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Nov. 21, 2024
| This week’s gene therapy news and insights for biopharma leaders
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NOTE FROM THE EDITOR
BioPharma Dive: Gene Therapy will not publish next Thursday, Nov. 28, as we take some time off for the Thanksgiving holiday. We’ll be back in your inboxes the following week as usual.
In the meantime, be sure to check out our new database of venture investment, which you can use to keep track of biotech funding rounds.
Thank you for reading!
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Delilah Alvarado
Staff Reporter, BioPharma Dive
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Deep Dive
Venture capital firms incubate, grow and finance dozens of new drug companies each year. Follow the money they channel into the industry with this database.
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The agency's openness to a targeted pivotal study shows it’s still willing to consider accelerated clearance for Duchenne gene therapies despite questions about their effectiveness.
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UPDATED
In buying Westlake-backed Kate Therapeutics, Novartis gets a handful of preclinical gene therapies targeting diseases like Duchenne muscular dystrophy and myotonic dystrophy type 1.
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See how a novel therapeutic company increased titers and percent-full capsids with the TransIT-AAViator transfection system, designed for maximal AAV production.
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Phase 1 data indicate Intellia’s medicine could be a powerful treatment for a cardiac form of ATTR amyloidosis. But rival drugs are further ahead.
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News roundup
The agency turned back Astellas’ attempt to update its drug Izervay’s labeling. Elsewhere, former NCI director Ned Sharpless founded a new startup and Novartis licensed another radiopharma drug.
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What We're Reading
STAT News
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