ARTICLE | Tools & Techniques

Neutralizing the negatives of RNAi

siRNN technology delivers RNAi to liver and other tissues

December 18, 2014 6:40 PM UTC

Despite years of attempts with liposomes, nanoparticles and conjugation strategies, delivery of siRNA to organs beyond the liver remains the biggest hurdle to use of the technology for a host of diseases. A UCSD group has changed the rules with a prodrug strategy that delivers siRNA into cells as an uncharged oligonucleotide, bypassing the limitations of siRNA's negative charge.1 But the real breakthrough will be to show the prodrugs work in a range of cells and tissues that standard siRNAs cannot easily reach.

Solstice Biologics LLC obtained an exclusive worldwide license to the technology last year and set up shop to translate the strategy into therapeutics. The company was co-founded by Steven Dowdy, the University of California, San Diego investigator behind the invention, and Curt Bradshaw, who is CSO...