RNA Interference is Coming of age as a Therapeutic Modality
News May 12, 2006
The Business Intelligence firm La Merie S.L. has reported that the RNAi therapeutic pipeline is rapidly filling and advancing towards clinical development.
Four RNAi therapeutic projects are in early clinical development. At least 32 RNAi projects are in preclinical development in a variety of therapeutic areas.
Viral diseases and cancer are the preferred indications. A total of 14 RNAi projects are approaching IND status within one year.
At least 20 projects are in advanced research stages. These results and more were found in a search conducted by La Merie Business Intelligence.
RNAi technology has been rapidly developed and adopted as a laboratory tool for potent and specific downregulation (silencing) of the expression of a gene at the post-transcriptional or even transcriptional level.
Now, RNAi has entered the clinical arena with therapeutic applications. Clinical use of RNAi was pioneered by Acuity Pharmaceuticals with Cand5.
The small interference RNA for VEGF completed phase II studies in wet AMD and diabetic macular edema.
In contrast to Acuity which is developing as an ophthalmic disease company, major RNAi players Alnylam Pharmaceuticals and Sirna Therapeutics as well as other companies are using delivery and protection technologies to broaden therapeutic applications of RNAi molecules by systemic delivery.
The availability of siRNA or locked nucleic acid RNA (LNA RNA) reduced the risk of immunogenicity of RNAi molecules.
The delivery technologies appear to solve the problem of large molecular weight and negative charge of siRNA duplexes which represents a major challenge for effective cellular uptake and intracellular delivery.
Chemical optimization of the RNAi molecules as well as coating technologies contributed to in vivo stabilization of the RNA molecules.
Apart from the rapidly filling pipeline, the increasing maturity of the RNAi field is also evidenced by alliances of Big Pharma companies GlaxoSmithKline and Novartis with RNAi players having a solid intellectual property position.
Back in 2009, researchers identified a herd of Awassi sheep suffering from "day blindness". As that term implies, these sheep were blind during the day (in bright light) but could see at night, in low-light conditions. After identifying the genetic basis of this blindness, researchers have now successfully used gene therapy to restore their daytime vision.READ MORE