Partnering Goes Live in
- 00Days
- 00Hrs
- 00Mins
- 00Secs
Main Conference – Day 2 - CET (Central European Time, GMT+01:00)
Our distinguished panelists will share their journeys of building successful biotech ventures, overcoming industry challenges, and translating cutting-edge RNA science into life-changing treatments. Discover the latest developments in RNA therapeutics, learn about emerging opportunities in this rapidly evolving field, and gain insights into what it takes to lead innovation in one of biotech's most promising sectors.
- Paola Pozzi - Partner, Sofinnova Telethon Fund, Sofinnova Partners
- Michelle Lynn Hall - General Partner, Entrée Capital
- Renee Williams - CBO, Signify Bio
- Karina Thorn - Vice President, Head of Nucleic Acid Research, Novo Nordisk
- Sonja Merkas, PhD - Founder, Creator and Doer, Livinovea
- Synthesis and Modifications of RNA Modalities
Overlooked Opportunities in RNA Therapeutics
Translational Challenges in Scaling RNA Medicines
PK/PD Measurement Requirements
Control Elements and UTR Considerations
Limitations of Animal Models and Human Alternatives
Safety versus Efficacy Balance in RNA Therapeutics
AI adoption for Discovery and Target Selection
Enzymatic Approaches in RNA Production
Extrahepatic delivery of RNA cargos
Immunogenicity of Oligonucleotides
- Sonja Merkas, PhD - Founder, Creator and Doer, Livinovea
- Peter Weinstein - Chief Executive Officer, Circurna
- Cesar Lopez-Camacho - Group Leader, University Of Oxford
- Marie Wikstrom-Lindholm - CSO, Silence Therapeutics
- Sudhir Agrawal - President and Founder, Arnay Sciences
- Miroslav Gaparek - CEO, Sensible Biotechnologies
- Steffen Schubert - Vice President- Drug Discovery, Silence Therapeutics
- Nick Lench - Executive Director, Nucleic Acid Therapy Accelerator
- Bogdan Rivoal - CEO, NeXendia
- Arun Kumar - RNA Innovations Programs Leader, CEPI
- Phase 1 study evaluates the safety, tolerability, and pharmacokinetics of QRL-201 in targeting Stathmin-2 for ALS treatment.
- QRL-201 demonstrates potential in restoring Stathmin-2 expression, a critical protein implicated in ALS pathology.
- Early clinical findings provide insights into the therapeutic viability of RNA-based approaches for neurodegenerative diseases like ALS.
Hagen Cramer, CTO, QurAlis
- Hagen Cramer, PhD - Chief Technology Officer, QurAlis
This presentation introduces a robust and scalable enzymatic ligation platform, designed to meet the stringent demands of modern oligonucleotide manufacturing.
Our approach utilizes proprietary, high-performance engineered ligases to assemble RNA constructs with superior precision and yield. The platform is built upon a combination of AI-optimized design and deep process knowledge, ensuring efficiency from sequence design to final product. We achieve consistent high purity through advanced impurity control and employ continuous processing strategies for enhanced productivity and control.
- Xuecheng Jiao, Ph.D - Vice General Manager, Asymchem Labs
- Causeway is developing a platform of microRNA-based therapeutics focused on tissue repair and regeneration. Our lead clinical asset, a proprietary miR-29a mimic, targets tendinopathy, a condition with a massive global burden and limited treatment options. Unlike single-target therapies, our miR-29a mimic exerts a pleiotropic effect, modulating multiple pathways to restore tendon homeostasis. This approach aims to redefine the standard of care for a condition that currently imposes a substantial clinical and financial burden on global healthcare.
- This presentation outlines the translation of TenoMiR®—a first-in-class microRNA-29a mimic—from preclinical discovery to clinical proof-of-concept. We will review the biological mechanism of miR-29a in restoring tendon homeostasis, alongside safety data from Phase 1 and the latest efficacy results from our Phase 2 trial. These clinical datasets demonstrate significant improvements in pain and function, providing the first robust evidence of disease-modifying regenerative potential in human tendon disease.
- Neal Miller - CMO, Causeway Therapeutics
- Paul Nioi, PhD - SVP, Alnylam Pharmaceuticals, Inc.
- Circurna has developed a proprietary circular RNA platform called ciRNA through the use of AI and other technologies that provides for thermal stable nanoparticles that result in efficient transduction of cells in vivo and high protein production following administration.
- Circurna has used novel probe technology to identify the cell populations that take up the ciRNA and express the therapeutic protein in vivo following administration.
- Circurna has developed with Kindeva Drug Delivery novel microneedle patch technology that allows for the efficient targeting of Circurna’s ciRNA to those cells that preferentially take up and express the therapeutic protein.
- Circurna’s ciRNA is robust and durable, lasting considerably longer in vivo than an mRNA expressing the same protein
- Peter Weinstein - Chief Executive Officer, Circurna
- Hans Huber - CEO, Biomay
- Nathaniel Wang, PhD - CEO, Replicate Bioscience
- circVec technology utilizes the process of back-splicing in order to substitute mRNA by engineered, transcribable circular RNA with high intracellular stability leading to enhanced and sustained protein expression
- incorporation of circVec cassettes into AAV or DNA vectors is used to develop more effective and safer gene therapies for both genetic and acquired diseases affecting various organs and tissues in which circular RNA outperforms mRNA
- circular RNA produced from circVec vectors can be loaded intracellularly into and delivered through virus like particles as an alternative to delivery of synthetic circular RNA through lipid nano-particles
- circVec technology has broad potential applications in different therapeutic areas
- Victor Levitsky - CSO, Circio
- 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
TGM-312 represents a groundbreaking approach to MASH treatment, leveraging Tangram Therapeutics' computational platform to silence a novel target. Preclinical studies, including the Gubra GAN DIO-MASH model, demonstrated significant improvements in steatosis and fibrosis progression, both as monotherapy and in combination with emerging therapies. With a low-burden, high-impact dosing profile supported by pharmacodynamic data in NHPs, TGM-312 is poised to transform MASH management. Following the recent submission of a Clinical Trial Application (CTA), Tangram Therapeutics is preparing to initiate a first-in-human study in early 2026, with initial data anticipated in the second half of 2026.
- Alan Whitmore - Chief Scientific Officer, Tangram Therapeutics
- This presentation highlights recent advances in siRNA therapeutics, including preclinical and clinical data demonstrating efficacy in cardiovascular disease. It also presents novel findings on targeted delivery to the kidney and discusses upcoming opportunities and challenges shaping the next generation of RNA-based therapies.
- John Taylor - Global Head of Business Development, Ribocure
RNA platforms enable swift responses to emerging infectious threats. Advances in delivery systems, RNA modalities, thermostability, and formulation have expanded their usability across diverse environments. Improved global access pathways help ensure equitable deployment and enhance overall preparedness.
- Arun Kumar - RNA Innovations Programs Leader, CEPI
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. - Chairman, Heqet Therapeutics
- DM1 is a rare neuromuscular disease with no disease modifying treatments. Arthex’s therapeutic approach in DM1 is miR-23b inhibition, which demonstrated a dual beneficial effect on DM1 models.
- In order to alleviate functional and molecular symptoms of DM1 disease, preferentially in the tissues affected by the disease, Arthex is developing a safe lipid-conjugated antimiR-23b oligonucleotide (ATX-01).
- Explore how the conjugated antimiR reaches muscle, heart, brain and diaphragm more efficiently than the naked molecule in the HSALR mice and shows improved PK/PD properties. Importantly, the level of compound delivered into brain by IV injection in the DMSXL animals was enough to produce important target engagement and concomitant rescue of exploratory behaviour of the mice.
- Diego Piqueras - Research Scientist, ARTHEx Biotech
PART ONE: RNA expansion into Europe, timing mistakes, and execution traps
PART TWO: Platform vs asset coherence as RNA companies scale multiple programs
- Siham Ceballos - Managing Partner, BioArtemis
- Explore the evolving geopolitical landscape and its influence on RNA research, development, and manufacturing across Europe, including industry partnerships and funding dynamics.
- Highlighting the future innovation potential of RNA therapeutics, focusing on next-generation approaches such as RNA editing, non-coding RNAs, and circular RNAs (circRNAs) to address complex diseases and advance precision medicine
- Paul Nioi, PhD - SVP, Alnylam Pharmaceuticals, Inc.
- Dee Datta - Co-founder & CEO, Switch Therapeutics
- Felix Schumacher - Mission Lead RNAHub, Roche
- Michelle Lynn Hall - General Partner, Entrée Capital
- Michael Hodges - CEO, 1Strand
- Gain insights into how investors and pharma are evaluating the full RNA platform, including the effectiveness of RNA drugs and delivery tools, the validity of novel targeted tissues and targets, and the selective advantages of RNA modalities over small molecules or biologics for specific therapeutic applications.
- Highlight what they seek in RNA-focused ventures and how external innovation is shaping the future of this transformative field.
- Paola Pozzi - Partner, Sofinnova Telethon Fund, Sofinnova Partners
- Ivan Burkov - Partner, INKEF Capital
- Sonia Hallen - Investment Manager, Gilde Healthcare
- Ulrik Lytt Rahbek - VP, Innovation & Portfolio Execution in Nucleic Acid Research, Novo Nordisk
- David Hardwicke - Co-Founder, Aerska