Biotech Strategy Blog

Commentary on Science, Innovation & New Products with a focus on Oncology, Hematology & Immunotherapy

Posts tagged ‘Thalassemia Gene Therapy’

Man measuring the clouds sculpture, Kanazawa

This summer I came across Jan Fabre’s fabulous bronze sculpture Man Measuring the Clouds (right) in Japan, of all places. It represents humanity’s ambition, idealism, and the pursuit of impossible goals.

The artwork depicts a life sized bronze figure of a man, often thought to be modelled after Fabre’s late brother, standing on a tall ladder and reaching upwards with a measuring rod as if trying to measure the sky. The piece captures a blend of optimism, futility, and persistence in the face of unattainable aspirations.

Similarly, we might consider the concept of tackling a variety of complex diseases from cancer and cardiac fibrosis to various genetic disorders with a tiny cell therapy involving a sample of just few millilitres seems equally apt and fitting.

Is it truly impossible though?

In our latest post, we argue no it’s not – and may actually be much closer to reality than many people realise.  What seemed like something on the distant horizon is likely to soon see not one, but multiple clinical trials opening up in other indications beyond blood cancers involving several cell therapy companies.

Following on from an inspiring talk this week, we offer 10 important lessons to think about and be challenged by – because the old yardsticks may well be a’changing sooner rather than later…

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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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