With news of the emerging biotech company, Sionna, coming out of stealth yesterday, there is, once again, a reinvigorated hope for more transformational cystic fibrosis medicines on the horizon. In case you missed it, Sionna is attempting to correct CFTR with a small molecule drug like Vertex’s class of modulators but is targeting a different...
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Gene therapies are all the rage these days in the world of emerging biotech. It’s not hard to understand why. It has never been cheaper (on a giant scale) to understand human genetics and the human genome. We’re also living at a point in time where technologies to deliver whole genes or “correct” existing genes...
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The Cystic Fibrosis Foundation has started off the new year with a handful of new investments that suggest the cystic fibrosis market is accelerated, and not decelerating as some had feared. Not long after Trikafta was approved by the FDA, the world rightly celebrated such an important milestone in the history of biomedical innovation. Cystic...
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Last week we celebrated news out of the North American Cystic Fibrosis Conference that median age of survival (remember, the age beyond which we expect 50% of people with CF born today to live, under the assumption that recent age-specific mortality rates will hold for the rest of their lives) climbed to 50 years old,...
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My timelines are on fire with the wonderful news out of the North American Cystic Fibrosis Conference that Median Predicted Survival in cystic fibrosis has been raised to 50 years old thanks to a hell of a lot of progress that our condition has made over the past several years. It’s not lost on me...
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