Bluebird’s Improved Vector Yields Promising Early Phase II Data In Beta Thalassemia
This article was originally published in The Pink Sheet Daily
Two patients achieve independence from transfusions and show normalizing levels of hemoglobin in Phase II trial of experimental viral vector-based gene therapy. Bluebird’s share price soared on the promising news.
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The gene therapy start-up taps diversified public and private investors, as well as strategic backer Shire, for one of the year’s biggest rounds of private capital funding for a biotech thus far.
bluebird bio LLC has taken in $65 million in venture capital over the past year, thanks to encouraging studies with two different kinds of lentiviral vectors that transfer new copies of genes into patients' own stem cells. Given that vectors have different properties - one integrates into the DNA coding region, the other does not - the company might eventually be able to mix and match therapeutic gene "fillings" to vectors as needed. The start-up is ready to start commercializing gene therapies for serious genetic disorders, starting with ALD and beta-thalassemia. .
The latest mega deals aimed at bringing Chinese innovation to the US have rekindled go-global hopes for a sector badly needing some cheer. But some investors say out-licensing may be the best route and that Chinese biotech must scale back its do-all mentality and focus on what it does best amid a new reality.