What Can You Expect from the 7th RNAi-Based Therapeutics Summit?
As landmark approvals in hemophilia and a flurry of billion-dollar partnerships for neurology and metabolic diseases validate RNAi's platform potential, the 7th RNAi-Based Therapeutics Summit is the industry’s definitive forum to translate these clinical and business successes into actionable development strategies that conquer extrahepatic delivery.Â
Through 30+ expert-led sessions, explore how pioneers are moving beyond the liver to unlock new disease frontiers:Â
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.Â
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.Â
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.Â
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.Â
Optimizing CMC & Formulation for Commercial Readiness:Â
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.Â
Join discovery to clinical development teams from top pharma and 80+ biotechs to de-risk your path to the clinic and accelerate your extrahepatic pipeline.Â
Unmissable Event HighlightsÂ
Pre-Conference Workshops:
Master the chemical engineering of RNAi constructs to enhance stability and extrahepatic delivery and harness AI-powered in silico tools for target discovery and lead candidate optimization, bridging the gap between preclinical promise and clinical success.
Extrahepatic Delivery Deep Dives:
Explore breakthrough strategies for targeted delivery to CNS, muscle, adipose, and cardiac tissue with novel platforms including antibody conjugates, DNA origami nanostructures, and optimized GalNAc chemistries, to unlock new therapeutic frontiers in neurology, metabolic disease, and beyond.
Clinical & CMC Roadmaps:
Decode the path to commercialization by tackling the unique synthesis, characterization, and regulatory hurdles of complex RNAi modalities, leveraging insights from leaders at Alnylam and AbbVie to de-risk your development strategy from IND filing to scale-up.Â
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Investor & Future-Facing Panels:
Gain critical insights on valuation drivers and pipeline strategy from top VCs and learn how to future-proof your RNAi therapeutics by integrating AI, multi-target silencing, and precision medicine combinations to lead the next wave of innovation.
Core Themes
Extrahepatic Delivery Breakthroughs:
Actionable strategies for targeted RNAi delivery to CNS, muscle, adipose, and cardiac tissue, moving beyond the liver to tackle neurology, metabolic disease, and fibrosis.Â
Clinical & CMC Roadmaps:
Exclusive data and development pathways from Alnylam, AbbVie, and pioneering biotechs on navigating complex synthesis, regulatory hurdles, and late-stage clinical development.
Next-Generation Modalities:
Explore cutting-edge platforms including antibody-oligonucleotide conjugates (AOCs), DNA origami, self-assembling nanostructures, and multi-targeting siRNA/miRNA approaches.Â
Investor & Industry Outlook:
Critical insights from top VCs and a cross-section of industry leaders on valuation drivers, partnership strategies, and future-proofing RNAi pipelines in a competitive landscape.
Explore the Full Event Guide
- 25+ expert speakers from pioneering biotechs and large pharma, including Sanegene Bio, AbbVie, Judo Bio, and more, sharing exclusive data and case studies.Â
- Actionable insights to conquer extrahepatic delivery for CNS, muscle, and cardiac tissues with novel strategies like aptamers, bispecifics, and exosomes.Â
- Dedicated deep-dives on the hottest applications: from obesity and metabolic disease to neurology, oncology, and the latest breakthroughs in microRNA therapeutics.Â
- Critical regulatory and CMC strategies to overcome complex synthesis, scale-up, and IND-enabling hurdles for late-stage clinical success.Â
- 8+ hours of dedicated networking with 80+ industry leaders to forge partnerships with key decision-makers from top RNAi innovators.Â
Attending Companies Include
Explore the Agenda
Hear the latest industry breakthroughs and gain exclusive insights during our packed agenda, interactive roundtables, and panel discussions.
Partner With Us
Position yourself alongside other leading solution providers to ensure your brand is at the heart of biopharma deals.
Join Biopharma Experts
Be part of the hottest conversations with like-minded attendees spanning all stages of drug development and make critical connections during our dedicated networking sessions.