Length:136 pages

Date Published:October 2010 

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RNAi Therapeutics: Second-Generation Candidates Build Momentum Report - Overview

Brochure Cover ImageAuthor:  Allan Haberman, PhD 

Many established pharma companies have entered into major deals with some of these RNAi therapeutics companies.  Also covered are companies focus on development of microRNA-based therapeutics and diagnostics.  These have attracted the interest of the investment community and the pharmaceutical industry.  This report discusses the outlook for the therapeutic RNAi/miRNA industry sector, including strategic issues such as technological prematurity and the development of enabling technologies, the role of Big Pharma investment, the impact of patent litigation and cross-licensing in shaping the RNAi/miRNA sector, and a scenario for the development of drugs in the therapeutic RNAi/miRNA sector.

Key topics covered include:

  • Delivery vehicle technologies
  • Oligo protective chemistries
  • Therapeutic indications in development
  • Phase I and II development highlights
  • Big Pharmas’ investments, activities and strategies
  • Profiles of leading companies


Following an overview of the discovery and evolution of the RNAi and miRNA field, RNAi Therapeutics: Second-Generation Candidates Build Momentum turns to the science behind therapeutic RNAi and miRNA, technologies for design of therapeutic oligonucleotides that work via an RNAi or miRNA-modulating mechanism, technologies for design of delivery vehicles, and leading companies in the therapeutic RNAi/miRNA industry sector as well as the role of large pharmaceutical companies in the sector.

 

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Selected leading therapeutic RNAi specialty companies discussed in Chapter 5 “RNA interference” (RNAi) refers to the inhibition of expression of specific genes by small doublestranded RNAs. Since the emergence of RNAi in the late 1990s, it hasbeen seen in the medical community as breakthrough science. More recently, the biology of microRNAs (miRNAs), a class of naturally occurring small RNAs, has also been found to be involved in gene regulation. The gene regulatory function of these molecules has generated intense interest in the development of oligonucleotide drugs to address targets that are considered to be “undruggable” by small-molecule compounds or biologics. The history of the development of antisense and aptamer drugs, though difficult, is providing the foundation for the successful development of oligonuclotide drugs. This has resulted in developing chemical modifications to prevent their destruction by nucleases, improve their pharmacokinetics, and enhance their target specificity and binding affinity.

RNAi Therapeutics: Second-Generation Candidates Build Momentum includes a review of RNAi company pipelines as well as activities within large pharmaceutical companies. Many established pharma companies have entered into major deals with some of these RNAi therapeutics companies. Also covered are companies’ focus on development of microRNA-based therapeutics and diagnostics. These have attracted the interest of the investment community and the pharmaceutical industry. This report discusses the outlook for the therapeutic RNAi/miRNA industry sector,
including: strategic issues such as technological prematurity and the development of enabling technologies, the role of Big Pharma investment, the impact of patent litigation and cross-licensing in shaping the RNAi/miRNA sector, and a scenario for the development of drugs in the therapeutic RNAi/miRNA sector.

About the Author:Allan B. Haberman, Ph.D., is Principal of Haberman Associates, a consulting firm specializing in science and technology strategy for pharmaceutical, biotechnology, and other life science companies. He is also a Principal and Founder of the Biopharmaceutical Consortium (www.biopharmconsortium.com), an expert team formed to assist life science companies, research groups, and emerging enterprises to identify and exploit promising breakthrough technologies. Dr. Haberman is also the author of numerous publications on the pharmaceutical and biotechnology industries, their technologies and products, and the major therapeutic areas for drug discovery and development. Formerly the Associate Director of the Biotechnology Engineering Center at Tufts University, he received his Ph.D. in biochemistry and molecular biology from Harvard University.