Uni-large is a new generation of gene editing techniques combining the precision of CRISPR/cas9 and the efficiency of viral vectors established in clinic, which allows the insertion of multi-kilobase genome fragments.
Congenital muscular dystrophy Type 1A (MDC1A) is an early onset and life-threatening disease that affects 1-9 in 1,000,000 people worldwide. It is caused by mutations in the laminin alpha-2 gene, a large gene that exceeds the size limit of adeno-associuated viral vectors (AAV), used for in vivo gene therapy. Therefore, it is necessary to develop alternative therapies. Uni-large will provide a safe and therapeutic opportunity.
Uni-Large combines the precision of modern programmable nucleases and specific DNA binding proteins (CRISPR/Cas9, Zinc Finger Proteins) and the efficiency and efficacy of classic vectors for integrative gene delivery (lentivirus, transposases). This technology will allow for targeted integration of large payloads (6-30kb) into the human genome, which constitute its differential value. The therapeutic effect does not depend on HDR (often limiting efficacy), and is potentially safer than competing technologies (absence of double-strand breaks, reduction of insertion mutagenesis).
- Safe and efficient, novel and specific treatment
- MDC1A is life-threatening, no treatment available
- Versatile strategy, applicable to other diseases
- Efficient for large genes
STATE OF DEVELOPMENT
The technology has been tested both in vitro in cells and in vivo using a mouse model of MDC1A with positive results. Further steps should be the development of regulatory preclinical assays and the study of the use of UniLarge to treat other indications.
A US patent application has been filed, obtaining a very positive patentability report.
UniLarge can be extended to other diseases, giving a valuable competitive advantage to the gene therapy industry, one of the most attractive markets in the pharmaceutical and technological sector ($ 7,500 million in 2024).
Technology available for licensing with technical cooperation.
Dr Cinta Diez
Technology Transfer Unit
Pompeu Fabra University
T. +34.93 316 09 13
Gene editing, genome editing, MDC1A, congenital muscular dystrophy, large genes, HDR.