Cure for Sickle Cell

A heartfelt applause goes out to my dear friend, David Altshuler and his team at Vertex Pharmaceuticals. David (EVP, Chief Scientific Officer at Vertex) presented the remarkable efficacy of Casgevy, a revolutionary CRISPR-based treatment, to the FDA last November and it was approved!  Sickle cell afflicts about 100,000 people in the US, including an estimated 1 of every 365 Black babies born and has long been considered neglected by the pharmaceutical industry. Casgevy eliminates the debilitating  pain of this genetic mutation.  The next drug in the works at Vertex is a painkiller that doesn’t cause addiction.