In the years since the discovery of RNA interference, researchers have found that this process is far more widespread than we might expect, and that small pieces of RNA play many functional roles. Small (or short) interfering RNA (siRNA) is the most commonly used RNA interference (RNAi) tool for inducing short-term silencing of protein coding genes.
siRNAs are widely used to assess the individual contributions of genes to an assortment of cellular phenotypes including cytokinesis, apoptosis, insulin signaling and cell differentiation. siRNA screens have been used to identify novel pathways and have significant impact in validating targets for a number of cellular processes and diseases including cancer, HIV infection and hepatitis.
siRNA is currently being evaluated for therapeutic opportunities and is soon to arrive at the destination of commercialization. siRNA is based on cells natural response when they detect infection or genetic abnormality. Two of the most popular methods of siRNA delivery are nanoparticles and liposome-based systems. The liposome-based systems have, up to now, been associated with liver-targeted pharmacokinetics, a phenomenon that restricts the therapeutic potential of RNAi to common diseases of the liver such as hepatitis, hepatocellular carcinoma, and hypercholesterolemia.
After the period of high expectations and blockbuster deals, the market recoiled in loss. Around 2009, big pharma companies had withdrawn their clinical candidates due to lack of clinical result in terms of safety and efficacy. Allergan had terminated its lead product AGN 211745 (formerly Sirna-027) for wet age-related macular edema in Phase II clinical trials. Opko health had withdrawn phase III study of its clinical candidates Bevasiranib in wet age-related macular edema and also Alnylam’s ALN-RSV01 approach for respiratory viral infection did not meet specific delivery chemistries.
Lately, Pharma industry took a giant leap in RNA therapies with the involvement of approximately 19+ active companies. More than 70+ products are in pipeline and Alnylam is the current leader for other siRNA related pharma companies. Alnylam alone has several RNAi-based drugs in pipeline and slowly moving through clinical trials. Alnylam even started its own initiative named “Alnylam 5 x 15”—an effort to get five products in advanced clinical development by the year 2015. Companies’ two products ALN-TTR02 and ALN-TTRsc are currently in Phase III clinical trials for the treatment of TTR-Mediated Amyloidosis an autosomal inherited disease that affects about 50,000 people worldwide and universally kills patients within five to 15 years of diagnosis. The company is heading towards introducing its products in the market which seem to hit the market very soon.
DelveInsight’s, Small interfering RNA (siRNA) Therapeutics- Competitive Landscape, Technology and Pipeline Insights, 2015 Report:
The Small interfering RNA (siRNA) Therapeutics- Competitive Landscape, Technology and Pipeline Insights, 2015 report provides information of 70+ pipeline drugs with 19+ companies actively involved in drug development. There are 2 drugs in phase III, 1 in phase II/III, 9 in phase II, 4 in phase I/II, 8 in phase I, 31 in pre-clinical, 12 in discovery, 3 in inactive and 4 in discontinued.
DelveInsight’s Small interfering RNA (siRNA) Therapeutics- Competitive Landscape, Technology and Pipeline Insights, 2015 report will help companies to optimize their portfolio by analyzing the market and creating effective counter-strategies for collaborations and licensing deals. Apart from this the report on Small interfering RNA (siRNA) Therapeutics- Competitive Landscape, Technology and Pipeline Insights will provide the technologies in use that will allow the Pharma manufacturers to attain high production at competitive pricing.
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