Editing the human genome allows scientists to make changes in DNA within the body to modify physical traits, reduce the risk of disease, and for the treatment of rare genetic conditions and cancer. It has the potential for the treatment of diseases for which there are no available therapies. Clustered Regularly Interspace Short Palindromic repeats (CRISPR) and CRISPR-associated protein 9 (Cas9) is the most widely used technology for editing the genome. While modifying DNA with CRISPR has become a common practice in basic scientific research, using the technology for disease modification in the clinic is associated with off-target mutations that can reduce efficacy and exhibit dangerous side effects.

The technology herein is a method called iCAP (in-situ-cut-and-paste) for improving CRISPR/Cas9 gene editing by utilizing a simplified approach for cutting DNA sequences and replacing them with pre-constructed templates. DNA fragments are preconstructed in vitro before editing and then precisely inserted at any location within the genome. This method could overcome the difficulties associated with current CRISPR/Cas9 editing techniques, thereby improving efficacy and reducing off-target toxicity. 

Potential Application

This technology could be used as a new treatment for a plethora of diseases such as rare genetic disorders (muscular dystrophy, hemophilia, & blindness), infectious diseases (AIDS) and cancer. It could also be used throughout the research and development industry as a basic science tool for investigating these same diseases.

Opportunity

Rising prevalence of genetic disorders, as well as a significant investment by biotechnology and pharmaceutical companies, are contributing to double-digit growth of the gene-editing industry. The global market for gene editing was $1.4 billion in 2018 and expected to exceed $4.4 billion by 2023 at a CAGR of 25.1 percent. CRISPR/Cas9 is the most prominent technology and expected to attribute $3 billion of the overall gene editing industry. Recently, gene-editing technologies have entered clinical trials for the treatment of rare diseases and cancers. The development of a more efficacious and safe method of gene editing is of significant interest to the pharmaceutical industry.

Rowan University is looking for a partner for further development and commercialization of this technology through a license.