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: Reversir™ 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 . 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 presentation entitled "Reversirs for Rapid and Potent Reversal of siRNA Silencing Activity," the company shared key scientific data characterizing the development of its Reversir platform. Reversir molecules are GalNAc-conjugated, single-stranded oligonucleotide constructs that are designed to recognize and bind to the complimentary RISC-bound antisense strand of an siRNA, leading to rapid and complete reversal of RNAi-mediated target gene silencing activity in vivo. Studies were performed in mice using a GalNAc-siRNA targeting Factor IX (FIX) and Reversir constructs designed to specifically reverse the FIX siRNA silencing activity. Specifically, a single subcutaneous dose of the FIX siRNA led to robust FIX knockdown of approximately 80% that was sustained for over 4 weeks. When a single subcutaneous dose of the Reversir molecule was administered on day 8, FIX knockdown was rapidly reversed, with levels returning to baseline within one week. A preliminary rat toxicology study showed that Reversir constructs are generally well tolerated with no significant findings to date, including lack of changes in body weight gain or serum chemistry. Alnylam believes that its Reversir platform can be deployed across existing and future pipeline programs to enable tailored control of RNAi pharmacology for precision medicines, and expects to advance initial Development Candidates from this platform in 2016.
