STAT+: Pharmalittle: CRISPR-based drug shows promise in preliminary study; FDA user fee program prompts fresh worries over conflicts
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 still in the planning stages, but we expect tidy up the Pharmalot grounds and promenade with the official mascot. We may even catch up on our reading. And what about you? This remains a fine time to enjoy the great outdoors — a walk in the park is good for the mind and body, after all. You could make some holiday purchases before prices rise still further. Or you could take a moment to reach out to someone special. Well, whatever you do, have a grand time. But be safe. Enjoy, and see you soon …
Intellia Therapeutics reported that the first six patients to receive its CRISPR-based treatment for a genetic swelling disorder have safely had small, corrective changes made to dysfunctional DNA inside their liver cells, STAT writes. Preliminary results from the study — just the second to show that CRISPR-based gene editing can be delivered systemically and performed inside the body — found the treatment, NTLA-2002, reduced levels of the disease-causing protein, kallikrein, by 65% and 92% in the low- and high-dose cohort, respectively. What NTLA-2002 is starting to show is the promise of a platform technology to address any genetic disease where there is a liver-expressed protein.
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