GZ: Antisense and siRNA may offer some advantages over...

GZ: Antisense and siRNA may offer some advantages over conventional therapies. The antisense complex can be synthesized chemically in a lab and then introduced into the cell. Once introduced, antisense can target virtually any protein synthesized by the body. This is a significant advantage over small molecule or antibody drug candidates that target only specific classes of proteins. With knowledge of the sequenced human genome, scientists should be able to develop antisense compounds for each and every gene and mRNA.

Additionally, the antisense identification and subsequent production process is more straightforward than for the traditional small molecule or antibody drug discovery platform. Scientists only need to identify the specific gene worth testing, and then synthesize the antisense oligonucleotide. This offers a significant time and cost-of-discovery advantage.

Although RNAi therapeutics is newer to the pharmaceutical industry than antisense, the structures are similar, with siRNA being a double-stranded nucleic acid complex compared to the single-stranded antisense oligonucleotide. As a result, the above-mentioned advantages of antisense over conventional therapies are also expected to apply to siRNA.

Antisense and siRNA are two of the most promising fields of targeted therapy. Development of antisense and RNAi therapeutics, however, has been limited by the lack of a suitable method to deliver these drugs to diseased cells, with high uptake and without causing toxicity. But great progress in RNA delivery has been made recently by companies including Tekmira and Bio-Path Holdings.


Isis is back at $17.34. Up 5 percent...tonight...ANP to follow shortly.