Transcending GalNAc Conjugates Through Chemical Modifications for Enhanced Delivery, Safety and Efficacy of RNAi Therapeutics

• Leveraging advanced chemical modifications to develop novel conjugate platforms, to enable delivery beyond the liver with high specificity, to expand treatable diseases to include neurological disorders and rare cancers
• Applying bespoke chemical modifications to peptide-based and other conjugate systems to improve endosomal escape and intracellular siRNA bioavailability to boost gene silencing efficacy while reducing dose frequency and toxicity
• Implementing a next-generation, data-rich platform for the rapid design and optimization of novel ligand-siRNA chemistries to accelerate optimization of delivery chemistries for unmet clinical needs, for future-proofing Alnylam’s pipeline against GalNAc’s limitations

Speakers:

Mano Manoharan

Senior Vice President, Innovation Chemistry

Alnylam Pharmaceuticals