CRISPR Therapeutics treats first patient with gene editing tech; Amicus nabs 'breakthrough' status for Pompe drug
→ The long awaited first-in-human testing of CRISPR gene editing tech — if you skip past the recent controversial case in China — has arrived. CRISPR Therapeutics is claiming the honor with the dosing of its Vertex-partnered drug, CTX001, in a patient with beta thalassemia. The biotech plans to treat one other patient in the Phase I/II open-label trial before opening enrollment up; it is also planning to treat a sickle cell disease patient with the drug in mid-2019 for a separate trial. Intellia and Editas are fast on its heels.
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