Bluebird bio ($BLUE) is an emerging biotech company with a novel lentiglobin gene therapy in development that could revolutionize the treatment of beta-thalassemia and sickle cell disease.
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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San Francisco – the 2014 annual meeting of the American Society of Hematology kicks off today. Yesterday was “Super Friday” – a day when the non-profit and industry sponsored satellite symposia and other ancillary meetings, take center stage.
Each day (Sat – Mon) at the ASH meeting here in San Francisco, we we’ll be sharing information on which sessions we are in. For all those who have asked how do we get a photo with our antibuddies: @gene_antibody, we’ll mention where they are if we see them 🙂
By the way to get a photo you have to be able to identify which one is which – tip: there’s a monoclonal, bispecific, ADC and glycoengineered. Can you work out which is which from the picture? If not, it’s time to brush up on your antibody structures!
In addition, throughout the day (schedule and wifi permitting) we’ll be updating the rolling blog with short comments on the oral sessions and posters we’ve been in and what’s captured our attention. The hematology community has embraced Twitter, with many of the leading experts in the field sharing commentary and insights on their specialized area. ASH is also particularly welcoming to patient advocates who will be live-tweeting too. Expect the #ASH14 Twitter hashtag to generate a lot of information. If you’d like to share the ASH journey with us over the next 3 days, you can purchase access by clicking on the blue icon at the end of the post. Existing subscribers already know how to login. Let the meeting commence!