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
- Malgorzata Gonciarz - Global Head xRNA Therapeutics, Novartis
- Tamar Grossman - CEO, La Jolla Labs
- 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
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.
- Nathaniel Wang, PhD - CEO, Replicate Biosciecne
- 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
- AI-driven methodologies enable the discovery of novel mechanisms in RNA-based therapeutic development.
- Advanced computational tools accelerate the design and optimization of RNA therapeutics for enhanced efficacy.
- Integrating AI with RNA research unlocks innovative approaches to target complex diseases with precision.
- Tamar Grossman - CEO, La Jolla Labs
- 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
This presentation will explore the transformative role of artificial intelligence (AI) in advancing circular RNA therapeutics. Key highlights include the use of AI to design next-generation, room-temperature stable nanoparticles in just 30 days—an achievement that previously took years. The talk will also cover AI-driven optimization of RNA sequences to enhance protein production and therapeutic efficacy, as well as its application in analyzing 10x genomics data to map RNA molecule distribution and protein expression. By leveraging AI, the development timeline for circular RNA platforms is being drastically reduced, paving the way for breakthroughs in oncology, autoimmune, and fibrosis treatments. Attendees will gain a deeper understanding of how AI is reshaping the future of RNA-based medicine, enabling faster, more cost-effective, and targeted therapeutic solutions.
- Peter Weinstein - Chief Executive Officer, Circurna
This forum will provide emerging leaders in RNA therapeutics with essential insights into intellectual property (IP) management and commercial strategy. Discussions will focus on navigating the complexities of IP protection for RNA technologies, identifying opportunities for market differentiation, and developing robust commercialization plans.
The forum will highlight examples of next-generation RNA therapeutics, such as self-amplifying RNA (saRNA), circular RNA (circRNA), and RNA editing technologies like CRISPR-Cas systems. Additionally, it will explore advancements in delivery tools, including lipid nanoparticles (LNPs), polymer-based carriers, and exosome-based delivery systems, which are critical for improving the precision and efficacy of RNA-based treatments.
Attendees will gain practical knowledge on how to align scientific innovation with strategic business objectives, secure competitive advantages, and position next-generation RNA therapeutics for long-term success in a rapidly evolving industry.
- Phase II/III clinical trial data demonstrate the efficacy, safety, and tolerability of Mivelsiran in treating hyperoxaluria.
- The RNAi-based mechanism of action of Mivelsiran reduces oxalate levels and addresses disease pathology.
- Biomarker-driven results highlight the potential for improved patient outcomes and quality of life in hyperoxaluria.
- Paul Nioi, PhD - SVP, Alnylam Pharmaceuticals, Inc.
- 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
Arcturus Therapeutics’ STARR® self-amplifying mRNA platform (sa-mRNA) enables potent, long-lasting immune responses with lower mRNA doses than conventional mRNA
The development and regulatory milestones of Kostaive, the world’s first approved sa-mRNA COVID-19 vaccine
The advantages and opportunities of sa-mRNA platform for next generation vaccines against infectious 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.
- 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.
- Beatriz Llamusi, PhD - Chief Executive Officer & Co Founder, Arthex Biotech S.L.
This forum brings together C-suite and senior leaders in RNA therapeutics to discuss strategies for bridging scientific innovation with business leadership. Explore how to build the right team, foster impactful partnerships, and navigate the challenges of scaling RNA technologies from research to commercialization. Gain valuable insights into aligning scientific goals with corporate strategy to drive success in the rapidly evolving RNA landscape.
- 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 - Program Leader Targeted Therapeutics, Roche
- 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
- Sanjay Mistry - Head of Alternative Credit, Pension Protection Fund