Main Conference – Day 1 - CET (Central European Time, GMT+01:00)
Chemically synthesized RNA oligonucleotides (oligos) have the potential of a powerful, multifaceted approach against cancer by acting as immune stimulators, RNA interference agents, and vaccines, as well as drug carriers. This presentation will first detail the simple chemical structure of RNA oligos that can be used for cancer vaccination. We will then introduce and discuss a new type of chemotherapeutic anticancer RNA oligo currently under development.
- Steve Pascolo - Researcher (Former Founder, CureVac), University Hospital of Zurich
- Rapidly growing therapeutic oligonucleotide demand for indications with large patient populations require innovative manufacturing solutions. Answering the call for a more scalable and sustainable process, Codexis introduced the ECO Synthesis® Manufacturing Platform — a fully aqueous, enzymatic high yielding production approach for safe, quality, and efficacious siRNA. In this presentation, we will demonstrate the scalability of ECO Synthesis while illustrating the ability to transition from research to manufacturing scale and cGMP production, highlighting its potential to exceed productivity of traditional SPOS.
- Robust in-process control (IPC) testing is critical to achieving consistent product quality and purity in oligonucleotide manufacturing. Conventional solid-phase oligonucleotide synthesis (SPOS) presents IPC testing limitations due to the closed reaction environment and resin-bound intermediates. This platform, ECO Synthesis, enables early detection of process performance and/or deviations, and the opportunity for closed loop adjustment of reaction parameters in the future, resulting in high standards of API product quality, ensuring patient safety.
- David Entwistle - Sr Director, Program & Pipeline Management, Pharma Manufacturing, Codexis
• Dysregulated mRNA processing has been identified as a new and druggable molecular hallmark of aging. Age-related changes in splicing factor expression disrupt physiological homeostasis, leading to loss of transcriptomic resilience, cellular senescence, and chronic diseases
• An antisense oligonucleotide approach has been developed to restore splicing regulator expression specifically in senescent cells. This method leverages endogenous autoregulation to achieve physiologically regulated expression of target genes within their normal homeostasis.
• Enabling pharmacokinetic (PK) properties and proof of principle for transcriptomic reprogramming of senescence have been demonstrated in an aged mouse model via an inhalation route.
• Initial applications of this technology target Idiopathic Pulmonary Fibrosis (IPF), a sentinel disease driven by senescence. Results show the ablation of harmful senescent properties in diseased cells and precision-cut lung slices (PCLS) from IPF patients, along with significant reductions in markers of inflammation and fibrosis
.• The approach has shown efficacy in senescent primary human cells across multiple cell lineages, suggesting potential applications for the treatment of various age-related diseases beyond respiratory conditions.
- Lorna Harries - CSO, ISCARNA
- Demonstrating Abogen's unique mRNA and LNP platform
- Sharing clinical data and progress of mRNA cancer vaccines
- Accelerating preclinical and clinical development of mRNA-based T cell engagers and in vivo CAR-T
- Bo Ying - CEO, Abogen Biosciences
- Synthetic, enzymatically produced DNA is shaping the future of mRNA manufacture: rapid, scalable GMP-grade production capable of handling complex sequences and long poly A tails without the bacterial contaminants.
- Explore opDNA®, an application-specific IVT template open at the 3’ end, facilitating direct use in the IVT reaction without enzymatic linearization. As a linear template without bacterial backbone sequences, equivalent mRNA yields are achieved with less DNA mass.
- Homologous recombination of polyA tails in bacterial hosts is a major limitation of plasmid DNA. 4basebio’s enzymatic platform can handle long polyA tails (>180 bp) encoded directly into the template, while our novel polyA analytics ensure homogeneity in every construct.
- Clinically validated and regulatory-ready, opDNA® supports efficient, flexible, and compliant manufacturing. This approach can support clinical programs at every scale, from large-scale production to small-batch and scale-out demands of personalized immunotherapies.
- Emily Young - Director of Scientific Applications, 4Basebio
- Develop modular mRNA vaccine platforms targeting priority tick-borne pathogens affecting both livestock and humans (e.g., Babesia, Anaplasma, and selected arboviruses), enabling rapid antigen substitution and comparative evaluation across species.
- Integrate anti-tick and anti-pathogen immunogens within single or combinatorial mRNA constructs to reduce vector competence, interrupt transmission cycles, and decrease reliance on acaricides, aligning with One Health control strategies.
- Establish end-to-end workflows spanning antigen design, in vitro transcription, LNP formulation, and preclinical validation in relevant animal models to generate translationally actionable efficacy and immunogenicity
- Cesar Lopez-Camacho - Group Leader, University Of Oxford