Abstract

RNA interference (RNAi) quietly crept into biological research in the 1990s when unexpected gene-silencing phenomena in plants and flatworms first perplexed scientists. Following the demonstration of RNAi in mammalian cells in 2001, it was quickly realized that this highly specific mechanism of sequence-specific gene silencing might be harnessed to develop a new class of drugs that interfere with disease-causing or disease-promoting genes. Here we discuss the considerations that go into developing RNAi-based therapeutics starting from in vitro lead design and identification, to in vivo pre-clinical drug delivery and testing. We conclude by reviewing the latest clinical experience with RNAi therapeutics.

Author affiliations

1. Antonin de Fougerolles and John Maraganore are at Alnylam Pharmaceuticals Inc., 300 Third Street, Cambridge, Massachusetts 02142, USA.
2. Hans-Peter Vornlocher is at Alnylam Europe AG, Fritz-Hornschuch-Str. 9, Kulmbach 95326, Germany.
3. Judy Lieberman is at the CBR Institute for Biomedical Research, 200 Longwood Avenue, Boston, Massachusetts 02115, USA.

Correspondence to: Antonin de Fougerolles¹ Email: tdefougerolles@alnylam.com

Correspondence to: Judy Lieberman³ Email: Lieberman@cbr.med.harvard.edu

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