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. .
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CEO Paul Stoffels said gaining US clearance for an IND for its novel CAR-T product was demanding, but now opens up a pathway towards a pivotal study starting in 2025.
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In this week's episode: Novo Nordisk’s CEO forced out; quantifying biopharma’s contribution to the US economy; a history of MASH deals; lift for AstraZeneca’s asthma ambitions; and major Japanese firms see US revenue growth.
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