Key Sessions
Marie Wikstrom-Lindholm
Keynote Panel: The Potential of RNA Therapeutics – Rare vs Chronic Applications
Silence Therapeutics
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Main Conference – Day 1 - CET (Central European Time, GMT+01:00)
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Main Conference – Day 1 - CET (Central European Time, GMT+01:00)
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mRNA & Next-Gen RNA: Translational Therapeutics & Enabling Platforms
Business Forum: Rapid-Fire Innovation Session
12:30 - 13:00
Presentation: Synthetic DNA: The new standard in scalable GMP-grade manufacture for mRNA therapeutics
- 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
11:30 - 11:45
Innovation Presentation: MEVs: A Versatile Delivery Platform for Advanced Therapeutics, Vaccines, and Gene Therapies
- Extracellular vesicles derived from microalgae (MEVs) are being developed as a universal delivery system for therapeutics, vaccines, and gene therapies.
- MEVs demonstrate high performance, safety, and flexibility in delivering diverse payloads, including mRNAs, siRNAs, oligos, plasmids, peptides, and proteins. They can be administered through various non-invasive routes, such as topical, ocular, oral, respiratory, intranasal, intramuscular, and intravenous, enabling delivery to hard-to-reach tissues while addressing limitations of existing delivery systems.
- MEV manufacturing is sustainable, simple, cost-effective, and GMP-ready. Production relies on environmentally friendly processes using light, fresh water, and minerals, ensuring a green and scalable approach.
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RNA Therapeutics: Next-Gen & Pre-Clinical
14:45 - 15:15
Presentation: Efficient Large-scale siRNA Manufacturing: Enzymatic Ligation of Short RNA Fragments
Linear synthesis by continuous-flow SPOS is the current state-of-the-art technology to manufacture oligonucleotide APIs. A very promising alternative, especially to manufacture large quantities, is the enzymatic ligation of short oligonucleotide fragments. This approach offers several advantages compared to the conventional strategy like higher yields and reduced purification burdens. Bachem will present a case study focusing on a therapeutically relevant siRNA sequence and benchmark different technologies used to manufacture siRNA fragments. We will discuss a holistic view of the manufacturing process and review control strategies for residual enzyme
- Sabine Fenner - Director Oligo R&D, bachem
Showing 1 of 1 Streams
mRNA & Next-Gen RNA: Targeted Delivery
16:45 - 17:15
Presentation: mRNA Synthesis & Delivery - Advancing Scalable Solutions for Precision Therapeutics
- Miroslav Gaparek - CEO, Sensible Biotechnologies
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