Granted, we are spoilt for choices here, but there is nothing more valuable in the human arsenal than our ability to grow on a consistent basis. You see; when an individual is able to get better under all possible situations, they, almost as a ripple effect, go on to hit some huge milestones along the way. This dynamic has already been reinforced by whatever we have achieved so far, with one huge piece of testimony coming from an idea called technology. While the reason why technology deserves a shoutout here is predicated upon its unprecedented skill-set, it also, at the same time, talks to the manner in which those skills were used. In fact, the latter factor will do a lot to give the creation a spectrum-wide presence, including a historical appearance on our healthcare sector. We deem it as historical because technology forayed into healthcare right when the sector was starting to buckle under its outdated structure. By instilling new and smarter ideas, the creation dramatically changed the stated reality, but even after doing so, it continued to improve the discipline under one capacity or the other. This dynamic, looking at a recent FDA green light, should only get stronger moving forward.
Bluebird Bio, a biotech based in Somerville, Massachusetts, has successfully bagged the FDA’s approval for its gene therapy, Zynteglo, which is designed to treat rare blood disorder beta thalassemia. Up until now, people suffering from severe forms of the said disease have had no option but to go through regular red blood cell transfusions for life. The regularity of these transfusions, like you can guess, takes a major toll on the person’s body, thus causing long-term healthcare complications. Bluebird’s Zynteglo, however, showcases a bit different approach here. Touted to be a one-time therapy, Zynteglo is constructed from the patient’s own stem cells that are usually harvested from their bone marrow. Once collected by the professional, the cells are then genetically modified within a lab setting so to produce a functional component of hemoglobin. Lastly, the resulting iteration is infused back into the patient’s body.
“After more than a decade of research and clinical development, and through the perseverance of clinicians, patients, and their families, the approval of ZYNTEGLO marks a watershed moment for the field of gene therapy. As the first ex-vivo lentiviral vector gene therapy approved in the U.S. for the treatment of people with beta-thalassemia, we are ushering in a new era in which gene therapy has the potential to transform existing treatment paradigms for diseases that currently carry a lifelong burden of care,” said Andrew Obenshain, CEO of Bluebird Bio.
According to certain reports, Bluebird tested the therapy in two single-arm open-label Phase 3 studies that had around 41 patients. Talk about results, they put-together an encouraging 89% success rate. Beyond the initial promise, the drug was also able to sustain its impact on the patient, with the longest follow-up stretching for over four years.
