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Novartis strengthens neuroscience pipeline with $12bn Avidity acquisition
Deal to advance potential first-in-class RNA therapeutics focused on genetic neuromuscular diseases such as Duchenne muscular dystrophy.
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Myotonic Dystrophy Type 1
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First targeted delivery of siRNA into muscle
A Phase I/II trial demonstrated the first successful targeted delivery of siRNA into muscle, using monoclonal antibody AOC 1001.
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$38 million will fund rare disease research over four-year period
Research for rare diseases including ALS will be supported over the next four years by $38 million in FDA funding.
