Gene-editing specialist CRISPR Therapeutics has been struggling to keep up with the market in the past year. However, their exa-cel potential treatment for sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT) is nearing approval, and could become a game-changer for patients suffering from these diseases.
Effective Treatment for SCD and TDT
Exa-cel has been found to be effective in treating both SCD and TDT in clinical trials, with rare adverse events. As a result, regulators are unlikely to deny approval for safety reasons unless there are very serious concerns. CRISPR Therapeutics and Vertex Pharmaceuticals plan to initially target 32,000 SCD and TDT patients in the US and Europe, creating a potential $64 billion opportunity.
Discussions with Insurance Companies and Government Programs
The two companies have been in discussions with insurance companies and government programs such as Medicaid to make the treatment accessible and affordable to patients. Although the cost may be lower than $2 million, exa-cel’s opportunity should still be enormous.
Enormous Opportunity for CRISPR Therapeutics
If we assume the $64 billion opportunity for exa-cel is somewhat accurate, CRISPR Therapeutics and Vertex Pharmaceuticals could grab 50% of this total, so $32 billion. At present, the market capitalization of CRISPR Therapeutics is only $3.6 billion. This indicates that if exa-cel earns approval, CRISPR Therapeutics’ shares could potentially jump as it takes a major share of the market.
Fairly Valued Stock
According to the author’s perspective, CRISPR Therapeutics is currently moderately priced, but there is a possibility of a surge in its stock price once exa-cel gets approved. Investors are likely watching this closely as it could be a huge breakthrough in the treatment of these diseases.
What is Sickle Cell Disease and Beta-Thalassemia?
Sickle cell disease is a group of inherited blood disorders that affect hemoglobin, the protein in red blood cells that carries oxygen throughout the body. The disorder causes the red blood cells to become misshapen and break down more quickly than normal, resulting in a shortage of healthy red blood cells. Beta-thalassemia is another inherited blood disorder that affects the production of hemoglobin. People with this condition have a shortage of red blood cells, which can lead to anemia and other health problems.
Overall, the approval of exa-cel could be a significant milestone for the treatment of SCD and TDT. With the potential to become a $64 billion market, this new treatment could also be a game-changer for CRISPR Therapeutics and Vertex Pharmaceuticals. Investors are keeping a close eye on this situation, waiting for the approval of exa-cel and its potential impact on the market.
