21 Jun, 2019 New RNAi Platform Advances, Including Oral Route of Administration and CNS and Ocular Delivery
We presented new advances in our RNAi therapeutics platform, including preclinical results demonstrating oral delivery of GalNAc-conjugated small interfering RNAs (siRNAs) directed to a liver target. The results were presented at the 3rd International Conference on the Long and the Short of Non-Coding RNAs, held June 18-23 in Crete, Greece. These new preclinical data are the first demonstration of functional delivery of GalNAc-siRNA conjugates via the oral route of administration, representing an important step forward in potentially advancing and expanding the clinical and commercial potential of RNAi therapeutics. We believe that this approach can be applied to existing and future liver-directed pipeline programs, potentially creating a relatively near-term opportunity for Alnylam.
In addition, we presented new advances in CNS and ocular delivery demonstrating durable and efficacious target silencing in rodents and non-human primates with a favorable profile compared with antisense oligonucleotides.
