The big jump today, though, isn't based on Editas' positive weekend update about its experimental gene-editing therapy EDIT-301. Editas announced after the market closed on Wednesday that it has submitted an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) for the initiation of a phase 1/2 clinical study evaluating the experimental therapy in treating sickle cell disease.
Editas Medicine CEO Cynthia Collins called the IND submission "a key milestone" for the company. She's right. Assuming the FDA gives a thumbs-up, EDIT-301 will become Editas' second clinical-stage gene-editing candidate.
Investors are especially excited about the potential for EDIT-301 after Editas' presentation of new preclinical data at the American Society of Hematology (ASH) on Saturday. The experimental therapy achieved high levels of gene editing in CD34+ bone marrow cells and what Editas referred to as "remarkable correction" of sickle cell disease.
It's definitely great news that Editas appears to be on track to advance EDIT-301 into clinical testing. However, it's also still really early. Most candidates that enter early stage clinical testing don't make it all the way to winning regulatory approvals.
Editas has already selected a lead principal investigator for its anticipated phase 1/2 study of EDIT-301 in treating sickle cell disease. The company has also lined up a clinical research organization to help with the study.
The next catalyst for the biotech stock, though, could come from another pipeline candidate. Editas is evaluating experimental gene-editing therapy EDIT-101 in a phase 1/2 study for treating Leber congenital amaurosis type 10 (LCA10), a leading case of inherited blindness.