April saw a wide range of exciting news in gene therapy clinical trials — let’s dive right in.
Beta-thalassemia was the subject of some promising clinical trial results in April. A gene therapy in development by bluebird bio modifies blood stem cells to replace the problematic gene behind the disease. In a phase 1/2 study of the treatment, researchers reported that 15 of 22 patients were able to significantly reduce or eliminate their reliance on blood transfusions as a result. An estimated 1 in 100,000 people have beta-thalassemia worldwide, according to the National Organization for Rare Disorders, and the company plans to seek European approval of the treatment by the end of the year.
In other gene therapy news, Chicago-area biotech AveXis, which is in the process of being acquired by Novartis, in April released its first data from a key clinical trial. The STR1VE trial is testing a one-time gene therapy called AVXS-101 as treatment for spinal muscular atrophy type 1, a progressive and often fatal genetic disease. AveXis told attendees at the American Academy of Neurology meeting that all six patients dosed more than a month prior had met the study’s goals of event-free survival and had seen improved motor function as well, Xconomy reported. No serious side effects have yet been seen. Following on the heels of those data, the company announced that it had dosed its first patient in a phase 3 clinical trial testing the same drug for pre-symptomatic SMA of various types.
And last, but not least, on the gene therapy front, the FDA has given the green light to the first clinical trial of gene therapy for glycogen storage disease, reports the Hartford Courant. The rare disease occurs when the liver fails to break down the body’s glycogen into glucose, leaving blood sugar dangerously low. California-based biotech Ultragenyx is developing a single-dose gene therapy, DTX401, designed to replace the deficient enzyme in the liver. The phase 1/2 trial may begin enrolling patients as soon as this month.
Now, switching gears to cell therapies, a panel event hosted by Alliance for Cancer Gene Therapy was the occasion for some interesting reflections by leaders in the field, as BioPharma Dive reported. Carl June, the scientist who led the University of Pennsylvania lab that developed Novartis’ Kymriah, pointed to some developments that may be on the horizon for CAR-T therapies, including the ability to “turn off” the cells when they’re no longer needed in the body. Additionally, June said, CAR-T’s biggest challenge will be in tackling solid tumors — but combinations with checkpoint therapies, or using CRISPR technology to knock out the checkpoint inhibitor, could be ways to overcome that challenge.