Ribozyme On Target To Move Drugs Into The Clinic

Ribozyme Pharmaceuticals Inc is gathering momentum to push its novel catalytic RNA-based therapeutics into the clinic, and expects to file an Investigational New Drug application for its lead product, for the treatment of HIV infection, before the end of the year.

Chief executive and president of RPI Ralph Christoffersen told a meeting in London, UK, recently that the trial protocol designed for the preliminary testing of the HIV product has been exempted from review by the US National Institutes of Heath's Recombinant DNA Advisory Committee, and that clinical trials should now begin in the early part of 1997. Furthermore, an application to do trials of a second product, for the treatment of diabetic retinopathy, could also be filed in the first half of next year.

Reviewing the company's technology, Dr Christoffersen told the meeting that RPI can now offer a broad platform combining both product development and drug discovery. The company's ribozyme products are designed to bind to a specific sequence of messenger RNA and cleave it, preventing the transcription of disease-associated proteins in the cell. This can be achieved without affecting other cellular systems, so ribozymes represent a wide-ranging and selective technology for the control of gene expression.