Important step in the research of RNAi therapeutics
Alnylam Pharmaceuticals, the leading RNAi therapeutics company, announced that they have achieved delivery of novel small interfering RNA (siRNA) conjugates to the central nervous system (CNS) and are planning to advance a pipeline of investigational RNAi therapeutics into clinical development. The conjugates achieved robust, durable, and broadly distributed silencing of CNS gene transcripts following a single intrathecal injection. Alnylam plans to complete selection of its first CNS-targeted development candidate in 2018, and then expects to file its first investigational new drug (IND) or IND equivalent in late 2019/early 2020, with the potential for one or more INDs per year thereafter.
This news confirms that the B-SMART consortium is on the right track with the development of effective nanoparticles that transport RNA therapeutics to the brain. The B-SMART strategy is designed to avoid the injection into the brain that was used by Alnylam for their proof of concept studies. It would avoid the obvious risks for side effects and a highly invasive procedure. The first B-SMART prototype is currently being tested in a preclinical study.
More on Alnylam’s news can be found here.