2025 is proving to be a landmark year for RNAi. From Alnylam’s first approval in hemophilia to Novartis’ $1.2 billion acquisition of Avidity Biosciences and GSK diving into COPD with an $85 million siRNA licensing deal, the headlines haven’t stopped. RNAi is officially outgrowing its liver roots and entering a new era of therapeutic expansion.

With so much momentum shaping the RNAi landscape, there’s never been a more pivotal time for the community to come together. The 7th RNAi-Based Therapeutics Summit (January 27-29, 2026 | Boston, MA) is the industry-dedicated forum uniting the entire RNAi ecosystem from discovery and complex chemistry to clinical development and CMC.

Learn more in the official event program here: https://ter.li/8c4zw3

Through 30+ expert-led sessions, explore how pioneers are moving beyond the liver to unlock new disease frontiers:

Expanding Horizons to Systemic & Extrahepatic Diseases:

Reprogram metabolic health with dual-targeting siRNA for obesity and leverage multi-gene silencing miRNA to overcome chemo-resistance, moving beyond appetite suppression and single-target inhibition for durable, tissue-specific efficacy.

Breaking Barriers in Brain & CNS Delivery:

Overcome the blood-brain barrier with Trojan Horse formulations and intrathecal delivery to achieve significant protein knockdown in neurodegenerative diseases, paving a clear path toward clinical development for severe neurological disorders.

Examining Innovative Delivery Strategies for Enhanced Targeting:

Utilize novel ligand platforms, antibody conjugates, and DNA origami nanostructures to achieve precise delivery to muscle, kidney, and adipose tissue, optimizing cellular uptake and endosomal escape to avoid the limitations of conventional LNPs.

Engineering Specificity & Safety Through Chemical Biology:

Apply advanced phosphorothioate patterns and 2′-modifications to transcend GalNAc, actively directing biodistribution and improving therapeutic indices to build safer, more precise RNAi drugs.

Optimizing CMC & Formulation for Commercial Readiness: 

Address the unique synthesis, characterization, and manufacturing challenges of complex conjugates and novel chemistries to ensure quality, stability, and scalability for clinical and commercial supply.

Join discovery to clinical development teams from the top pharma and biotechs to de-risk your path to the clinic and accelerate your extrahepatic pipeline.

Want to find more about registration options? Follow this link to go to the registration platform on our website: https://ter.li/n7bmw8