Today we celebrate a game-changing tool in research and medicine. Ten years ago, one of Intellia’s founders, Dr. Jennifer Doudna, and her collaborator, Dr. Emmanuelle Charpentier, published a paper in Science, detailing the power of CRISPR/Cas9 to edit strands of DNA at precise locations.
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.