FRANKFURT, Germany — Even as other cutting-edge genetic therapies for sickle cell disease move closer to approval, the CRISPR company Editas Medicine believes there is still a need and a commercial opportunity for its treatment, which remains further behind in development.
On Friday, Editas offered a preliminary update on the handful of patients who’ve received its gene-editing-based therapy for sickle cell, which is also being tested for another blood disorder, beta thalassemia. The company reported that the first four patients to get the one-time CRISPR therapy saw promising improvements in their levels of a particular blood protein, and had been living free of the dire pain crises their condition causes.
The results, which also showed no serious adverse events tied to the therapy, Edit-301, were announced coinciding with the European Hematology Association’s annual meeting in Frankfurt.
About the Author Reprints
Exciting news! STAT has moved its comment section to our subscriber-only app, STAT+ Connect. Subscribe to STAT+ today to join the conversation or join us on Twitter, Facebook, LinkedIn, and Threads. Let's stay connected!
To submit a correction request, please visit our Contact Us page.