I’ve been part of Intellia since its beginning in 2014, and it’s been an incredible ride so far with many people who have contributed along the way. Back then, we recognized the tremendous promise of Also called gene editing. Genome editing collectively refers to a set of technologies, including CRISPR/Cas9, that can be used to cut and modify DNA. Genome editing uses systems to make the DNA change inside the cell. These cells can be edited in the body (in vivo) or outside the body (ex vivo) from a patient or donor. and its potential to transform people’s lives. But there’s a difference between recognizing possibilities and realizing them.
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.
It’s important to note the clinical trial for NTLA-2001 is still in the early stages. We have much more to learn and prove in our development programs for ATTR amyloidosis and other diseases. For me, that is exactly what makes this moment so exciting. We are in the middle of stepping from one world into another — from turning a promising technology into a prospective medicine — and one step closer to the vision of seeing it make a difference in people’s lives.
We are in the middle of stepping from one world into another.
What will this new world eventually become? It’s hard to say for sure. Not long after I was named CEO of Intellia in 2018, I joined several other executives invited to the Vatican for a panel discussion about the medical potential of CRISPR. This was not a religious meeting, but rather a scientific discussion of sorts. After the public discussion, we joined a smaller group of church officials in a smaller room, where someone asked very simply, “What should we make of all this, and where are we headed?”
I answered with what I know — in coming decades, I predict we will see a revolutionary advancement in the treatment of whole categories of disease. As I headed home, I thought about the people whom I had just met, living and working in an ancient city. The Roman Catholic Church is two-millennia-old. Where will we be on that time scale? What is possible if scientists keep chipping away over time, expanding our ability to reprogram our own genetic software? We may be able to address the treatment and perhaps even the prevention of disease in ways we never imagined.
I predict we will see a revolutionary advancement in the treatment of whole categories of disease.
How? Well, we are only just getting started. Intellia’s work is firmly rooted in developing one-time, durable treatments for serious diseases. That alone is enough to keep us busy for decades to come. Yet, I still can’t help but wonder about this new era we are stepping into and wishing for a glimpse at where future generations will take it.
This article contains “forward-looking statements” of Intellia Therapeutics, Inc. (“Intellia”, “we” or “our”) within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellia’s beliefs and expectations regarding the safety, efficacy and advancement of our clinical programs, including NTLA-2001; its ability to demonstrate and leverage its platform’s modularity to advance its product candidates and to develop and commercialize a pipeline of CRISPR-based therapies; and its ability to replicate or apply results achieved in preclinical or clinical studies to advance its clinical programs, including NTLA-2001. Any forward-looking statements in this article are based on our expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, risks related to Intellia’s ability to protect and maintain its position and rights regarding its intellectual property portfolio; risks related to the development and commercialization of any of Intellia’s product candidates, including NTLA-2001; the risk that the results of preclinical studies or clinical studies will not be predictive of future results in connection with future studies; and the risk that Intellia will not be able to demonstrate its platform’s modularity and replicate or apply results achieved in preclinical studies to develop additional product candidates. For a discussion of other risks and uncertainties, and other important factors, any of which could cause Intellia’s actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in Intellia’s most recent annual report on Form 10-K filed with the Securities and Exchange Commission, as well as discussions of potential risks, uncertainties, and other important factors in Intellia’s subsequent filings with the Securities and Exchange Commission. All information in this statement is as of the date of this article, and Intellia undertakes no duty to update this information unless required by law.