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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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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San Francisco – “Manic Monday” is what I call Monday at the annual meeting of the American Society of Hematology. It’s when the majority of oral presentations take place in multiple parallel sessions that require you to run between meeting rooms if you want to follow a particular drug across different blood cancers.
It’s even more challenging this year by the fact the conference is in three buildings at the Moscone Center in San Francisco. While Moscone North and South are interconnected thanks to an underground atrium, to get to sessions in Moscone West from North/South you have to go out of the building, cross one or two main roads, then go up elevators to the second or third floors. Not ideal! I think ASH is now too big for the venue.
Looking back on yesterday, it was a privilege to be in the audience when Dr Kanti Rai received a well-deserved lifetime achievement award for his work in chronic lymphocytic leukemia (CLL). A visibily moved Dr Rai was given a standing ovation by the thousands present in the plenary hall.
Expect the #ASH14 Twitter stream today to be like opening the tap to run a bath. I congratulate all the hematology experts who have shared data and commentary from sessions via social media. #ASH14 stands out in terms of expert engagement and a high signal to noise ratio.
If there was an award for best conference coverage of #ASH14 on Twitter I would nominate @drmiguelperales.
Not only does Dr Perales from Sloan-Kettering share tweets from the sessions that he is in that are accurate and informative, but he frequently offers links to relevant papers for those that want to learn more. In addition to showcasing his expertise, this is a really good way to use social media to educate and inform. I look forward to his commentary, particularly if I am in another session at ASH. A must follow on Twitter!
To the extent possible we’ll be providing updates to today’s live blog throughout the date, subscribers can login to read more or you can purchase access by clicking on the blue icon at the end.