Main Conference – Day 1 - CET (Central European Time, GMT+01:00)
- The transformative potential of RNA-based therapies will be explored, focusing on their applications in treating both rare genetic disorders and chronic diseases.
- Experts will discuss the innovative approaches RNA therapeutics offer for targeted treatment, emphasising their ability to address unmet medical needs and improve patient outcomes.
- Scientific, regulatory, and commercial challenges in developing RNA therapies will be examined, alongside breakthroughs that are shaping the future of medicine.
- Marie Wikstrom-Lindholm - SVP and Head of Molecular Design, Silence Therapeutics
- Malgorzata Gonciarz - Global Head xRNA Therapeutics, Novartis
- Nathaniel Wang, PhD - CEO, Replacate Biosciecne
- Review the latest advancements in RNA platforms as of 2025, showcasing key innovations and breakthroughs.
- Clinical milestones achieved with RNA-based therapeutics will be highlighted, offering insights into their impact on medicine.
- The session will provide an overview of the evolving RNA technology landscape and future opportunities in the field.
- Renee Williams - Founder and Managing Partner, Williams Biotech Consulting, LLC
- Haya Therapeutics' pioneering approach to developing lncRNA-targeting therapeutics for precision medicine focuses on their proprietary technology platform, SENIS™. The discussion highlights how lncRNA modulation enables targeted delivery to cardiac tissue, addressing unmet needs in fibrotic and other cardiac diseases.
- The presentation explores preclinical and clinical design data showcasing the efficacy and specificity of Haya Therapeutics' lncRNA-based therapies, including insights into their innovative strategies for identifying and validating disease-specific lncRNA targets.
- Daniel Blessing - CTO, HAYA Therapeutics
• Dysregulated mRNA processing has been identified as a new and druggable molecular hallmark of aging. Age-related changes in splicing factor expression disrupt physiological homeostasis, leading to loss of transcriptomic resilience, cellular senescence, and chronic diseases
• An antisense oligonucleotide approach has been developed to restore splicing regulator expression specifically in senescent cells. This method leverages endogenous autoregulation to achieve physiologically regulated expression of target genes within their normal homeostasis.
• Enabling pharmacokinetic (PK) properties and proof of principle for transcriptomic reprogramming of senescence have been demonstrated in an aged mouse model via an inhalation route.
• Initial applications of this technology target Idiopathic Pulmonary Fibrosis (IPF), a sentinel disease driven by senescence. Results show the ablation of harmful senescent properties in diseased cells and precision-cut lung slices (PCLS) from IPF patients, along with significant reductions in markers of inflammation and fibrosis
.• The approach has shown efficacy in senescent primary human cells across multiple cell lineages, suggesting potential applications for the treatment of various age-related diseases beyond respiratory conditions.
- Lorna Harries - CSO, Senisca
Cardiovascular disease is the leading cause of death worldwide; heart failure carries high mortality, and substantial health-economic burden. Cardiac infarcts can lead to loss of heart function and heart failure. Cardiomyocytes have limited regenerative capacity and current therapies only improve residual function and are not curative.
- Small non-coding microRNAs delivered as synthetic mimics in cardiomyocyte specific lipid nanoparticles can reactivate cardiomyocyte proliferation in the adult heart. Preclinical studies in mice and pigs demonstrate increased cardiomyocyte division, reduced infarct size, and improved function after myocardial infarction.
- The miRNA–LNP approach provides potent, transient, and repeatable effects with efficient myocardial delivery; Compared with AAV, protein and cell therapies, miRNA–LNPs offer better control of dose and optimal duration.
- miRNA-based regenerative therapy represents a promising, scalable strategy for curative treatment against heart failure, warranting further clinical development and safety evaluation.
- Bo Rode Hansen, Ph.D. - Global Head RTR & GM RICC, RTR (RNA Rx Research), Roche Innovation Center Copenhagen, Denmark
• 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.
- Bogdan Rivoal - Group Leader, University Of Zürich
- Focused on enhancing mRNA vaccine platforms, the research explores novel strategies to improve stability, immunogenicity, and delivery efficiency, addressing critical challenges in vaccine development for infectious diseases.
- Investigating the application of mRNA technologies in global health, the group aims to develop scalable and adaptable solutions for rapid vaccine production, targeting emerging and re-emerging pathogens.
- Cesar Lopex - Group Leader, University Of Oxford
- 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.
- Emily Young - Director of Scientific Applications, 4Basebio
- Extracellular vesicles derived from microalgae (MEVs) are being developed as a universal delivery system for therapeutics, vaccines, and gene therapies.
- MEVs demonstrate high performance, safety, and flexibility in delivering diverse payloads, including mRNAs, siRNAs, oligos, plasmids, peptides, and proteins. They can be administered through various non-invasive routes, such as topical, ocular, oral, respiratory, intranasal, intramuscular, and intravenous, enabling delivery to hard-to-reach tissues while addressing limitations of existing delivery systems.
- MEV manufacturing is sustainable, simple, cost-effective, and GMP-ready. Production relies on environmentally friendly processes using light, fresh water, and minerals, ensuring a green and scalable approach.
- Marie-Hélène Leopold - CCO, AGS Therapeutics
- Splice Editors increase trans-splicing efficiency by leveraging CRISPR-Cas systems
- Splice Editor-enabled multi-kilobase edits unlock several key indications and pathogenic variants
- Splice Editing is a promising new modality for RNA editing
- Jacob Borrajo - CEO, Amber Bio
- Developing an innovative RNA splicing modulation platform to selectively target pseudo-exons, addressing genetic mutations that lead to aberrant splicing and disease progression.
- Leveraging advanced technologies to restore normal splicing patterns, enabling precise therapeutic interventions for a range of genetic disorders with high unmet medical needs.
- Poul Sørensen - CEO, Inverna therapeutics
Please contact the RNA Leaders Team (jade.osei-tutu@informa.com) if you are interested in presenting
Please contact the RNA Leaders Team (jade.osei-tutu@informa.com) if you are interested in presenting
- The development of transient cyclic structures in oligonucleotides enhances their drug-like properties by optimizing spatial configurations that bring the 3'- and 5'-ends into proximity.
- These cyclic oligonucleotide structures exhibit reduced protein binding and diminished interactions with pattern recognition receptors due to the inaccessibility of the 5’-end, improving their therapeutic profile.
- Transient cyclic oligonucleotides enable broad applications in RNA therapeutics, including RNaseH-mediated knockdown, splicing modulation, siRNA delivery, and gRNA-based mechanisms.
- Sudhir Agrawal - President and Founder, Arnay Sciences
- Employing transcriptomic approaches to identify and quantify off-target effects in RNA therapeutics, ensuring precision and minimizing unintended interactions.
- Utilizing high-throughput and long-read sequencing technologies to map RNA interactions and assess global gene expression impacts.
- Integrating transcriptomic data with computational models to optimize therapeutic design and improve safety profiles for RNA-based drugs.
- Nicola Guzzi - Associate Principal Scientist, AstraZeneca
- 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.
- Michael Wolfinger - Principal Investigator, University of Vienna
Please contact the RNA Leaders Team (Jennifer.Wickett@informa.com) if you are interested in presenting.
- 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
- Mark Dickman - Professor, University of Sheffield
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
- Developing self-assembling siRNA therapeutics designed for effective and targeted delivery via inhalation to treat respiratory diseases.
- Optimizing siRNA formulations to ensure stability, efficient pulmonary uptake, and minimal off-target effects.
- Demonstrating safety and therapeutic efficacy through advanced preclinical models of respiratory disease
- Philippe Vollmer Barbosa - Project Manager, Fraunhofer
- Addressing the barriers to effective transport of nucleic acid therapeutics, such as stability, cellular uptake, and tissue specificity.
- Exploring lipid nanoparticles, polymer-based carriers, and conjugate systems for precise and efficient delivery.
- Highlighting preclinical and clinical advancements in delivery technologies for nucleic acid-based therapies.
- Discussing emerging approaches, such as extracellular vesicles and targeted delivery systems, to enhance therapeutic outcomes.
- Nick Lench - Executive Director, Nucleic Acid Therapy Accelerator
- Engineering personalized mRNA vaccines customized to individual immune characteristics for improved effectiveness and specificity.
- Enhancing delivery platforms to ensure stability, targeted cellular entry, and strong immune response activation.
- Assessing safety and immunological outcomes through cutting-edge preclinical and clinical evaluations to advance personalized vaccine development.
- Steve Pascolo - Researcher (Former Founder, CureVac), University Hospital of Zurich
- Miroslav Gaparek - CEO, Sensible Biotechnologies
Please contact the RNA Leaders Team (Jennifer.Wickett@informa.com) if you are interested in presenting.
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.