RNAi-Based Therapeutics Summit 2026
Nucleic Acid Insights 2026; 3(1), 1–3
DOI: 10.18609/nuc.2026.001
As part of our ongoing coverage of major gatherings in the nucleic acid therapeutics space, Nucleic Acid Insights presents a preview of the RNAi-Based Therapeutics Summit 2026. Taking place January 27–29, 2026, in Boston, Massachusetts, the summit will convene stakeholders from across the RNA interference (RNAi) ecosystem to examine how small interfering RNA (siRNA) and microRNA therapeutics are progressing beyond established hepatic indications toward broader systemic and extrahepatic applications.
Against a backdrop of recent regulatory approvals, late-stage clinical advances, and major strategic partnerships, the meeting will focus on translating RNAi innovation into durable clinical and commercial success. With a strong emphasis on delivery technologies, novel chemistries, and emerging disease areas, the summit provides a forum to address the scientific and operational challenges that continue to shape RNAi development.
Extrahepatic delivery: expanding RNAi beyond the liver
A central theme of the 2026 agenda is the advancement of RNAi delivery strategies capable of targeting tissues beyond the liver, including the kidney, muscle, adipose tissue, and central nervous system (CNS). Sessions will examine how conjugates, ligand-based targeting, and non-lipid nanoparticle (LNP) approaches are redefining the therapeutic reach of RNAi.
David Jackson (CEO, Ceria Therapeutics) will present a novel RNAi delivery platform designed for extrahepatic targeting, outlining how unique uptake and release kinetics can enable acute RNAi therapeutics in previously inaccessible tissues. He will also discuss the use of selective translational models to de-risk development and accelerate pipeline progression. Alfica Sehgal (CSO, Judo Biosciences) will highlight the STRIKE™ platform, which employs proprietary ligand–siRNA conjugates to achieve receptor-mediated uptake in kidney cells. Her presentation will explore how targeted delivery minimizes systemic exposure while enabling potent and durable gene silencing for renal and systemic diseases. Audrey Bernstein (CSO, DUB Therapeutics) will focus on self-delivering siRNA technologies for fibrotic disease, describing how targeting integrin pathways can reverse pathological scarring and promote regenerative healing in chronic fibrosis models.
RNAi across diverse disease indications
The summit will showcase how RNAi is being applied across a widening range of disease areas, from metabolic disorders to immunology, fibrosis, and neurodegeneration. Andrew Coles (Senior Scientist, AbbVie) will discuss the application of RNAi in complex immunological and fibrotic conditions, examining how targeted LNP engineering and optimized siRNA design can improve biodistribution and therapeutic response. Uyanga Tsedev (CSO, Gensaic) will present an AI-designed, dual-targeting siRNA therapeutic for sarcopenia. Weimin Wang (Founder and CEO, Sangene Bio) will share insights into the LEAD™ RNAi technology platform for obesity and metabolic disease. His session will explore ligand-enhanced delivery strategies designed to achieve durable, tissue-specific silencing across metabolic compartments. Yacoub Habib (CEO, Ophidion Inc.) will address systemic RNAi delivery to the brain, describing Trojan Horse formulations that enhance blood–brain barrier penetration and enable the treatment of neurodegenerative diseases associated with gain-of-function mutations.
Novel delivery architectures: RNA & DNA origami
Emerging delivery vehicles based on programmable nucleic acid architectures will also feature prominently, reflecting growing interest in non-traditional RNAi carriers. Claire Zeng (Chief Executive and Technology Officer, DoriNano) will explore DNA origami-assisted siRNA delivery, detailing how multivalent, folded DNA nanostructures can be engineered to optimize cellular uptake and endosomal escape. Her presentation will demonstrate how modular nanoscale design enables precision delivery while overcoming limitations associated with conventional lipid-based systems. James Carroll (President and CEO, RNA NanoBiotics) will discuss self-assembling RNA nanostructures, highlighting how RNA origami can function as a programmable nanocarrier to improve tissue specificity and payload delivery efficiency.
Investment, integration, & the future of RNAi
In addition to presentations, the summit will feature dedicated panel discussions addressing the evolving RNAi investment landscape and the integration of emerging technologies. A panel focused on investor perspectives will examine how delivery platform versatility, clinical differentiation, and manufacturability influence asset valuation and partnership potential. Another forward-looking discussion will explore how RNAi pipelines can be future-proofed through AI-enabled target discovery, multi-target silencing strategies, and combination approaches with other therapeutic modalities. Together, these sessions will provide strategic context for aligning scientific innovation with long-term development and commercialization goals.
As the premier industry-led forum dedicated to translating RNAi beyond the liver, the 7th RNAi-Based Therapeutics Summit is the definitive meeting for 80+ senior leaders from large pharma, innovative biotech, and investment firms driving the next wave of siRNA and microRNA drugs for neurology, cardiometabolic, oncology, and rare diseases. This year’s conversation goes beyond the liver, focusing on the novel strategies turning extra-hepatic delivery into a clinical reality, from next-generation LNPs and conjugates to emerging platforms targeting the lungs, CNS, and skeletal muscle. You can find out more about the RNAi-Based Therapeutics Summit events here.
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