UNI-LARGE. Technology for advanced gene editing

Marc Güell Cargol

UPF, Universitat Pompeu Fabra, Spain

Uni-Large has been developed as a new generation of drugs that combines the precision of the gene-editing tool CRISPR/Cas9 and the efficacy of clinically established viral vectors.

A first Uni-Large-based therapy is currently being developed to treat congenital dystrophy type 1A (MDC1A), a disease that affects 1 in 30,000 people. It is produced by mutations in laminin alpha-2, a protein that exceeds the size limit of currently used vectors, so alternative therapies need to be developed.

Uni-Large will provide a safe opportunity for healing. This technology platform can be extended to other diseases, providing a valuable competitive advantage in the gene editing industry, one of the most attractive markets in the pharmaceutical and technology sectors. Uni-Large solves several challenges that prevent a broader deployment of gene editing technologies. It allows for larger genomic modifications and is potentially safer than competing technologies. This project proposes carrying out the necessary steps for the preclinical development of this technology for its safe use in humans to treat muscular dystrophies and other diseases.