CRISPR: Cutting Its Way Through the Market

David Salem [1]
ROBIC, LLP
Lawyers, Patent and Trademark Agents

The field of genome editing remains heavily dominated by the CRISPR-Cas system (CRISPR – “Clustered Regularly Interspaced Short Palindromic Repeats”). What started as a controversial discovery of a bacterial defense mechanism, has now developed into an industry currently worth over a billion dollars and increasing rapidly. Leading the way are three major publicly traded companies: CRISPR Therapeutics, Inc., Editas Medicine, Inc., and Intellia Therapeutics, Inc., each founded by their respective inventors of the technology. Since the birth of these companies in 2013-2014, several successful start-ups, as well as existing companies, have found a way to occupy a piece of the technology either through licensing of the original CRISPR patents or development of novel nucleases and gene editing mechanisms. Increasing support from venture capital is quickly propelling R&D. Since 2012, the number of patent applications filed has increased exponentially. After only ten days into the new decade there are already 76 new patent applications mentioning CRISPR that have been published (Fig. 1). The United Stated tops the world in the number of CRISPR-related patent applications filed, followed by China, Europe, and Canada, a large proportion of which filed by the conglomerate MIT-Broad Institute (Fig. 2). Consequently, the growth of CRISPR and the genomic engineering industry is predicted to reach between 5-10 billion USD in the next five to six years[2][3].

Figure 1

Figure 2

We are already beginning to see a promising therapeutic return on investment in CRISPR research. In November 2019, results from two joint human clinical trials conducted by CRISPR Therapeutics and Vertex Pharmaceuticals have revealed that reintroduction of CRISPR-edited bone marrow cells into two patients was able to dramatically treat beta-thalassemia and sickle cell disease[‡]. After just a single treatment, termed CTX001, the mutation of a single gene causing these hematological diseases is reverted, and the cells carrying the corrected copy of the gene repopulate the body and reverse the effects. Despite the side effects, notably from the required intense chemotherapy for destroying the existing bone marrow cells, as well as the high costs of the treatment being a major concern, CTX001 has received Fast Track designation from the US Food and Drug Administration (FDA). Editas Medicine and Allergan are now beginning to enroll patients for their CRISPR trial in treating Leber congenital amaurosis type 10. This rapid growth in genome editing research and intellectual property is directly impacting the soar of CRISPR companies in the stock market and the development of potential curative therapies of genetically acquired diseases.