Shares of Editas Medicine (EDIT 2.28%) were jumping 10.5% as of 3:26 p.m. EST on Tuesday. This marked the second consecutive day of strong momentum after the biotech presented new data for its experimental gene-editing therapy EDIT-301 at the American Society of Hematology (ASH) annual meeting last weekend.
It's understandable why investors continue to be bullish about Editas' prospects. The company's update at ASH highlighted promising preclinical data for EDIT-301 in treating sickle cell disease with high levels of gene editing and reduction in the sickling of cells. Editas also reported good news about its manufacturing process for the gene-editing therapeutic candidate, stating that the large-scale process was "shown to be consistent and robust."
These are only preclinical results, though. Editas has plenty of hurdles to jump before it will have a shot at winning regulatory approval for EDIT-301 in treating sickle cell disease or the related genetic blood disorder beta-thalassemia.
Still, there's a reason for cautious optimism. Editas Medicine chief scientific officer Charles Albright noted, "If these preclinical results translate to the clinic, we believe our editing approach may yield a safer and more effective medicine, addressing a significant unmet need for a transformative, durable treatment with the potential to transform the lives of people living with sickle cell disease and beta-thalassemia."
Editas remains on track to submit a filing to the Federal Drug Administration (FDA) by the end of this year to advance EDIT-301 into a phase 1/2 clinical study. Its lead candidate, EDIT-101, could provide another catalyst for the biotech stock in the not-too-distant future. Editas is evaluating the gene-editing candidate in a phase 1/2 study targeting the treatment of rare genetic eye disorder Leber congenital amaurosis type 10 (LCA10).