In addition to effective gene silencing, these candidates can be deployed for applications related to precision medicine. Studies have also demonstrated the safety of RNAi therapeutics for in systemic delivery, paving the way for systemic applications of the therapy. A number of novel and sophisticated technologies / platforms have been / are being developed to overcome the challenges associated with RNAi therapeutics, as well as further enhance their specificity and stability. Further, considering their high knockdown efficiency, high target specificity and extended silencing ability, RNAi has now been established as a powerful tool for gene silencing. It is worth highlighting, RNAi has potential applications in treating several viral infections and tumors in addition to its ability in gene function determination. However, despite the numerous benefits offered by RNAi therapies, developers often face concerns related to the stability and targeted delivery of their candidates. In order to mitigate the aforementioned challenges, several players have developed novel technologies and delivery systems to ensure target specific delivery of these molecules.
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With four approved drugs and several therapy candidates being evaluated in late stages of clinical development, the RNAi therapeutics domain presents a significant opportunity for biopharmaceutical developers. In addition, a number of RNAi therapeutics are being developed to target rare and genetic clinical conditions, such as Alpha 1-Antitrypsin Disease, Alport Syndrome, Amyloidosis and Muscular Dystrophy (Facioscapulohumeral Muscular Dystrophy and Oculopharyngeal Muscular Dystrophy). It is also worth highlighting that over 2,100 patents have been filed / granted, highlighting the continuous pace of ongoing innovation in this field. Given the high research activity and ongoing technology advancements, the RNAi therapeutics market is poised to grow at a steady pace in the foreseen future. An insightful technology competitiveness analysis, benchmarking RNAi technologies, based on supplier power (in terms of company size and years of experience) and key technology specifications. The analysis was designed to enable stakeholder companies to compare their existing capabilities within and beyond their respective peer groups and identify opportunities to achieve a competitive edge in the industry.
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