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3.3.7 Ribozyme Therapy

Ribozymes, discovered in 1981 by Cech et al. ,Cell 26: 487-496), are RNA molecules capable of catalyzing RNA cleavage in a special way.

Heptazyme (also known as Ribozyme Gene Therapy or LY 466700), was a product of Ribozyme Pharmaceuticals, which is now called Sirna Therapeutics. Its Phase II trials for the treatment of chronic Hepatitis C, were stopped due to blindness that occurred in one animal during toxicology testing. Attempts were to be made to determine whether this outcome was related the use of the drug.

Immusol HCV Ribozyme Gene Therapy Laboratory studies have shown this kind of ribozyme gene therapy inhibits the formation of new hepatitis C virus particles, and may be especially useful in combating some problems with anti-HCV drug design, such as emergence of drug resistant virus types. The company is collaborating with Vertex.

Atugen Biotechnology GmbH (Berlin, Germany) The company is developing two technologies: one is proprietary oligonucleotides, small segments of RNA that inhibit expression of genes, in a program called GeneBloc; the other is ribozymes. The company is working together with others, including Schering, Roche and Ribozyme Pharmaceuticals.

Other companies that were working with ribozyme therapy were Innovir Laboratories, and VimRx Pharmaceuticals Inc., a subsidiary ( www.biospace.com).



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