What happened

Shares of Editas Medicine (NASDAQ:EDIT) climbed 18.5% in March, according to data from S&P Global Market Intelligence, in the wake of the gene-editing leader's latest quarterly results.

The stock skyrocketed nearly 15% on March 1 alone -- the first trading day following its quarterly release -- after Editas provided an update on its financials for the fourth quarter of 2018. But that's not to say its headline numbers looked like anything special at first glance; Editas incurred a net loss of just over $25 million, or $0.25 per share, on revenue of roughly $6.1 million, with the latter still driven by collaboration and research and development deals under alliances with partners like Allergan and Juno Therapeutics.

Rather, investors were more concerned with the company's progress advancing its pipeline of novel medicines and drug candidates in these early stages of its growth.

Strand of DNA with a piece being removed

Image source: Getty Images.

So what

Editas' most exciting news concerned the FDA's approval of its investigational new drug (IND) application for the company's primary drug candidate, EDIT-101. It's designed to treat Leber Congenital Amaurosis type 10 (LCA10), the leading cause of childhood blindness. Following the approval, Editas received a $25 million milestone payment from Allergan under the terms of the companies' strategic alliance.

The next step, according to Editas, is enrolling 10 to 20 patients in a phase 1/2 open-label dose-escalation study later this year to evaluate EDIT-101's effectiveness. Assuming all goes as planned, this trial would mark the world's first in vivo, or editing inside the body, CRISPR medicine administered to human patients.

Now what

Editas also provided updates on its other earlier-stage efforts, including an upcoming presentation of in vivo proof-of-concept data for another CRISPR gene-editing approach to treat Usher syndrome 2A (USH2A). The company also plans to soon begin IND-enabling activities for another experimental medicine candidate to treat sickle cell disease and beta thalassemia. 

We can be sure, however, that in the coming quarters all eyes will turn first toward Editas' continued progress advancing toward commercialization of EDIT-101. Given its encouraging update to that end early last month, it was no surprise to see the stock jumping in response.