Vertex Pharmaceuticals (NASDAQ: VRTX) is the leader in cystic fibrosis (CF) treatment -- and it expects to hold that title until at least the late 2030s. There's a lot to love about the company's position and the resulting billions of dollars in annual revenue. Still, investors' main worry has been this: Can Vertex succeed beyond CF?
The biotech company may be about to prove that, yes, it can. Vertex CEO Reshma Kewalramani made a statement about one of the company's most innovative, exciting programs during the recent earnings call. And her words mean Vertex soon may have another billion-dollar therapy to sell.
Image source: Getty Images.
Work with CRISPR
Vertex is working on CTX001, a gene-editing therapy, with partner CRISPR Therapeutics (NASDAQ: CRSP) for blood disorders beta thalassemia and sickle cell disease. Beta thalassemia and sickle cell disease both hamper the ability of hemoglobin to deliver oxygen throughout the body. CTX001 involves the altering of a patient's stem cells to make a high level of fetal hemoglobin, an oxygen-carrying form of hemoglobin that we have at birth.
Based on conversations [with regulators] to date, we believe regulatory submissions for approval of CTX001 may be possible in the next 18 to 24 months, CEO Kewalramani said during the earnings call last week.