A Milestone in Science and Medicine

November 19, 2019 12:00 pm

A Milestone in Science and Medicine

Today we announced the first data from our CRISPR gene-editing program for sickle cell disease and transfusion-dependent beta thalassemia. These early results are remarkable and speak to the potential that our investigational CRISPR/Cas9-based gene-editing therapy has for people with sickle cell disease and beta thalassemia.

Seventy years ago, Linus Pauling and colleagues described sickle cell anemia as the first “molecular disease.” Today, for the first time, we are testing one of the first true molecular medicines for its treatment. As a physician-scientist who has spent more than 40 years in science and medicine, it’s exciting to consider what a combination of remarkable scientific innovation and the willingness of companies to take risks and invest in this cutting-edge technology could provide for people with these devastating diseases.

When I started my career in medicine, I remember the frustration of taking care of patients for whom no effective treatment was available. As a cardiologist, I cared for countless heart attack patients for whom we lacked adequate prevention or therapy. I had to tell patients that an HIV infection was likely a death sentence. But over the course of my career, I have been fortunate to witness the discovery of statins, implantable defibrillators and drug-coated stents for the treatment of heart attacks and the development of highly effective triple combination antiretroviral therapies for HIV. Scientific innovations such as these have dramatically improved both the morbidity and mortality of serious diseases for millions of patients around the world.

The discovery of CRISPR-mediated gene editing and now the first data from the first patients treated with CRISPR-based therapies, represent yet another extraordinary scientific innovation that holds the potential for revolutionizing the treatment of multiple diseases. These results also validate the importance of companies like ours investing in new tools and capabilities that allow us to go after diseases that were previously intractable.

As we look at these encouraging early results, I am struck by the courage and resilience of the people who volunteer to participate in these clinical studies. They are pioneers and their bravery will help so many people today and, in the future, as we continue to explore the full potential of this therapy and technology.

While incredibly encouraging, it’s important to remember that it’s still early days in these clinical programs. There is more work to be done — together with physicians, patients, caregivers and the scientists behind this potential therapy — to further validate these results and ultimately reach our goal of bringing forward the best possible therapy for people with these devastating diseases. We look forward to updating you on our progress along the way.