Date: 2015-10-13
Type of information: Presentation of results at a congress
phase:
Announcement: presentation of results at the 11th Annual Meeting of the Oligonucleotide Therapeutics Society (OTS), held October 11 - 14, 2015, in Leiden, Netherlands
Company: Alnylam Therapeutics (USA - MA)
Product: Bis-RNAi™ Platform
Action
mechanism: RNAi
Disease:
Therapeutic area: Technology - Services
Country:
Trial details:
Latest
news: * On October 13, 2015, Alnylam Pharmaceuticals announced advancement of innovations in RNAi therapeutics with presentation of scientific data on two new platform technologies. The research was presented at the 11th Annual Meeting of the Oligonucleotide Therapeutics Society (OTS), held October 11 - 14, 2015, in Leiden, Netherlands . First, the company presented data on its Bis-RNAi™ platform that enables simultaneous knockdown of two distinct disease genes with a single, subcutaneously administered molecular entity. Further, the company presented data on its Reversir™ platform. Reversir molecules enable tailored control of RNAi pharmacology with rapid reversal of target gene silencing effects. The company believes that these new platform technologies greatly expand the universe of opportunities for RNAi therapeutics.
In a poster presentation entitled "Bis-RNAi™ Conjugates for Simultaneous Silencing of Two Transcripts," Alnylam scientists described a novel approach whereby a single molecular entity consisting of two linked siRNAs conjugated to a GalNAc moiety can simultaneously achieve robust silencing of two different mRNA transcripts. Multiple Bis-RNAi designs were evaluated to determine the effects of linker structure and GalNAc ligand placement on in vivo activity. In rodent and non-human primate studies, a Bis-RNAi tool compound targeting transthyretin (TTR) and Factor VII (FVII) achieved similar levels of target gene knockdown as that observed with a "parent" mixture consisting of the individual siRNAs at a 1:1 ratio. In addition, the duration of silencing effects for the Bis-RNAi compound was found to be similar to that of the individual siRNAs in the parent mixture. Furthermore, the Bis-RNAi constructs were found to lack immune activity as measured in both an in vitro human whole blood assay and in an in vivo mouse model, with cytokine profiles comparable to the parent mixture and untreated controls. Alnylam believes that there are many potential clinical applications for the Bis-RNAi platform, including in the company's Cardio-Metabolic and Hepatic Infectious Disease STArs, and expects to advance its first Development Candidate from this platform in 2016.
