January 13, 2022

The Difference a Year Makes

John Leonard, M.D.
President and Chief Executive Officer
Intellia Therapeutics

A groundbreaking announcement

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.

Intellia made the announcement with our collaborator Regeneron on June 26 with a presentation at the annual meeting of the Peripheral Nerve Society and a paper published in the New England Journal of Medicine. The news regarding our investigational treatment for transthyretin (ATTR) amyloidosis was groundbreaking because it showed both that our lipid nanoparticle delivery system could carry the gene-editing machinery where it needed to go – in this case the liver – and that CRISPR technology could be harnessed as an in vivo treatment to potentially cure genetic diseases.

Another eventful year

Looking ahead, I believe 2022 can be just as eventful as 2021 for CRISPR genome editing.

There is a great future ahead of patients in terms of slowing, stopping and reversing diseases with genetic origins. And because most diseases have some genetic component, the possibilities are far-reaching.

The genome editing revolution has begun

Those may sound like far-off dreams, but it took less than 10 years for CRISPR genome editing to go from scientific discovery to medical application. For those of us who dedicate ourselves to turning this revolutionary science into potential benefits for patients, that decade felt like a long time.

But once a transformative technology finds its footing, something that might seem like science fiction can then become a reality. Consider the seemingly endless acceleration of digital technology since the development of semiconductors in the middle of the 20th century. The explosion of computing power gave us an abundance of information that has revolutionized our everyday lives with search engines, video conferencing, and ridesharing. In a similar way, advances in genome editing have the potential to deliver a new era of medicine that can transform our relationship to potentially treating disease. We don’t know exactly what that will look like yet, but for some conditions, a single treatment might be able to prevent years of treatment burden, physical suffering and shortened lifespan.

While this genomic medicine revolution is just starting to cross the radar for the general public, at Intellia, we live it every day.