The FDA approved gene-editing therapy Casgevy to treat sickle cell disease. Developed by Vertex Pharmaceuticals (VRTX) and CRISPR Therapeutics (CRSP), the one-time treatment harnesses gene editing advancements. Vertex CEO Dr. Reshma Kewalramani says the approach involves extracting a patient's stem cells, correcting the defect in those cells using CRISPR gene editing tools, then re-infusing the edited cells into the patient - relieving painful symptoms. Patients who undergo this $2.2 million procedure may eliminate painful sickle cell crises called VOC's. Kewalramani contrasts the cost against the $4-6 million lifetime price tag for conventional sickle cell management. For more expert insight and the latest market action, click here to watch this full episode of Yahoo Finance Live.
(Bloomberg) -- The US Food and Drug Administration approved the first gene editing therapy using Crispr technology on Friday, a collaboration between Vertex Pharmaceuticals Inc. and Crispr Therapeutics AG for treating sickle cell disease.Most Read from BloombergApps That Use AI to Undress Women in Photos Soaring in UseThe Record Rush to Buy a Rolex or a Patek Philippe Is OverApple’s iPhone and Watch Product Design Chief to Leave in Shake-UpAmericans Rush to Portugal Ahead of Changes to Expat Tax
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