Clinical and Pre-Clinical Data on RNAi Therapeutics at 10th Oligonucleotide Therapeutics Society Meeting

We presented new data from multiple clinical and pre-clinical studies at the 10th Annual Meeting of the Oligonucleotide Therapeutics Society (OTS), held October 12 – 15, 2014 in San Diego.  Among multiple presentations, we presented additional data from our Phase 1 trial with ALN-TTRsc showing rapid, dose-dependent, stable, and durable knockdown of serum TTR of up to 96.2%.  In addition, we presented pre-clinical data from a new program, ALN-GO1, an investigational RNAi therapeutic targeting glycolate oxidase (GO) in development for the treatment of primary hyperoxaluria type 1 (PH1), showing efficacy in rodent disease models.  Finally, we presented new pre-clinical research demonstrating that delivery of Enhanced Stabilization Chemistry (ESC)-GalNAc-siRNA conjugates to the lung achieves similar plasma exposure, efficacy, and duration of liver gene silencing as achieved by subcutaneous delivery.  This finding opens up the possibility for needle-less administration of RNAi therapeutics via inhalation for knockdown of liver disease genes.