Three years ago, the idea of editing genes inside the human body seemed like science fiction to many. Sure, it sounded exciting, but would it really be possible? When I think about the journey since then — and where we are today — it’s truly remarkable.
On March 2, the FDA cleared our IND application for NTLA-2002, Intellia’s in vivo CRISPR-based investigational therapy for the treatment of hereditary angioedema (HAE). This clearance allows us to include U.S. patients, for the first time, in our clinical development program. When I first learned the news, it was a mix of joy and surprise as we weren’t expecting to hear until the next day. I was happy for our team and incredibly proud of everything we accomplished.
Although I’ve worked in drug development for many years and have witnessed numerous milestones, what I felt at that moment was different. The emotion was greater than anything I’ve ever experienced in my career. I think it’s part and parcel of everything that led up to the IND clearance and the extraordinary team that made it happen.
We have great technology and great science, but most importantly, we have an exceptional team.
Being a Part of Something Big
We have great technology and great science, but most importantly, we have an exceptional team. We mesh well. We challenge each other. But also, we respect one another.
When we were putting the IND application together, it was all hands on deck. From a cross-disciplinary perspective, everyone did what they had to do — selflessly. It’s part of who we are as individuals, the culture here at Intellia, and a testament to our senior leadership team, who not only empowers and challenges us, but also trusts and supports us.
There are few places I’ve felt that. It’s like a family.
Putting Patients First
Planning and executing a clinical trial is not easy. We still have a way to go, and inevitably there will be challenges in completing our development plan, but I am confident in our team. There will be ups, and there will be downs. And during the down times, it’s the patients that will help push us through.
Back in September, at the Bradykinin symposium in Berlin, we met with a couple of the investigators leading our trial outside of the U.S. I felt compelled to express my gratitude. There’s no way we’d be here without those patients. If you ask me, they are the heroes in this ongoing story. They took a chance, having been the very first patients to volunteer to participate in the first-in-human study of NTLA-2002. And now, we can’t wait to begin the next chapter of our story, as we work to expand enrollment and advance the program.
This blog post 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 its clinical program for NTLA-2002 for the treatment of hereditary angioedema, including its ability to expand enrollment and advance the program. Any forward-looking statements in this blog post 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; and risks related to the development and commercialization of its product candidates, including NTLA-2002. 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 accurate as of the date of this article, and Intellia undertakes no duty to update this information unless required by law.