Thursday, September 24Thursday, 24 September 2026 - EST (Eastern Time, UTC-05:00

In-cell selection methodology for identifying small molecules that bind to RNA targets within living cells, enabling discovery of compounds that can effectively reach and engage RNA in the complex cellular environment rather than only in vitro conditions

Application of this approach to develop RNA-targeted therapeutics with improved cellular activity and specificity

Exploring small nucleolar RNAs (snoRNAs) as programmable tools to control protein localization within cells, leveraging the natural RNA-guided mechanisms of snoRNAs to direct proteins to specific subcellular compartments

Extending this snoRNA-based system to regulate protein localization both intracellularly and extracellularly, offering a novel platform for spatial control of protein function with potential applications in targeted therapeutics and cellular engineering

Exploring development of covalent chemical strategies to selectively target RNA molecules.

Application of covalent modification approaches to overcome selectivity challenges in RNA-targeted drug discovery, enabling more precise targeting of disease-relevant RNAs while minimizing off-target effects

Please contact Jennifer Wickett for more information: Jennifer.Wickett@informa.com

Development of mRNA-based immunotherapy platforms specifically designed to treat adult solid tumors and pediatric sarcomas, utilizing messenger RNA technology to stimulate immune responses against cancer cells in these challenging-to-treat malignancies

Development of self-amplifying RNA (saRNA) therapeutics with chemical modifications and sequence optimization to achieve sustained, durable protein expression.

Manufacturing success of RNA therapeutics depends on precise control of quality of raw materials, in vitro transcription (IVT) reaction conditions and purification strategies that apply across constructs. The presentation will outline a conceptual framework for mRNA and saRNA production that integrates rapid at-line analytics to monitor IVT kinetics, enabling data-driven endpoint selection and feed strategies that raise yield and batch-to-batch consistency. A Quality by Design approach maps critical IVT factors—NTP/Mg2+ ratio, polymerase selection, temperature and time, template design, and capping strategy—to critical quality attributes (CQAs) and cost, defining a robust design space that optimizes productivity without compromising quality.

Downstream processing requires scalable, high recovery purification tools and techniques that effectively remove double-stranded RNA, RNA fragments, residual reaction components (NTPs, T7), to reduce immunogenicity and increase cellular potency for both mRNA and saRNA. Affinity, multimodal and reverse-phase chromatography each offer technical advantages and practical challenges that have to be understood and managed in manufacturing environments.

Universal requirements (integrity, dsRNA burden, residuals) and format-specific needs (capping for mRNA; long-transcript handling and hydrolytic potential of saRNA) demand decision frameworks for selecting appropriate IVT strategies and purification approaches. The ultimate goal is a manufacturing approach that scales efficiently and accelerates onboarding of new RNA constructs—translating bench-top control into reliable, multi-product production platform.

Exploration of circRNA as an emerging therapeutic modality with advantages over linear RNA, including enhanced stability due to resistance to degradation, prolonged expression, and reduced immunogenicity.

Discussion of circRNA design, production, and delivery challenges alongside clinical translation opportunities

Exploring the application of transformer-based language models to mRNA design, demonstrating how AI can accelerate the optimization of codon sequences for improved vaccine and therapeutic performance.

Development of stabilized nanoparticle formulations designed to enable multiple routes of mRNA delivery beyond traditional intravenous administration, including inhaled, topical, and other localized delivery methods to expand therapeutic applications.

Engineering nanoparticle platforms with enhanced stability and tissue-specific targeting capabilities, allowing mRNA therapeutics to be delivered directly to disease sites through diverse administration routes while maintaining cargo integrity.

Development of tissue-specific lipid nanoparticle (LNP) formulations engineered to selectively deliver mRNA therapeutics to muscle tissue, bone marrow, and immune cells, utilizing targeted delivery strategies to enhance therapeutic efficacy and reduce systemic exposure

• From screening early-stage nucleic acid payloads up to GMP-scale production, researchers need tools that grow up with their workflow and keep the process consistent.

•Sunshine and Stunner AF team up to cover the critical steps in LNP development

•See how this duo helps you hit the ground running with your first gene therapy, reduces hands-on time, and delivers more meaningful sample