Editas Medicine has treated the first patient in a trial of its experimental gene editing treatment for sickle cell disease, a step forward in the company’s development of CRISPR-based medicines.
The treatment, which consists of genetically modified stem cells, successfully took root in the patient’s bone marrow following transplant, Editas said Wednesday. This process, known as “engraftment,” is an important first step for the treatment to work as intended.
Editas, one of several companies testing CRISPR gene editing in humans, plans to enroll a total of 40 men and women with severe sickle cell disease in the study, which is enrolling at centers in the U.S. and Canada. Preliminary data should be available by the end of the year, the biotechnology company said.
Also on Wednesday, Editas revealed the Food and Drug Administration has lifted a partial clinical hold that would have limited its ability to conduct the study.
The treatment, dubbed EDIT-301, is the second gene editing medicine that Editas has advanced into clinical testing, following a therapy for an inherited form of vision loss. Initial results for that first therapy were, after long delays, released last September.
Editas was one of the biotechs formed to transform CRISPR from laboratory breakthrough to genetic medicines. But it has struggled to advance its research as quickly as some of its peers, and has been hampered by significant executive turnover.
This year, the company appointed a new CEO, Gilmore O’Neill, and recently replaced its chief medical officer.
With EDIT-301, Editas is attempting a similar approach to rival CRISPR Therapeutics, which together with partner Vertex Pharmaceutical has advanced a sickle cell treatment that also uses gene-edited stem cells. Clinical trial results have shown promise and the companies expect to soon ask the FDA for approval.
Both treatments aim to restore production of a form of the oxygen-carrying protein hemoglobin that normally disappears soon after birth. Restoration of this protein, the thinking goes, should reduce or eliminate sickle cell symptoms.
Editas, however, is using a new kind of CRISPR component — an enzyme called AsCas12e — that it claims can edit DNA with high efficiency and specificity.