CRISPR Therapeutics may have run into a hurdle for its clinical program to advance CTX001 in severe hemoglobinopathies, but the FDA is on record that it will do everything it can to get the drug back on track and headed to the finish line.
CRISPR and its partners at Vertex announced Monday morning that the FDA had awarded them the Regenerative Medicine Advanced Therapy — RMAT — designation for their program to treat severe sickle cell disease and transfusion-dependent beta thalassemia. And that should give them an open door as they hustle through the clinical program.
The move comes a little more than 5 months after researchers offered a snapshot look at positive results for the first patient in each of those 2 programs. And it follows close on the heels of an SEC filing noting that researchers here had to stop enrolling new patients due to the Covid-19 outbreak.
In the program, researchers extract hematopoietic stem and progenitor cells from patients and then re-engineer them using CRISPR/Cas9 tech. Those cells are then infused back into patients.
At the time, Vertex executive chairman Jeff Leiden heralded the snapshot results as evidence of its “potential to be a curative CRISPR/Cas9-based gene-editing therapy.”
