An approval filing for a breakthrough treatment of sickle cell disease should be done by March,
Crispr Therapeutics
said, highlighting the company’s lead in a competitive area of medical research and triggering a gain in the stock.
If approved, the exa-cel treatment from Crispr (ticker: CRSP) and its partner
Vertex Pharmaceuticals
(VRTX) would be the first U.S.-marketed therapy based on Crispr—a Nobel-winning technology that rewrites faulty genes. Patients who received the one-time treatment in clinical trials have remained free of the red blood cell disorder‘s agonizing symptoms.
Source: https://www.barrons.com/articles/crispr-therapeutics-stock-fda-sickle-cell-gene-therapy-bf56a18c?siteid=yhoof2&yptr=yahoo