Biotechnology company
Appears in 2 stories
Subsidiary of Novartis;flagship asset in limbo
Novartis' experimental muscle-disease drug del-desiran failed its phase 3 Harbor trial, missing the primary endpoint and sending shares down. The drug was the centerpiece of the company's $12 billion acquisition of Avidity Biosciences, which closed last year.
Updated 4 days ago
Leading exon-44 race with 25% dystrophin data
Boys born with Duchenne muscular dystrophy typically lose the ability to walk before their teens and rarely live past 30. The first drugs designed to slow that decline reached the market in 2016, but they restore less than 1% of normal dystrophin, the muscle protein patients are missing. A decade later, a new wave of biotech companies is trying to deliver far more of that protein into muscle cells using engineered molecular shuttles.
Updated May 31
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