What happened
Shares of Editas Medicine (EDIT 0.37%) were soaring 15.4% higher as of 11:05 a.m. EST on Monday. The big gain came after Editas presented new data for its gene-editing candidate EDIT-301 at the American Society of Hematology (ASH) annual meeting held virtually on Saturday.
So what
The presentations at the annual ASH meeting often provide catalysts for quite a few biotech stocks. The good news for EDIT-301 certainly warranted a nice bump for Editas' shares.
Image source: Getty Images.
One of the challenges of gene editing is editing enough cells to make a difference in treating a disease. Editas announced that high levels of editing in red blood cells obtained from patients with and without sickle cell disease were achieved in preclinical testing. Most importantly, this led to robust induction of fetal hemoglobin and resulted in what the biotech said was "remarkable correction" of sickle cell disease in cells.
Another challenge for gene-editing therapies is making them in large quantities. Editas stated that its "large-scale manufacturing process was shown to be consistent and robust."
Now what
Editas is well behind CRISPR Therapeutics and its big partner Vertex Pharmaceuticals in the development of a gene-editing therapy for sickle cell disease and a related genetic blood disease beta-thalassemia. However, Editas thinks that EDIT-301 could be the best in its class, with a safer and more effective approach. The biotech expects to file for FDA approval by the end of this year to advance EDIT-301 into a phase 1 clinical study in treating sickle cell disease.
