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Sept. 4, 2025
| This week’s gene therapy news and insights for biopharma leaders
The agency's two main review offices will work together to flexibly evaluate medicines for serious conditions that affect fewer than 1,000 people in the U.S.
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Updated findings from Wave’s pioneering trial continued to show its RNA editing therapy is working as intended, but wasn’t as effective in testing as investors had hoped.
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The alliance, which could be worth more than $5 billion, is one of a handful of deals Novartis has struck with China-based biotechs in recent years.
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Gene therapy is once again at the forefront of biomedical research, catalyzed by advances in safer delivery of genes to cells. Explore how the industry is evolving in
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Co-founded by RNA interference pioneer John Maraganore and former Medicines Company head Clive Meanwell, Corsera Health is developing diagnostic tools and medicines for heart health.
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Tryngolza, the first wholly owned drug Ionis has launched, helped people with high triglyceride levels that aren’t genetically driven.
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