Interfering RNA (RNAi) offers tremendous therapeutic promise to silence genes that give rise to bad, proteins and, therefore, disease. RNAi Therapeutics: Challenges in Drug Development and Delivery thoroughly evaluates the field of RNAi therapeutics, with particular attention to the prospects and challenges faced in RNAi delivery. This report is a comprehensive blueprint for moving RNAi from the bench to the bedside. 

RNAi research is accelerating rapidly. Two clinical trials are in progress to determine RNAi s effectiveness for treating age-related macular degeneration, and many products have already reached the market to test the promise that RNAi holds. As RNAi technology progresses, many companies are now trying to increase their shares in the research market by shifting some of their resources to in vitro and in vivo work. 

The real prize, however, is in therapeutics, and a few companies have been started solely with this objective. To establish priority, patents that stake claims in many possible (as well as speculative) applications of RNAi are not only being filed but disputed.

RNAi Therapeutics: Challenges in Drug Development and Delivery analyzes these and other important developments in this dynamic field. Key points include the following: 

• Background is provided on various attempts to silence genes before the advent of RNAi, namely aptamers, antisense, and ribozyme. Lessons learned from the failures of antisense clinical trials are discussed. 

• The promise of tackling diseases using RNAi is evaluated in detail. Examples are included that support this technology, from human cell cultures, to preclinical models, to the first batch of clinical trials using siRNA (small interfering RNA). Wide-ranging diseases cancer, influenza, malaria, HIV, rotavirus, Huntington s disease, and Lou Gehrig s disease are discussed. 

• Advances in in vitro work with cultured cells are examined. Progress made in in vivo gene silencing is catalogued, including the latest results in detection of in vivo knockdown in whole mice. 

• The problems of RNAi delivery and possible solutions to overcome them are presented from the point of view of leading scientists in academia and pharmaceutical companies. 

• The therapeutic potential, specific applications, and advantages and challenges of RNAi are considered, in particular, the prospects of improving on existing treatment modalities for some cancers such as chronic myelogenous leukemia. 

• The market potential of RNAi drugs is assessed. 

• Leading scientists in academia and industry are interviewed. 

• Twenty-three companies are profiled, 13 of which are primarily engaged in RNAi therapeutics-related work and 10 of which are suppliers and service providers. 

About the Author 
S.B. Dev, Ph.D., is an independent analyst specializing in gene therapy, with special emphasis on delivery of genes and nucleotides. He serves as Chief Scientist at Gene Delivery & Expression Sciences (San Diego, CA). Dr. Dev has over 20 years’ experience in the area of life sciences and application of physical techniques to biology, with more than 12 years of work in drug and gene delivery. Past positions have included Director of Research/Principal Scientist at Genetronics, Inc., and Visiting Scientist at the M.I.T. Department of Applied Biological Sciences. Dr. Dev has had 11 issued U.S. patents. He is the author of numerous publications and speaks at international conferences on drug delivery and gene therapy. Dr. Dev received his Ph.D. from Chelsea College (now King’s College, London).