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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