Editas Medicine (NASDAQ: EDIT) is a biotechnology company that focuses on researching drugs using CRISPR. CRISPR, short for clustered regularly interspaced short palindromic repeats, is a technology developed as a result of observing a process that naturally occurs within viruses. By isolating this CRISPR segment of the virus responsible for replication and deletion, scientists have been able to develop “genetic scissors”, which can delete, remove, and modify portions of DNA. With these treatments, the company is hopeful that some of the most serious diseases in the world, including many that do not currently have treatment options yet, can be cured.
What is Editas Medicine (EDIT)?
Editas Medicine was founded in 2013 by a team of scientists with ties to Harvard and MIT. With a stellar scientific team at the helm, the company was able to secure funding early on to further pivotal CRISPR research. Through their first round of Series B funding, the company received interest from a significant group of investors that included Bill Gates and his chief science and tech advisor Boris Nikolic. Editas was able to use this interest to raise over $120 million dollars, and also form valuable connections with larger companies in the sector. Early on, the company launched a collaboration with Juno Therapeutics to develop therapies to treat cancer. The two companies estimated that the potential value of the collaboration was $737 million dollars.
The companies research and development team focuses on both in vivo medicines, which edit genes inside the body, as well as ex vivo medicines, which edit genes outside of the body and transfer them back into the patient. The platform used for the CRISPR drug development involves both the Cas9 and Cas12a nucleases. These two different proteins bind to DNA and allow for gene editing in slightly different ways. These two CRISPR variants allow for different targeting and editing abilities, giving researchers multiple options when considering the best choice for drug development to target specific diseases.
Why does Editas Medicine (EDIT) Matter?
Editas has a pipeline of drugs in development that focus on three broad categories: ocular diseases, blood diseases, and oncology. Furthest along are their gene-editing medicines to treat ocular disease. Leber congenital amaurosis, or LCA, is an inherited genetic mutation in at least 18 different genes. LCA is the leading cause of color blindness in children and can lead to significant vision loss over time. The mutation which causes LCA is found in 3 out of 100,000 children, and there is currently no cure for LCA on the market. Equally far along are their treatments for blood diseases known as hemoglobinopathies. Editas is slightly behind CRISPR Therapeutics, another company operating in this space that has multiple drugs to treat sickle cell disease and β-Thalassemia in clinical trials. Another category of drugs in the pipeline is their oncological treatment drugs, which focus on editing T cells to make them better at fighting off cancer in the body.
The novel approach Editas is taking to CRISPR-based medicines is helping it stand out from the competition. Editas was the first company to develop and test a drug for in-vivo usage. Through this technique, Editas researchers injected a virus that contained edited DNA into a patient suffering from LCA. This edited DNA contained instructions that told the body how to modify its own DNA to remove the genetic segment that causes the disorder. While still in the early stages, the potential for this type of treatment option only further cements the potential for CRISPR in the future of medicine.
Editas Medicine (EDIT) Prospects
Editas currently has close to a dozen different CRISPR-developed medicines at various stages of production. Many of these are being developed in partnership with larger companies like Allergan Pharmaceuticals. Through these partnerships, the medicines are co-developed and then profits are shared equally, with the bigger company responsible for a majority of the commercialization process. Both Editas and partners have developed a robust internal and external manufacturing process, and the company is ready to scale if their drugs reach the approval stage. On the oncology side, the company has partnered with industry giant Bristol Myers Squibb. Through this relationship, Editas has been provided with over $100 million in funding thus far.
Aside from their current focus on ocular disease, blood disease, and oncology, the methods developed thus far by the company are setting the stage for future medicines to treat a variety of other genetic disorders. The company estimates that there are over 6,000 human genetic disorders, all of which have the potential to be cured by genetic editing. Any research done by Editas today using the CRISPR platform will only accelerate future growth into other areas. The company is using many new methods, and has been protecting these methods through patents. In total, the company holds over 220 patents and has an additional 800 pending. Experiments such as their first in the world in-vivo treatment options are continuing to push the boundaries of what CRISPR technology is capable of.
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Editas is one of many newer biotechnology companies using the CRISPR platform to research and develop new drugs. To stand out in a crowded field, the company has funneled money into research and development, taking the current knowledge on CRISPR and looking for new and innovative ways to push for wider spread applications. CRISPR undoubtedly will be at the forefront of the next generation of medicine, and Editas has positioned itself well to remain a global leader.