Main Conference – Day 1Wednesday, 18 March 2026 - CET (Central European Time, GMT+01:00)

• Cell-free RNAs (cfRNAs), stabilized by extracellular vesicles (EVs), lipoproteins, and RNA-binding proteins (RBPs), are emerging as key players in RNA therapeutics and biomarker research, with a focus on their role in cell-to-cell communication.

• Recent findings emphasize the importance of RBPs in cfRNA biology, highlighting their potential to enhance stability, specificity, and targeted uptake for next-generation RNA therapeutics. cfRNAs also show promise as non-invasive biomarkers for real-time monitoring of therapeutic efficacy and optimizing personalized treatments.

• Integrating cfRNA discoveries into RNA therapeutic pipelines offers the potential to advance individualized treatment strategies, improve patient outcomes, and set new standards in RNA-based medicine.

• Synthetic, enzymatically produced DNA is shaping the future of mRNA manufacture: rapid, scalable GMP-grade production capable of handling complex sequences and long poly A tails without the bacterial contaminants.
• Explore opDNA®, an application-specific IVT template open at the 3’ end, facilitating direct use in the IVT reaction without enzymatic linearization. As a linear template without bacterial backbone sequences, equivalent mRNA yields are achieved with less DNA mass.
• Homologous recombination of polyA tails in bacterial hosts is a major limitation of plasmid DNA. 4basebio’s enzymatic platform can handle long polyA tails (>180 bp) encoded directly into the template, while our novel polyA analytics ensure homogeneity in every construct.
• Clinically validated and regulatory-ready, opDNA® supports efficient, flexible, and compliant manufacturing. This approach can support clinical programs at every scale, from large-scale production to small-batch and scale-out demands of personalized immunotherapies.

• A novel RNA splicing modulation platform that selectively activates pseudo-exons to precisely control gene and protein expression at the source of disease.

• Targeted oligonucleotides function as an innovative molecular switch, displacing repressive RNA-binding proteins to enable pseudo-exon inclusion during splicing, deliberately altering mRNA and downstream protein production.

• Powered by a proprietary map spanning over 90% of spliced protein-coding genes with pseudo-exons, the platform unlocks a vast therapeutic targeting space with broad applicability across many conditions.

• Splice Editors are capable of 70% RNA editing in vivo
• Splice Editor-enabled multi-kilobase edits unlock several key indications and pathogenic variants
• Splice Editing is a promising new modality for RNA editing

• Proprietary LNP platform at Certest Pharma: Development of lipid nanoparticles based on proprietary ionizable lipids that enable efficient nucleic acid delivery and allow modulation of the inherent hepatic tropism of LNPs toward extrahepatic organs, particularly the spleen.
• Peptide-based active targeting technology: Introduction of a recently developed proprietary strategy for the covalent anchoring of peptide ligands onto the surface of LNPs, enabling active targeting and improved tissue and cell selectivity beyond passive biodistribution mechanisms.
• Application to tumor targeting: Demonstration of enhanced tumor accumulation and delivery performance of peptide-functionalized LNPs in mouse cancer models, highlighting the potential of combining advanced ionizable lipid design with active targeting strategies for oncology applications.

• RNA modifications play a critical role in influencing RNA structure and function, yet traditional prediction methods often fail to account for these molecular marks.
• The ViennaRNA Package introduces an advanced approach to RNA structure prediction by integrating modified nucleotides through custom alphabets, tailored energy parameters, and adapted folding algorithms.
• Experimental data on modified bases is incorporated into the prediction process, enhancing the accuracy of RNA structure modeling.
• Case studies highlight the improved prediction accuracy for known modified RNAs, demonstrating the effectiveness of this approach in addressing challenges associated with modified RNA structure analysis.

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.

• Characterisation of mRNA critical quality attributes using LC MS
• Automated online direct mRNA sequencing mapping using partial RNase T1 digests
• Analysis of mRNA multimerisation (aggregation) using ion-pair reversed phase HPLC

PartneringONE Clinic: dedicated support time where attendees can receive personalized assistance setting up meetings and navigating the PartneringONE event platform, ensuring you maximize networking opportunities throughout the RNA Leaders conference

RNA nanotherapeutics have risen to prominence following recent clinical successes, yet their broader potential remains constrained by liver biased biodistribution, a restricted cell tropism, and inadequate cell delivery due to poor endosomal escape. To gain a deeper understanding of the many complex in-vivo mechanisms and to identify the optimal drug characteristics needed to navigate these processes, we must devise a robust platform of assays and models capable of evaluating each relevant property of the drug. Establishing such a comprehensive and predictive preclinical cascade is essential to support the rational design and optimisation of next-generation RNA delivery vehicles, ultimately accelerating the future success of RNA therapeutics. To address this, five UK partner organisations have established the Intracellular Drug Delivery Centre (IDDC), creating a centre of excellence for lipid nanoparticle (LNP) innovation. The centre integrates high-throughput screening of novel lipid chemistries and formulation with mechanistic assessment of key formulations in cell models and small mammals. Our data from thousands of LNP formulations reveals the complexity of their structure-activity relationships, allowing us to map specific biological functions to distinct LNP components. Furthermore, we find that exceptions to the internalisation rupture-expression model highlight as yet unappreciated biological intricacies that control LNP functionality in target cells. Altogether, this work has advanced the rational design of next-generation RNA nanotherapeutics and revealed principles to guide the design of future LNPs.

• Current industry bottlenecks: Why narrow signal peptide testing limits process performance today and how the secretory pathway remains a manufacturing ‘black box’ 

• Common challenges and solutions in signal peptide selection for improving manufacturability 

• Latest technological and computational advances, moving from testing dozens of signal peptides to high-throughput screening of thousands

• Case examples on how choice of signal peptide impacts protein expression

Join a dynamic and engaging discussion where leading investors in the field of nucleic acid therapeutics share their perspectives on strategic funding priorities. This session will delve into the factors driving investment decisions, including emerging technologies, promising therapeutic platforms, and the evolving landscape of RNA and DNA-based treatments.

A key feature of this roundtable will be an open Q&A session, providing attendees with the unique opportunity to ask investors direct questions and seek tailored advice on securing funding, navigating commercialization challenges, and building successful partnerships. Investors will offer insights into the challenges and opportunities shaping the industry, highlight key areas of innovation they are focusing on, and discuss how they evaluate potential ventures.

Gain valuable knowledge on the financial trends influencing the growth of nucleic acid therapeutics and learn what it takes to attract funding in this rapidly advancing field. This interactive format ensures participants leave with actionable strategies and a deeper understanding of investor priorities.