Below is a statement from Eric Kmiec, Ph.D., founder and executive director, ChristinaCare Gene Editing Institute, regarding the expected approval by the Food and Drug Administration of exa-cel (Casgevy), the CRISPR-driven treatment for sickle cell disease and betta thalassemia. If you wish to interview Dr. Kmiec, please contact Mak Sisson, Makenzie.sisson@christinacare.org,302-623-5306 or Anna Chen, achen@burness.com, 215-262-7670.
“As scientists, the fact that we have arrived at a potential curative treatment for sickle cell disease in a relatively brief period is a testament to the power of resolute researchers in this field who have never stopped. And the FDA’s expected approval comes with many firsts. For the first time we have what appears to be a safe and curative treatment for one of the most painful diseases that cuts life short. And it is remarkable that finally we are focusing on the Black population first, who are most affected by this disease. This priority is long, long overdue.
The challenge, however, is the very people we want to help may not be able to get access to or afford the million-dollar treatment and the length of time it will take to be treated — weeks and weeks in the hospital. The numbers of people who can be treated are limited. We must work with the health care industry and pharmaceutical companies who will market produce and deliver the treatments to make sure that all people can get access. What can they do to make treatments more affordable and more available? What can they do to support continued research to assess the long-term effects this treatment may have? And how can we make this easier to deliver?
As important as it is to have developed this new treatment, right now we must do our best to communicate well to the public what new findings like this mean. Take the time to explain it all. It cannot be oversold. We must make the communication about this first CRISPR-driven treatment as important as the science itself.”
Eric B. Kmiec, Ph.D., is the founder and executive director of the Gene Editing Institute at ChristianaCare. He is also co-founder and chief scientific officer of CorriXR Therapeutics. Widely recognized for his pioneering work in the fields of molecular medicine and gene editing, Dr. Kmiec has developed CRISPR based genetic therapies for Sickle Cell Disease and Non-Small Cell Lung Cancer. He is Editor-in-Chief of the journal, Gene and Genome Editing.
