Main Conference – Day 2Thursday, 19 March 2026 - 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.

• 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

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.

• 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.

RNA interference–mediated reduction of hepatic plasminogen (PLG) represents a novel approach to inhibit fibrinolysis and improve hemostasis across bleeding disorders. To assess thrombotic safety, human genetic and proteomic analyses from the UK Biobank were integrated with murine thrombosis models and early clinical pharmacodynamic data. Low PLG levels were not associated with increased thrombotic risk in population analyses, and profound PLG knockdown in mice did not accelerate thrombus formation. Initial clinical findings demonstrated antifibrinolytic activity following investigational PLG-targeting siRNA administration. These results support continued clinical development of PLG RNAi therapeutics for bleeding disorders.

• HAYA Therapeutics is pioneering a new generation of programmable RNA therapies by targeting long non-coding RNAs (lncRNAs) to reverse disease-driving cell states and restore cellular health. This session highlights HAYA’s proprietary Regulatory Genome platform, an innovative engine used to identify, track, and design precision therapeutics against disease-causing lncRNAs.
• This presentation explores preclinical data for HTX-001, a first-in-class antisense oligonucleotide (ASO) targeting the lncRNA Wisper. By selectively reverting pathological cardiac myofibroblasts to a healthy state, HTX-001 offers a novel treatment for cardiac fibrosis, a common pathology process across multiple heart diseases.

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.

• 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.

• 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
• Explore the evolving geopolitical landscape and its influence on RNA research, development, and manufacturing across Europe and globally, including industry partnerships and funding dynamics

• 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.