Beta thalassemia isn’t something you read much about in the medical lay press, at least until recently. Part of the problem is the lack of approved therapies, as well as the dearth of new products being evaluated in this condition. It’s also more common in the Mediterranean, Middle East and Asia compared to the US, where it’s medical cousin, sickle cell anemia, dominates.
Things are changing on the horizon though with the advent of new approaches in gene therapy and gene editing, which have enabled new compounds to be developed that strike right at the source of the problem – mutated genes – rather than tackle the symptoms associated with the complications that can arise.
As such, this new approach is potentially transformative and therefore of great medical interest.
In the first of our two part series, we take a look at what the clinical impact of treating thalassemia patients really means and what’s happening next in sickle cell disease in a interview with Dr Alexis Thompson, the PI for the Bluebird Bio Northstar trial with lentiglobin that was presented at ASH last month.
Her perspectives offer a fascinating insight into this novel therapy now that the first 4 patients have been successfully treated.
In the second part of the series later this week, we will also take a look at Bluebird Bio the company, and their approach to gene therapy with lentiglobin and CAR T cell therapy with an interview with their CEO, Nick Leschly.
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