Keep an eye on CRISPR Therapeutics (CRSP).
At the moment, it’s halted. But it could get explosive if the U.S. Food and Drug Administration’s (FDA) Cellular, Tissue, and Gene Therapies Advisory Committee green lights the company’s Biologics License Application (BLA) for exagamglogene autotemcel (exa-cel) for the treatment of sickle cell disease (SCD) in people ages 12 and older with recurrent vaso-occlusive crises.
Developed with Vertex Pharmaceuticals (VRTX), if exa-cel is approved, it could help treat both sickle cell and beta-thalassemia, an inherited blood disorder.
Even better, according to Vertex CEO Reshma Kewalrami, exa-cel is a “multibillion-dollar opportunity in the future,” as noted by Seeking Alpha. Plus, if approved, the company says it already has 50 treatment centers in the U.S. ready to administer the therapy, and 25 in Europe.
Success could also boost its competition including Editas Medicine (EDIT), and even ETFs, such as the Global X Genomics and Biotechnology ETF (GNOM).