Genetic alterations either cause or contribute to a number of diseases, such as cancer, cystic fibrosis and asthma. Gene therapy could be a successful strategy to treat them, but current technologies have significant limitations. Uni-large aims to provide a solution to gene therapy limitations by combining the precision of CRISPR/Cas9 methodologies with the efficiency of viral vectors. This combination would allow for the precise and efficient delivery of large genomic fragments to treat diseases, as well as safer DNA editing.
- To develop the first drug based on Uni-large, aimed at treating congenital muscular dystrophy type 1A (MDC1A). This disease is an inherited muscular disorder that affects children and results in devastating clinical complications.
Problem to Solve
Uni-large seeks to solve two problems at the same time. First, the drug aims to overcome some of the limitations of traditional gene therapy, such as random gene insertion, restrictions on the size of the DNA fragment, and the risks of genome toxicity.
Second, Uni-large focuses on finding a treatment for MDC1A. This rare genetic disease affects over 35,000 patients in the US and EU combined. Patients show severe neuromuscular complications that lead to respiratory and feeding difficulties, motor development delay, and other problems. There is currently no treatment for MDC1A. Patients are managed with palliative treatments, mainly addressing their symptoms, disabilities and the clinical consequences of their disease.
Uni-large addresses all the limitations described above, which are inherent to current gene editing technologies. It can insert bigger genome fragments, the location of the genomic insert can be controlled, and it reduces the risk of genome toxicity enormously.
Level of Innovation
The Uni-large technology was created and tested in the lab. Its deployment and further development will offer a new and innovative gene editing platform to significantly increase the treatment options for MDC1A as well as for other genetic disorders.