By now we’re all aware of CRISPR’s ability to splice and edit genes, but recent article from says SingularityHub there’s a simpler way that could be the new class of blockbuster drugs. Gene silencing destroys RNA, the “messenger” that uses DNA information to dictate protein production. This beautifully simple idea has been around for twenty years, but pharmaceutical companies lost interest when CRISPR moved in and changed the conversation to gene editing. But it’s making a comeback.
Back in December, Ionis Pharmaceutical announced a positive trial of their gene silencer for Huntington’s disease. The drug is an anti-sense oligonucleotide that successfully lowered toxic protein levels in the brains of 46 patients with early-stage Huntington’s. Roche caught wind of this and licensed the drug for $45 million in an effort to further development toward larger trials. Although it’s not the first gene-silencing drug to have success, it is the first to focus on an incurable brain disease, which is huge.