There’s a tremendous global unmet medical need for new effective treatments that could potentially result in transfusion independence.
Earlier this week we published an interview with Dr Alexis Thompson, the PI for the Northstar clinical trial of lentiglobin in beta-thalassemia that was presented at ASH last month.
The story continues with an interview with bluebird bio CEO Nick Leschly in which he discusses in more detail how their “one time” treatment could potentially be transformative.
He says, “we have to figure out a way to get this in the hands of global regions, because thalassemia and sickle cell together are the most common genetic disorders in the world.”
Whether countries that aren’t as wealthy as the US can afford novel gene therapy treatments is a big question, it will be interesting to see how that pans out, but it’s exciting to have potentially transformative new treatments in development.
Leschly says the mission of Bluebird bio is “making hope a reality.” It’s a compelling vision that he’s well on the way to pulling off with lentiglobin.
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