News briefing: Intellia hits clinic with CRISPR therapy; Vifor continues to spend, paying up to $340M for DGF program
Another CRISPR therapy has entered the clinic. Intellia Therapeutics, the Cambridge gene editing biotech, announced today that they dosed their first patient with a therapy meant to treat transthyretin amyloidosis, a rare genetic disease caused by a mutation in a liver protein.
Although a handful of CRISPR therapies have already entered the clinic at Intellia and elsewhere, the new study is notable for being just the second study to inject CRISPR directly into a patient. Other approaches, such as those meant to treat sickle cell anemia, involve taking cells out of a patient, editing them, and then infusing them back in.
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