And so, another working week will soon draw to a close. Not a moment too soon, yes? This is, you may recall, our treasured signal to daydream about weekend plans. Our agenda is quite modest. We plan to catch up on our reader, stroll about the town with the official mascot, and hang with Mrs. Pharmalot. And what about you? There are innumerable outdoor activities from which to choose — trails to hike, country roads to drive, boats to navigate or paddle. Take your pick. Or you could shop for sundry items and stock up before prices rise still more. This may also be a fine time to reach out to someone special and catch up on life. Well, whatever you do, have a grand time. But be safe. Enjoy, and see you soon …
A Food and Drug Administration advisory panel voted unanimously in favor of approving an experimental gene therapy from Bluebird Bio, concluding that its benefits for children with a rare and deadly disorder outweigh a demonstrable risk of causing cancer, STAT reports. The medicine, eli-cel, is a one-time treatment for cerebral adrenoleukodystrophy, a genetic neurological disorder that affects young boys. On Friday, the committee will consider a Bluebird gene therapy for beta thalassemia. FDA decisions will be of existential importance to Bluebird, a one-time trailblazer in gene therapy that has recently cut deep into its business and has only enough cash to last until next year.
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