CRISPR Therapeutics and Moderna, both innovative biotechs in rapidly evolving fields, present compelling investment opportunities despite recent stock declines. CRISPR Therapeutics (NASDAQ: CRSP) has seen its shares drop by 27% year-to-date, while Moderna (NASDAQ: MRNA) is down by 41%. However, key developments suggest strong potential for future growth.
CRISPR Therapeutics: Casgevy Approval and Market Expansion
CRISPR Therapeutics achieved a significant milestone with the approval of Casgevy, a gene-editing therapy for sickle cell disease and transfusion-dependent beta-thalassemia. This marks the first market approval for a therapy utilizing CRISPR gene-editing technology. Despite this breakthrough, the company's stock performance has lagged, potentially due to profit-taking after the approval and the time required for Casgevy to significantly impact financial results. The ex vivo nature of Casgevy, which involves collecting, modifying, and re-inserting patient cells, necessitates specialized treatment centers and a longer treatment timeline.
However, the long-term prospects for Casgevy appear promising. CRISPR Therapeutics has partnered with Vertex Pharmaceuticals to develop and market the therapy, expanding its reach to markets like Saudi Arabia and Bahrain, which have a combined eligible patient pool of approximately 23,000. The partners estimate an addressable market of 35,000 individuals in the U.S. and Europe. With a price of $2.2 million in the U.S. and limited competition, Casgevy has the potential to exceed $1 billion in sales.
Moderna: Expanding Vaccine Portfolio
Moderna, known for its successful COVID-19 vaccine, is building a robust pipeline to offset declining vaccine sales. The company has recently gained approval for its RSV vaccine, mRESVIA, and anticipates launching at least one more product in the coming years. A combined coronavirus/flu vaccine has also demonstrated promising results in Phase 3 trials, eliciting stronger immune responses compared to individual vaccines.
Moderna's pipeline includes several other Phase 3 candidates, such as a stand-alone flu vaccine, a cytomegalovirus vaccine (currently unavailable), a norovirus vaccine, and a personalized cancer vaccine developed in collaboration with Merck. These developments could serve as catalysts to boost the stock price, which has fallen by 41% year-to-date. The company's mRNA-based platform positions it well for developing a range of successful products in the long term.