Main Conference – Day 1 - CET (Central European Time, GMT+01:00)
• A novel RNA splicing modulation platform that selectively activates pseudo-exons to precisely control gene and protein expression at the source of disease.
• Targeted oligonucleotides function as an innovative molecular switch, displacing repressive RNA-binding proteins to enable pseudo-exon inclusion during splicing, deliberately altering mRNA and downstream protein production.
• Powered by a proprietary map spanning over 90% of spliced protein-coding genes with pseudo-exons, the platform unlocks a vast therapeutic targeting space with broad applicability across many conditions.
- Poul Sørensen - CEO, Inverna therapeutics
- Splice Editors are capable of 70% RNA editing in vivo
- Splice Editor-enabled multi-kilobase edits unlock several key indications and pathogenic variants
- Splice Editing is a promising new modality for RNA editing
- Jacob Borrajo - CEO, Amber Bio
- Proprietary LNP platform at Certest Pharma: Development of lipid nanoparticles based on proprietary ionizable lipids that enable efficient nucleic acid delivery and allow modulation of the inherent hepatic tropism of LNPs toward extrahepatic organs, particularly the spleen.
- Peptide-based active targeting technology: Introduction of a recently developed proprietary strategy for the covalent anchoring of peptide ligands onto the surface of LNPs, enabling active targeting and improved tissue and cell selectivity beyond passive biodistribution mechanisms.
- Application to tumor targeting: Demonstration of enhanced tumor accumulation and delivery performance of peptide-functionalized LNPs in mouse cancer models, highlighting the potential of combining advanced ionizable lipid design with active targeting strategies for oncology applications.
- Alejandro Mahía Moros - Senior Researcher, Certest
- Innovative shielding lipids enabling superior RNA delivery: Curapath’s next‑generation shielding lipids -including polyoxazolines and proprietary polypeptoid and polypeptide derivatives- demonstrate enhanced mRNA expression compared to PEG‑based LNPs, highlighting the meaningful impact of optimized shielding lipids on RNA delivery systems.
- Stability, integrity, and immunogenicity: We evaluated the physicochemical stability, freeze‑drying compatibility, and immunogenicity profiles of our proprietary shielding‑lipid‑based LNPs. The results show robust structural integrity post‑lyophilization and low immunogenicity, critical attributes for emerging RNA therapeutics and vaccines.
- Enabling active targeting through modular functionalization: Using Curapath’s licensed click‑chemistry conjugation technology, we demonstrate precise and efficient surface functionalization of LNPs, enabling tunable, receptor‑specific targeting strategies and expanding LNP utility across diverse therapeutic applications.
- Víctor R. de la Rosa - Commercial Product Manager, CURAPATH
- Solving the Delivery Bottleneck: Introduction of a patented peptide-based platform achieving >99% selective delivery to lymphoid organs with negligible liver accumulation (<1%), bypassing the primary dose-limiting toxicity of current LNP technologies.
- Superior Immunogenic Potency: Comparative data demonstrating a 10-fold increase in antigen-specific killer T-cell activation versus industry-standard benchmarks, significantly widening the therapeutic window for mRNA-based vaccines.
- Neutralizing the 'Cold' Tumor Shield: Application of a dual-action strategy in MSS Colorectal Cancer that simultaneously targets multiple cancer and stromal antigens to dismantle the immunosuppressive tumor microenvironment and drive clinical efficacy.
- Kaido Kurrikoff - CEO, Vectiopep
Data will be presented validating two Argonaute dual targeting siRNAs (bis-siRNAs), respectively addressing residual risk/unmet need for ASCVD, including heart attacks and stroke, and fatty liver diseases (MASLD/MASH). We will present compelling and comprehensive results from in vitro and large in vivo studies, including extensive biochemical, gene expression and metabolomics assays demonstrating the extraordinary efficacy and safety of these agents. Together a strong case will be made for pursuing clinical development of these agents with the aim of launching first in man studies in the next 12-18 months.
- Michael Khan - CEO, ARGONAUTE RNA LIMITED
