Alnylam Pharmaceuticals and Quark Pharmaceuticals emerged as leading players in this domain. It is worth mentioning that Alnylam Pharmaceuticals is a large company that has an experience of over 17 years in developing RNAi based drug candidates. The company has a robust pipeline with two marketed drugs, Onpattro and Givosiran. Further, Onpattro has been marketed across US, Europe, Canada and Japan, for the treatment of genetic disorders, while Givosiran is presently approved in the US alone. Alnylam’s pipeline consists of a total of 11 drug candidates, out of which four are currently in phase II/III and above; these are Fitusiran (phase III), Inclisiran (phase III), Lumasiran (phase III) and Vutrisiran (phase III). The company is currently developing drug candidates for eight therapeutic areas, including (in alphabetical order) cardiovascular disorders, genetic disorders, hematological disorders, immunological disorders, infectious diseases, metabolic disorders, oncology and others. In addition, Alnylam has developed its proprietary delivery technology, ESC-GalNAc, which is being used to develop the company’s potential drug candidates.
Quark Pharmaceuticals, established in 1994, is a mid-sized company. It is worth noting that, close to 30% of the company’s pipeline candidates are currently in various phases of clinical development; examples of its clinical stage molecules are QPI-1002 (phase III), QPI-1007 (phase III) and PF-655 (phase II). The company has licensed a technology from Dicerna Pharmaceuticals that enables them to deliver QPI-1002 and PF-655 in the form of naked siRNA. Quark has also developed its proprietary delivery technology, BiFAR target discovery platform, which it intends to use for other pipeline molecules. Currently, pipeline molecules of the company target six therapeutic areas, including (arranged in alphabetical order) cardiovascular disorders, dermatological disorders, oncology, ophthalmic diseases, respiratory disorders and others.
Other companies with high competitiveness are (in decreasing order) Dicerna Pharmaceuticals, Arrowhead Pharmaceuticals, Olix Pharmaceuticals, Sirnaomics, Gradalis and Souzhou Ribo Life Sciences; interestingly, pipeline molecules of these companies target at least five therapeutic areas, except Gradalis. However, Gradalis still has high pipeline strength owing to its clinical pipeline and proprietary delivery technology, vigil. It is worth mentioning that, even though these companies are significantly less experienced, they have strong pipeline for RNAi based therapeutics, which target different therapeutic areas.