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Vertex exec says prices for gene therapies are ‘in the ballpark’



Rise and shine, everyone, another busy day is on the way. And this is especially true for us, since we hit the proverbial pause button yesterday to observe ancient rituals. Now, though, we have returned and there is much to do. To cope, yes, we are firing up the coffee kettle to brew another cup of stimulation. Our choice today is Jack Daniels. And yes, this is made available by the same folks who operate the distillery. Please feel free to join us. Meanwhile, here is the latest menu of tidbits to help you on your own journey, which we hope is pleasant and productive. So time to get cracking. Have a wonderful day, and do keep in touch. We are always happy to make new pen pals. …

Multimillion-dollar prices for a rival’s treatments with the potential to cure rare diseases are “in the right ballpark,” according to Stuart Arbuckle, chief operating officer at Vertex Pharmaceuticals, which is preparing to market its own treatment for sickle-cell disease, Bloomberg News tells us. Even a drug-price watchdog, the Institute for Clinical and Economic Review, deemed the $2.8 million tag as cost effective for a Bluebird Bio drug called Zynteglo for the blood disorder beta thalassemia, Arbuckle said in an interview. Bluebird also recently said it would charge $3 million for Skysona, the gene therapy it just launched for a rare brain-wasting disease.

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