12 Sep, 2011 Delivery Advances at 2011 OTS Meeting
In September 2011, we and our collaborators presented new data at the 7th Annual Meeting of the Oligonucleotide Therapeutics Society demonstrating substantial new advances in our efforts around systemic delivery of RNAi therapeutics.
A few key highlights include:
- discovery of a completely novel class of third generation LNPs that show dramatic improvements in therapeutic index based on our current analysis;
- a new lipidoid formulation discovered in our MIT collaboration that sets a new potency benchmark for the field; and,
- significant advancements with our conjugate platform that enables clear translation into the clinic.
Mechanistic Insights Into Lipid Nanoparticle (LNP)- Mediated Delivery of siRNA
This poster illustrates recent advancements in LNP-based delivery of RNAi therapeutics, including the discovery of a novel class of third generation LNPs, or “reLNPs.” These novel lipids exhibit promising improvement in therapeutic index. Additionally, we presented data on the “MD1” lipid, derived from our collaboration with Daniel Anderson’s laboratory at MIT, which demonstrates an exciting new potency benchmark for systemic delivery.
Hepatocyte-Specific Targeting and Delivery of siRNA-Carbohydrate Conjugates
In this poster, we presented significant new data demonstrating advancements related to the stability, potency, and efficacy of novel GalNAc-conjugated siRNAs for systemic delivery of RNAi therapeutics. This new data exhibited that improvements in chemistry led to significant increases in potency for in vivo target gene silencing with GalNAc-conjugated siRNAs administered by subcutaneous injection.