CRISPR Therapeutics (NASDAQ: CRSP) is a biotechnology company that is developing therapeutics using a new and innovative methodology. CRISPR Therapeutics is named after a genetic editing technique developed by one of its co-founders. Known informally as “genetic scissors”, CRISPR technology brings the ability to cut, replace, and edit genes to the scientific research community. By developing this technique, the company has opened the door to an entirely new way of developing treatment options for a variety of diseases. These new options are changing the way we look at diseases, and the potential to completely eliminate inherited diseases is now becoming a real possibility.

What is CRISPR Therapeutics (CRSP)

The company is named after CRISPR, short for clustered regularly interspaced short palindromic repeats. CRISPR is a type of DNA sequence that detects and destroys bacteriophage, the portion of viruses that are responsible for replicating and infecting the body. The company is named after CRISPR because one of its co-founders, Emmanuelle Charpentier, was the pioneer behind developing a way to use CRISPR in a scientific setting to edit genetic material. Charpentier, along with colleague Jennifer Doudna, was able to decipher this molecular behaviour in bacteria and apply the lessons learnt from observation into developing a genetic editing tool. By isolating this mechanism found in bacteria, the two scientists were able to create a simple way to make cuts into DNA with ease, allowing for the addition or replacement of certain segments of genetic material. This was a groundbreaking discovery that allowed researchers around the world to easily research organisms and develop a much deeper understanding of the role DNA plays in diseases. For their contributions, Charpentier and Doudna won the Nobel Prize in Chemistry for 2020.

The company is headquartered in Switzerland and also has research and development facilities in the United States. A few short years after Charpentier began working on CRISPR in 2011, she co-founded the company in 2013. The company received significant interest early on with Bayer, one of the largest pharmaceutical companies in the world, investing in the company. Since then, the company has grown to over 300 employees and IPO’d in 2016. The company has developed several therapeutics using CRISPR that are in various stages of the clinical trial process.

Why does CRISPR Therapeutics (CRSP) Matter?

The CRISPR gene-editing platform is a revolutionary method with the potential to address a number of serious diseases. Several breakthrough medicines are currently undergoing clinical testing. Furthest along the path towards regulatory approval is the company’s Transfusion-dependent β-Thalassemia (TDT) and Sickle Cell Disease (SCD) treatment, which addresses a serious blood disease with high mortality rates. The aim of this treatment is to mimic a genetic variation in certain individuals that result in them having reduced or no symptoms when diagnosed. By creating this variation in patients who are suffering from SCD and β-Thalassemia, symptoms have been shown to be reduced. This group of diseases, grouped under the umbrella of hemoglobinopathies, usually require a lifetime of treatment, including blood transfusions and frequent hospitalizations. Annually, 360,000 are born with these diseases, although not all cases are severe enough to warrant lifelong treatment. The goal of this CRISPR-developed therapeutic is to reduce the fatality rate and make the disease more manageable for patients. While SCD and β-Thalassemia treatments are the furthest along in clinical trials, the company also has several other promising candidates in the pipeline.

CRISPR Therapeutics (CRSP) Prospects

The current work on SCD and β-Thalassemia is just one very small but promising way that the CRISPR technology can be applied to medicine. In the U.S and Europe alone where clinical trials are being conducted, the number of potential patients that would benefit from this treatment as it stands right now is over 30,000. These are only the most severe cases, and CRISPR Therapeutics estimates that once the treatment regimen becomes more simplified, those who have milder cases will also seek treatment. This would bring the total number of patients to over 160,000 in the U.S and Europe alone. If approved by regulations in the EU and the United States, it is likely that the drug will be adopted in other countries as well. The rates of this disease within the population are much higher in places like Africa, and widespread distribution would have a large effect on populations around the world.

While these statistics are impressive, SCD and β-Thalassemia are just one of many diseases that CRISPR technology has the potential to treat. At various other stages of the development process are CRISPR-based regimens to treat diseases such as cancer, diabetes and cystic fibrosis. Of the ongoing trials, close to half are collaborations with Bayer and other large companies. This allows the company to reduce risk upon themselves and also lends CRISPR Therapeutics a wealth of knowledge and expertise. Other partnerships include American companies Viacyte and Vertex Pharmaceuticals. At one point, there was speculation that Vertex was interested in merging with CRISPR, news that resulted in a spike in stock price. With a talented scientific team and cutting-edge research being conducted by CRISPR, the company has captured the attention of the industry, and the long-term potential of the company remains highly promising to investors.

Where to Buy CRISPR Therapeutics (CRSP)?

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Summary

CRISPR technology is one of the most exciting and promising scientific discoveries in recent memory. By creating a way to study and change DNA in a precise manner, the ability to cure inherited diseases is closer to becoming a reality. With a talented scientific team, the company is very far along the drug development process and has several promising therapeutics in the testing and approval process. Along with their connections to major pharmaceutical companies, CRISPR Therapeutics has all the tools in place to become a major player in the space.



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