Gene Editing with CRISPR Technology: Keeping the Focus on Patients

Ever since a proof-of-concept paper was published in 2012, the gene-editing technique CRISPR-Cas9 has been making waves as a major scientific breakthrough—one that has captured the imaginations of the scientific community and public alike.

Amid patent wars, China’s first use of CRISPR in patients, and even a fictional bioterror drama produced by Jennifer Lopez, the story of CRISPR is unfolding before our eyes, with each headline capturing its transformation from a sci-fi fantasy to a ground-breaking tool that can change lives.

But with all the excitement comes a great deal of trepidation. For a tool that’s so new and has such a breadth of applications, there’s still a lot we don’t know about its safe and ethical use.

While CRISPR’s implications on society will be a subject of ongoing debate, the technology is currently being focused on helping patients with chronic diseases for which there is no cure or treatment. Simply put, CRISPR is opening new doors for therapeutic options.

We had the opportunity to attend a talk presented by Intellia Therapeutics, a company that believes CRISPR has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course.

Nessan Bermingham, PhD, Intellia’s CEO and co-founder, and John Leonard, MD, Intellia’s EVP, R&D, gave an overview of the company’s mission and latest data at a presentation hosted by MATTER, Chicago’s incubator for healthcare and biotech start-ups, on Wednesday, March 1.

In August, Intellia took one step closer to its goal of leveraging CRISPR technology to create a new class of therapeutic products by publishing compelling preclinical data that demonstrated progress in in vivo gene editing. According to the study, lipid nanoparticles (tiny fat globules) can be used to deliver CRISPR/Cas9 components to liver cells in mice, allowing editing of target DNA within the body.

Building on the preclinical data, the company expects to begin clinical studies on patients as early as 2018. Dr. Bermingham said obtaining preliminary data from patients will allow the company to develop treatments quickly. The pace of discovery enabled by CRISPR is nothing short of extraordinary, he noted. If all goes to plan, it would take just six years to advance an observation published in a scientific journal to a life-saving treatment delivered to patients.

His colleague, Dr. Leonard, is no stranger to having a front-row-seat for scientific breakthroughs, having previously directed protease inhibitor studies at Abbott Laboratories that contributed to HIV becoming a controllable infection.

He summarized CRISPR’s potential to revolutionize life as we know it by saying, “The ability to pinpoint a specific spot in DNA is something people have dreamed about being able to do. It’s a truly revolutionary time. I’m confident the world will be different. What we will be able to do in the course of our lives will be breathtaking.”