A CRISPR Moment in Time
When I graduated medical school in 1983, if someone had asked me then, “What do you think the medical field will look like in 40 years?” I couldn’t have imagined the possibility of where we are today. The idea of editing DNA to possibly treat someone of a rare, genetic disease would have seemed unreachable.
This is what science is all about. There’s the day before the insight, and then there’s the day after. With the discovery of CRISPR in 2012, I feel fortunate to have witnessed both sides of time. Fast-forward 10 years later, Intellia has already demonstrated proof of concept that underpins the true promise of this technology.
Assembling the puzzle
From the very beginning, Intellia sought out to create a genome editing platform that could be built upon over time. With each data readout from our programs, we have started to put the pieces of the CRISPR puzzle together. Now, the picture of what could be possible with genome editing is starting to take shape.
Intellia’s September 16 announcement was twofold:
It was the first time we shared interim data from our Phase 1/2 study of NTLA-2002 for the treatment of hereditary angioedema. And, for the second time, we demonstrated we can precisely edit target cells in the body with a single dose of a CRISPR-based therapy. These data also highlight the modularity of our platform to target different genetic diseases.
We also shared initial results from the cardiomyopathy arm of our Phase 1 study of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis. These data showed a profound reduction in serum TTR levels in patients with this manifestation of ATTR, giving rise to our belief NTLA-2001 may potentially help those living with not only the neurological manifestations of ATTR, but also the more common cardiac manifestations.
Beyond beginner’s luck
There is no magician behind some curtain, and this is not beginner’s luck. The promise of CRISPR technology is real, and Intellia’s early successes can be attributed to our rigorous preclinical work and the support of our world-class team. As we continue to advance our science, we will look for opportunities to reach broad patient populations.
Also, we wouldn’t be able to share these data without the individuals who enrolled in our clinical studies and embarked on this journey with us. As for my fellow colleagues, no single achievement of ours has come without working together as one team. We all have our roles and responsibilities, which add up to be something quite special. Together, we’re charting a new path forward in the field of medicine, and I’m humbled to be part of 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 and NTLA-2002; the modularity of our platform to target different genetic diseases; our ability to advance product candidates and to develop and commercialize a pipeline of CRISPR-based therapies to reach broad patient populations; and its ability to replicate or apply results achieved in preclinical or clinical studies to advance its clinical programs, including NTLA-2001 and NTLA-2002. 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 and NTLS-2002; 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.