RNAi’s Drugs
Novel therapies fill the pipeline
Pharmaceutical giant Merck’s purchase last fall of Sirna, a San Francisco biotech company specializing in RNA interference (RNAi), was just the latest indication that the technology is gaining momentum. Researchers are currently trying to use RNAi to do everything from treat flu to permanently remove hair. In RNAi therapy, small pieces of double-stranded RNA shut down the genes whose sequences they match. Below is a sampling of RNAi therapies under investigation.
Disease/company
Approach
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Status
Age-related macular
degeneration
Acuity (Philadelphia, PA) and Sirna (San Francisco, CA)
Eye injection of RNA that blocks a gene responsible for excessive growth of blood vessels
In phase II clinical trials
Viral lung infections
Alnylam (Cambridge, MA)
Inhalation of drug containing RNA that shuts down viral genes
Therapy for respiratory syncytial virus in phase I trials
Parkinson’s disease
Alnylam and Sirna
Injection of RNA to block a gene that kills brain cells
In preclinical development
Huntington’s disease
Sirna
Injection of RNA to block a gene that kills brain cells
In preclinical development
Hair removal
Sirna
Application of cream containing RNA that blocks hair growth genes
Application to begin clinical trials in progress
