Full-Spectrum Blog

Being full spectrum not only refers to using the technology for in vivo and ex vivo therapeutic applications. It also encompasses our mission to create a toolbox of technology enablers and solutions — from various gene editing and delivery modalities to development capabilities.

With Intellia’s February 28 announcement, highlighting the latest data from our Phase 1 clinical trial of NTLA-2001 for the treatment of ATTR amyloidosis, we are seeing CRISPR’s potential as a one-time treatment for genetic diseases. Fifteen individuals with their own unique lives and stories are participating in Part 1 of our first-in-human Phase 1 study. And initial clinical test results suggest that relevant aspects of their disease have been significantly impacted.

A year ago, when we laid out Intellia’s strategic priorities for 2021, we announced our goal of evaluating the initial clinical profile of NTLA-2001, the first-ever systemically delivered CRISPR therapy dosed in a patient. Even though the interim clinical data announcement happened in June last year, I’m still struck by how much it changed everything – not just for Intellia, but for the field of genome editing and even the trajectory of medicine.

This week, we entered a new era of medicine. Intellia Therapeutics, the company I lead, announced the first clinical data in history suggesting that we can harness the genome editing technology CRISPR to precisely edit target cells within the body to treat — and potentially cure — a genetic disease.

The year was 2000, and I was part of an advance crew scouring different parts of Africa in search of regions where we could – realistically – make Abbott’s HIV treatments readily available. I will never forget how village elders were left to care for their grandchildren. These kids’ parents, a whole generation of middle-aged adults, had died from HIV/AIDS.