LONDON Proteome Sciences plc, of Cobham, Surrey, said it has developed technology for repairing the genetic mutations found in the mRNA which is defective in cystic fibrosis. The company said it believes the technology can be applied to a wide range of other genetic and viral diseases, such as cancer and HIV.
The ability to reprogram defective genes would mark a significant advance in gene therapy. The technology was presented Jan. 17 at the Keystone Symposium for Molecular and Cellular Biology of Gene Therapy in Salt Lake City, by Mariano Garcia-Blanco, director of research at Proteome Sciences¿ U.S. subsidiary, Intronn LLC, of Raleigh-Durham, N.C. Garcia-Blanco is also associate professor of cancer biology at Duke University Medical Center in Raleigh-Durham.
According to Garcia-Blanco, the technology involves the development of therapeutic RNA molecules which target the mutated, abnormal cystic fibrosis mRNA. The targeted cells are thus reprogrammed to make the correct gene products and, in effect, repair themselves. ¿This new tool for gene therapy and RNA repair appears to be a major scientific advance,¿ Garcia-Blanco said. ¿In addition to its potential application in cystic fibrosis, we have seen some excellent results both in cancer cells in culture and in mice tumors.¿
Intronn says its patented Spliceosome Mediated RNA Trans-splicing (SMaRT) technology allows virtually any mutated gene to be targeted and reprogrammed to produce a new gene product useful in treating a particular disease.
SMaRT offers key advantages over conventional gene therapy, in which the aim is to deliver the correct version of the whole gene. First, the treatment can be more easily accommodated by viral delivery vectors, and second, it is targeted to the faulty cystic fibrosis cells, preventing healthy cells from being damaged.
The share price of Proteome Sciences, which is listed on London¿s Alternative Investment Market, rose 5 pence to 15 pence when the news was announced. James Malthouse, finance director for the company, told BioWorld International the company ¿is actively looking for partners. There is interest, but it is impossible to say at this stage what kind of deal we will do,¿ he said. ¿It depends what is on offer. If someone wanted to invest in Intronn, we would look at that. Or, if they wanted to invest by disease area, we would look at that, too.¿ n