Gene therapy with the CRISPR-Cas9 technology holds the potential to treat previously incurable genetic diseases. It allows to repair disease- driving mutations in the genetic code. Despite fast progress in research & development, the CRISPR-Cas9 system still remains inefficient in correcting patient cells. This is the major hurdle encountered by the pharmaceutical and biotech industry, holding their CRISPR-Cas9 gene therapy portfolio back from clinical application.
We are developing a small molecule drug which enhances the efficiency of CRISPR-Cas9 gene therapies. Our drug will accelerate the application of gene editing technologies in biotech and the clinics.