BioPharma Dive November 6, 2024
Empowered by new delivery tools, a handful of young biotechs aim to take the drugmaking technology in new directions.
Research into RNA interference, a Nobel Prize-winning technique to mute disease-causing gene mutations, has gained momentum in recent years. Since 2018, six medicines based on the technology have won approval to treat a variety of rare diseases, as well as high cholesterol. Dozens more are in clinical testing.
Yet the field’s best-known companies, led by Alnylam Pharmaceuticals, have only taken RNAi so far. Their drugs are still largely focused on disease targets in the liver, limiting RNAi’s reach compared to other types of medicines. The technology’s maturation has also come at a time of fast progress for genetic medicines, like those...
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