Main Conference – Day 2 - CET (Central European Time, GMT+01:00)
- 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 Millar - CMO, Causeway Therapeutics
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.
- Paul Nioi, PhD - SVP, Alnylam Pharmaceuticals, Inc.
- 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
- 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
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 and brain efficiently and shows improved PK/PD properties. Additionally, see how conjugated oligonucleotide enhances CNS biodistribution following IV and IT administrations.Importantly, the level of compound delivered into brain by IV injection was enough to produce important
- Diego Piqueras-Losilla - Research Scientist, ARTHEx Biotech