic Biology Research Group at the Department of Experimental and Health Sciences (DCEXS) at UPF. It aims to create technology that serves to treat genetic diseases and some cancers arising from gene malfunction (such as leukaemia, for example). Uni-large proposes adding a copy in good condition of the damaged gene to the genome of the patient so that it regains its normal function and the disease remits. UPF has protected this invention with a patent application.

It aims to create technology that serves to treat genetic diseases and some cancers arising from gene malfunction.

Currently, the project team is investigating the usefulness of its gene editing technology in the treatment of type 1A (MDC1A) congenital muscular dystrophy, a hereditary disease for which there is no treatment. This is the most common type of congenital muscular dystrophy, causing progressive weakness and the loss of muscle mass.

Güell highlights three advantages of Uni-large. On the one hand, its “universality”: other gene editing techniques such as the CRISPR method, could correct individual mutations of each patient, while the new proposal would help all patients with a given disease. Moreover, it has safety reasons in its favour: “with CRISPR, you have to make a cut in the genome to repair the mutation and this can cause accidents such as the loss or breakage of chromosomes, whereas our technology does not cut, we just attach a gene”, which would prevent risks. Finally, Güell stresses the effectiveness of the method, especially for diseases like MDC1A where “the gene involved is very large, and in these cases, with other technologies, repairs are less efficient”.

The programme will provide funding to complete project development, and also advice from experts who will provide support for the drafting of valorization and commercialization plans.

This first edition of the Caixaimpulse Consolidate programme will provide UPF researchers with until 300,000 euros, which will be used to complete project development, and also advice from experts who will provide support for the drafting of valorization and commercialization plans. The scientists aim to take all the necessary steps to make the project commercially viable, taking it to an “advanced preclinical phase” in 2022.

“Exceptional” support

Güell shows his “immense gratitude” for having the obtained the grant and believes it will have a “major impact” on the development of Uni-large: “now, more than ever, we need this help because we are in a fairly advanced stage for a scientific project, but we are not as advanced as most of the projects receiving investment; hence, having this exceptional support is highly valuable and will enable us to work to reach patients as soon as possible”, he assesses.

In 2018, Uni-large was one of the projects selected by the Caixaimpulse Validate programme, which supports research initiatives at an earlier stage of development. On that occasion, the project received guidance and was awarded 70,000 euros, which according to Güell proved “very important” to give impetus to the project.