(Bloomberg) -- Two new cutting-edge therapies for sickle cell disease were approved in the US Friday, paving the way for costly treatments for a chronic and at-times debilitating disease that affects some 100,000 Americans.Most Read from BloombergApps That Use AI to Undress Women in Photos Soaring in UseCarlyle’s Rubenstein Is in Talks to Acquire Baltimore OriolesHunter Biden Indicted in Tax Case as White House Woes MountThe Record Rush to Buy a Rolex or a Patek Philippe Is OverAmericans Rush to
BOSTON & ZUG, Switzerland, December 08, 2023--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) announced today that the U.S. Food and Drug Administration (FDA) has approved CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 genome-edited cell therapy, for the treatment of sickle cell disease (SCD) in patients 12 years and older with recurrent vaso-occlusive crises (VOCs). This approval means that for the first time, approximately 16,000 patients
Regulators on Friday approved two gene therapies for sickle cell disease that doctors hope can cure the painful, inherited blood disorder that afflicts mostly Black people in the U.S. The Food and Drug Administration said the one-time treatments can be used for patients 12 and older with severe forms of the disease. One, made by Vertex Pharmaceuticals and CRISPR Therapeutics, is the first approved therapy based on CRISPR, the gene editing tool that won its inventors the Nobel Prize in 2020.