RNAi Therapeutics: Challenges in Drug Development and Delivery

CHI Insight Pharma Reports
October 1, 2005
120 Pages - SKU: CHI1197143
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Table of Contents Related Reports
Interfering RNA (RNAi) offers tremendous therapeutic promise. This report:
  • Evaluates the prospects for curing disease using RNAi.
  • Examines the pros and cons of all the major known methods of delivering RNAi.
  • Describes advances in in vitro work with cultured cells.
  • Assesses the market potential of RNAi drugs.
  • Presents the opinions of leading scientists in academia and industry.
  • Profiles twenty-three companies.
Interfering RNA (RNAi) offers tremendous therapeutic promise to silence genes that give rise to bad, proteins and, therefore, disease. Theoretically, any gene can be targeted for silencing by RNAi; hence, the possibilities are limitless. The principle has already been proved not only in cell culture but also in in vivo major disease-related animal models.

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. Before RNAi, finding gene function by knockdown or knockout technology was a laborious and time-consuming process. 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.


 

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