Tag Archives: (NTLA)

One Infusion To Rule Them All?

In 2020, for the first time in history, a doctor pushed a CRISPR-based drug into a vein, let it ride the bloodstream like an Uber through downtown physiology, and watched it edit a gene inside the liver in real time. That experiment – Intellia’s (NTLA) NTLA-2001, now dubbed nexiguran ziclumeran, or nex-z – delivered an […]

Turns Out, The Cure Was The Easy Part

I met a young woman in Geneva last year. Mid-20s, poised, bright. She had sickle cell disease and told me her childhood had been a blur of hospital beds and missed school years. Then she mentioned something I didn’t expect. She was part of a Casgevy clinical trial. No crises in over two years. She […]

A Double Helix Of Opportunity

I never fully appreciated the potential of gene therapy until last fall when my college friend Eric called with surprising news. His 14-year-old daughter Sophie, who’d struggled with sickle cell disease her whole life, had undergone treatment with Casgevy, a CRISPR-based gene therapy developed by CRISPR Therapeutics (CRSP) and Vertex (VRTX). Six months later, she […]