In a stroke, one would say the iPhone started the mobile phone market with its internet-browsing capabilities, camera, interactive touchscreen, and app store. Only it didn’t. Smartphone manufacturer LG released the LG Prada in 2006 that came with a camera, app store, internet-browsing capabilities, and the same type of interactive touchscreen as the iPhone. LG had put on the right technology, but Apple did it better. A similar thing might now be happening in the world of gene editing.
Even though there are three different gene-editing tools used today, CRISPR is the most widely recognized of them all. CRISPR, or clustered regularly interspaced short palindromic repeats, largely dominates because of its simplicity and versatility. CRISPR Therapeutics is a leading gene-editing company that uses CRISPR to develop transformative medicines for treating diseases. Despite being one of the most significant discoveries of the 21st century, new methods are being discovered that could make CRISPR-Cas9 the LG Prada of the gene-editing world.
The Current Situation
Jennifer Doudna, a Biochemist and Professor of Biology and Structural Chemistry at the University of California, Berkeley, along with Emmanuelle Charpentier, the head of the lab at the University of Vienna, stated that CRISPR could allow scientists to rewrite DNA in June 2012. However, the Broad Institute of MIT and Harvard University filed a patent seven months later that tried to invalidate their claim to bring the technique into the marketplace. They did so by providing a more detailed description of the method in the cells that make up living beings.
In spite of these challenges, Doudna and Charpentier won the 2020 Nobel Prize in Chemistry for the discovery of bacteria’s gene-editing capabilities using CRISPR-Cas9. While the patent is still valid, other companies are finding new CRISPR tools to be even more effective at editing genes in some cases.
The novel CRISPR approach, CRISPR-Cas9, makes a blunt double-stranded DNA break. One major challenge of this tool is its ability to occasionally cut the DNA at unintended sites. Although scientists and researchers are trying hard to overcome this challenge, it’s a frightening possible side effect. The next popular tool was CRISPR-Cas12, which makes cuts and can edit epigenomes. These are chemical compounds that tell genes to turn on or off.
CRISPR-Cas13, on the other hand, has an impact on gene expression as it targets RNA instead of DNA. However, the latest studies have discovered CRISPR-CasX and CasY, wherein CasX is smaller than Cas9 and can be used to control gene expression, alongside gene-editing. CasY is similar to Cas9 in some ways but is made up of a completely different protein structure. This allows it to function in various circumstances.
In 2017, Excision BioTherapeutics, a leading biotechnology company, obtained an exclusive license for both the new gene-editing tools from Doudna’s lab at UC Berkeley. In March 2020, the company successfully used Cas9 to remove immunodeficiency virus (SIV) genomes from monkeys. Furthermore, the company is effectively working on other gene-editing tools for treating herpes and a rare disease that attacks the central nervous system (CNS). Excision is also employing CasX and CAS9 to find a potential treatment for Hepatitis B and COVID-19, respectively.
It is safe to assume that CRISPR Therapeutics is still dominating the marketplace. The company is currently targeting beta-thalassemia and sickle cell disease and several immune cell products to kill cancerous tumors. In spite of all these advances in gene-editing approaches, even Excision BioTherapeutics is dependent on the CRISPR-Cas9 tool for conducting most of its clinical studies.
The Bottom Line
Researchers and scientists will continue to find better and more effective gene-editing approaches to treat and cure diseases. The fact that a biotech startup managed to grab an exclusive license from a Nobel Prize-winning lab that basically invented gene editing should be worrisome. Investors and shareholders should look out for indications as to whether these gene-editing approaches are being applied in domains once assumed to be the specialty of CRISPR Therapeutics’, and its Cas9 methods.