In particular, Editas confirmed that, together with strategic partner Allergan and following the U.S. Food and Drug Administration's approval of the company's investigational new drug (IND) application for its primary EDIT-101 drug candidate during the quarter, it expects to enroll 10 to 20 patients in a phase 1/2 open-label, dose-escalation study during the second half of this year. The study will evaluate EDIT-101 for treating Leber Congential Amaurosis type 10 (LCA10). LCA10 is the leading cause of childhood blindness.
Assuming the study progresses as planned, it will make EDIT-101 the first-ever in vivo, or editing inside the body, CRISPR medicine administered to patients.
But EDIT-101 isn't its only drug candidate in the pipeline. Editas expects to start IND-enabling activities for another experimental medicine candidate to treat sickle cell disease and beta-thalassemia this year. At the upcoming 22nd Annual Meeting of the American Society of Gene & Cell Therapy, the company also plans to present in vivo proof-of-concept data for a CRISPR gene-editing approach for Usher syndrome 2A. And under a collaboration with Juno Therapeutics, Editas is increasing its 2019 investments in engineered T-cell medicines to treat cancer.
Of course, Editas has yet to achieve sustained profitability in these early stages of its business. But the company ended 2018 with cash, cash equivalents, and marketable securities of $369 million, enough for at least two years of operating expenses and capital expenditures without the need for raising additional funds.
In any case, with Editas Medicine as close as it's ever been to the commercialization of its primary EDIT-101 drug candidate, it's no surprise to see the stock soaring today.