Main Conference – Day 2 - CET (Central European Time, GMT+01:00)
- 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
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
- 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.
